|
|
|
FDA
Home Page | Search FDA Site |
FDA A-Z Index | Contact FDA
Orphan Products are for diseases that affect few people. Researchers studying these diseases often have difficulty finding enough people who have the disease and are eligible and willing to participate in their studies. Below are listed studies that are funded by the Orphan Products Grants Program for which the investigators are seeking research subjects. If you have the disease or condition, meet the criteria mentioned, and are interested or think you may be interested in participating in the study, contact the person mentioned in the entry.
Disease/Condition: Liver Disease Associate with TPN
Description: This study is for patients aged 18-85 male or female who require long term home TPN that have abnormally elevated liver tests and fatty liver (hepatic steatosis). Patients may be eligible if they have received TPN nightly for at least 12 weeks and it supplies a minimum of 85% of required daily calories. Patients who will require TPN for less than 85% of the daily nutritional needs, will require TPN for less than 34 weeks, have AIDS, active malignancy, liver or kidney failure, are not eligible for inclusion. Eligible patients who have hepatic steatosis or a screening CT scan will be randomized to have choline chloride to their usual TPN or placebo. Follow-up examinations include blood tests and CT scans. Patients must be medically stable to come to Houston, TX. Where possible, and necessary commercial air flight and overnight hotel accommodations may be provided without charge.
Contact Person: Alan L. Buchman, M.D., M.S.P.H.
Baylor College of Medicine
6550 Fannin, SM 1122
Houston, TX 77030
Telephone: (713) 790-5808
Fax: (713) 790-6216
E-mail: ABuchman@BCM.TMC.EDU
Disease/Condition: Neuroblastoma
Description: N7: Maximal Chemotherapy Dose Intensity Plus Monoclonal Antibody 3F8 to increase the cure rate of children with stage 4 neuroblastoma. Indication: Stage 4 neuroblastoma in children greater than or equal to 1 year old at diagnosis. Treatment: Induction therapy consists of high dose noncross-resistant drug combinations (cyclophosphamide/Adriamycin/vincristine, cisplatin/Etoposide) intended to achieve rapid remission. Stem cells are harvested. Surgery and external radiation are used for local control. Consolidation utilizes the monoclonal antibody 3F8 to target iodine-131 to radiate residual tumor sites, followed by stem cell rescue. Unlabeled 3F8, which kills tumor cells via interaction with complement and leukocytes, is infused to eradicate minimal residual disease. 3F8 is specific for ganglioside GD2, a tumor antigen on neuroblastoma. Inclusion criteria: Children greater than or equal to 1 year old at diagnosis with histologically proven GD2 positive neuroblastoma and evaluable disease.
Contact Person: Nai-Kong V. Cheung, M.D., or Brian H. Kushner
Memorial Sloan-Kettering Cancer Center
1275 York Avenue
New York, NY 10021
Telephone: (212) 639-8401
Fax: (212) 744-2245
E-mail: cheungn@mskcc.org
Disease/Condition: Common Variable Immunodeficiency
Description: This is a phase II/III of an investigational treatment for hypogammaglobulinemia. Individuals with common variable immunodeficiency (a congenital immunodeficiency disease giving low serum immunoglobulin levels) will be treated in a controlled randomized study, with weekly self-administered subcutaneous injections of polyethylene glycol-interleukin 2, to stimulate T-Cell immunity and perhaps antibody production. The study will last for 18 months and will include collection of diary information regarding illnesses as well as immunologic information. To be included, potential participants must be over 2 years of age, diagnosed with common variable immunodeficiency, HIV negative.
Contact Person: Charlotte Cunningham-Rundles, M.D., Ph.D.
Mount Sinai Medical
Center
One Gustave Levy Place
New York, NY 10029
Telephone: (212) 241-4014
Fax: (212) 987-593
E-mail: ccunnin@smt.plink.mssm.edu
Disease/Condition: Spinal Cord Injury - Ventilator Dependent
Description: Dr. Anthony F. DiMarco, in affiliation with Case Western Reserve University and Metro Health Medical Center, in Cleveland, OH, is directing a research project that provides respiration by stimulation of the rib cage muscles and diaphragm. Potential candidate must be between the ages of 18 and 50 and dependent on mechanical ventilation due to a spinal cord injury. All treatment and follow-up are free. This treatment may provide freedom from mechanical ventilation.
Contact Person: Janet Petro,
RRT,
Research Assistant
MetroHealth Medical Center
Pulmonary Division
2500 MetroHealth Drive
Cleveland, OH 44109
Telephone: (216) 778-3612
Fax: (216) 778-8215
E-mail: jan2@po.cwru.edu
Disease/Condition: Familial Amyloidtic Polyneuropathy
Description: This study has been initiated to determine if DMSA (dimercaptosuccinic acid) is safe in the treatment of Familial Amyloidotic Polyneuropathy (FAP). FAP is an autosomal dominant neurodegenerative disorder characterized by deposition of amyloid fibrils in peripheral nerves and various organs. Onset is in adult life and the course is invariably fatal. There is no medical treatment for FAP; however, liver transplantation has been successful in preventing disease progression in many cases. FAP is an orphan disease, with approximately 2,000 estimated affected individuals in the United States. We have discovered that TTR-amyloid, the protein fibril that is built up in the body as a result of FAP, can be partially solubilized in vitro by DMSA. In the United States, Chemet (trade name for DMSA) is FDA approved for use in lead poisoning and has been administered for this purpose for a period no greateer than 3 weeks. The effects of ingesting Chemet for longer periods of time are unknown. The main objective of this study is to assess the safety of long-term treatment of Chemet, although we will also monitor several measures of efficacy, such as nerve conduction studies. If the results of the study indicate that Chemet is safe, we plan to initiate another, prolonged study to investigate its efficacy in the treatment of FAP. The total study period is 15 months. The participant is required to take Chemet in pill form, for a period of 12 months and will be monitored closely. Eligibility criteria: Diagnosis of Familial Amyloidosis with positive gene analysis, presence of decrease in sensory or motor function of arms, hands, legs or feet, must not have kidney disease or unstable heart disease.
Contact Person: Joseph Herbert, M.D., Associate Professor of Neuro
NYU/Hospital
for Joint Diseases
301 East 17th Street, Room 1615
New York, NY 10003
Telephone: (212) 598-6305
Fax: (212) 598-6047
E-mail:
Disease/Condition: Giant Cell Arteritis, Temporal Arteritis, Cranial Arteritis
Description: The 3 names for this disease are all synonymous. They describe a process in which there is inflammation of blood vessels, usually of large and medium caliber. This condition generally affects individuals 50 years of age or greater. It usually responds dramatically to treatment of high doses of corticosteroids (e.g. prednisone), but long term treatment is often associated with severe side effects. Thirty to 50% of patients may experience permanent side effects including cataracts, fractures, and accelerated atherosclerosis. In some instances, treatment may aggravate hypertension or precipitate diabetes. The International Network for the Study of Systemic Vasculitides includes 25 medical centers around the world. This network is exploring the utility of adding methotrexate to the usual treatment with corticosteroids, to determine whether methotrexate may lead to more successful reduction of corticosteroid therapy, shorter courses of therapy and fewer side effects. To be included in this study, patients should not have received prolonged therapy with corticosteroids for more than 20 days prior to study entry. Although biopsy proof of giant cell arteritis is desirable, it is not essential for patients with classical clinical features. Patients would be excluded if they had a history of noncompliance, liver disease or recently diagnosed malignancy.
Contact Person: Gary S. Hoffman, M.D., or Carol Tuggle, R.N.
Cleveland
Clinic A50
9500 Euclid Avenue
Cleveland, OH 44195
Telephone: (216) 445-6996
Fax: (216) 445-7569
E-mail: hoffmag@cesmtp.ccf.org
Disease/Condition: Body Dysmorphic Disorder
Description: Body dysmorphic disorder (BDD) is characterized by a preoccupation with imagined defect in appearance, leading to unnecessary cosmetic surgery and significant distress or impairment in social, occupational or other important areas of functioning. If a slight physical anomaly exists, the person's concern is markedly excessive. The purpose of this research study is to examine the effect of clomipramine and desipramine on BDD. Clomipramine (Anafranil) is a marketed medication approved by the Food and Drug Administration for the treatment of obsessive compulsive disorder and desipramine (Norpramin) for treatment of depression. Neither medication is currently approved for the treatment of BDD. In order to determine whether either medication is effective in treating BDD, subjects will be treated with both medications consecutively. Subjects will receive 10 weeks of the first medication and 8 weeks of the other, during which time, the subject will not know which of the two medications he/she is on. Subjects are scheduled for clinic visits with a study psychiatrist every one to two weeks, at which time medication is adjusted/monitored and several rating scales measuring type, severity, and change (if any) of BDD symptoms are administered.
Contact Person: Eric Hollander, M.D. or Jee Kwon
Mount Sinai Medical Center/Psychiatry
Box 1230
One Gustave L. Levy Place
New York, NY 10029
Telephone: (212) 241-3176
Fax: (212) 987-4031
E-mail:
Disease/Condition: Short Bowel Syndrome (SBS)
Description: Randomized, prospective, double-blind trial to determine if growth hormone (GH) enhances nutrient absorption allowing TPN dependent individuals to be removed from their parenteral nutrition. Eligible patients will be admitted to Brigham and Women's Hospital for 4-6 weeks. Patients will be placed on a standardized oral diet with Glutamine-enriched TPN, and undergo nutrient balance, nutrient absorption and body composition studies weeks 1 and 4. During weeks 2 through 6, patients will be randomized to receive either daily injections of GH or placebo. Attempts to wean off TPN will be made after week 4. Inclusion criteria: 1) SBS secondary to vascular occlusive disease, malrotation, volvulus or inflammatory bowel disease, 2) >1.5 years from resection, 3) TPN composes > 50% of estimate caloric needs, 4) >15 cm of jejunum/ileum, without fistula, 5) 18-70 years old, 6) weight within 10% of ideal body weight, 7) no nutrient deficiencies and 8) no significant systemic diseases.
Contact Person: Michael S. Wong, M.D.
Laboratory for Surgical Metabolism
and Nutrition
20 Shattuck Street, Thorn Building, Room 1403
Boston, MA 02115
Telephone: (617) 732-5284
Fax: (617) 732-8305
E-mail: mwong3@bics.bwh.harvard.edu
Disease/Condition: Hodgkins Disease, NHL, CML, and AML
Description: Phase II clinical trials using humanized anti-tac antibody against the interleukin 2 receptor (IL2R-alpha, CD25, Tac) in the treatment of lymphohematopoetic malignancies. All diseases must demonstrate Tac (CD25) on the malignant cells by flow cytometry, except Hodgkins' Disease. The Reed-Sternberg cells are known to be uniformly positive for this market. All patients must have measurable disease by physical exam, radiologic criteria and/or peripheral blood malignant cell count. Other entry criteria are PS-02, not pregnant, 18 years or older, life expectancy greater than 2 months, and no other severe medical condition. The treatment is a rapid IV infusion of antibody in our outpatient clinics. Side effects are minimal or none and pre-medication is not required. The treatment is approximately once per week to two weeks depending on the half-life of drug in the patient. Patients are evaluated for response or non-response at the end of one month. If patients respond to therapy, they will be allowed to continue treatment.
Contact Person: Heather Morein
Beth Israel Deaconess Medical Center
Harvard Medical School
99 Brookline Avenue, Room 387
Boston, MA 02215
Telephone: (617) 632-0316
Fax: (617) 632-0998
E-mail:
Disease/Condition: Erythropoietic Protoporphyria
Description: The Erythropoietic Protoporphyria Research and Education Fund (EPPREF) is a support group for patients with erythropoietic protoporphyria (EPP) which also acts as a registry of patients with EPP. Membership in EPPREF is free: we publish a newsletter for our members, "EPPREF NEWS" in which we publish copying tips and other information of use to people with EPP. We also list research studies which are looking for EPP people; we publish a description of the study and the name, address, and telephone number of the study director. Our registry maintains your confidentiality. Our mailing list is not given out. It is up to EPPREF NEWS readers to contact the directors of studies in which they wish to participate. We invite all United States EPP patients to join EPPREF (there are support groups in other countries with which we should not compete for members.
Contact Person: Micheline M. Mathews-Roth, M.D.
Harvard Medical School
Channing Laboratory
181 Longwood Avenue
Boston, MA 02115
Telephone: (617) 525-2249
Fax: (617) 731-1541
E-mail: mmmathroth@bics.bwh.harvard.edu
Disease/Condition: Hematological Maligancies, lymphoma
Description: Our study is to give interleukin 2 activated cells in the context of bone marrow transplantation for hematologic malignancies, specifically lymphoma. Patients will be included who have good end organ function such as cardiac, pulmonary, liver and renal. Patients will be having lymphoma that has relapsed and is beyond first complete remission.
Contact Person: Amitabha Mazumder, M.D.
University of Miami School of Medicine
Sylvester Comprehensive Cancer Center
Division of Hematology/Oncology
1475 NW 12th Avenue, (D8-4), Suite 3300
Miami, FL 33136
Telephone: (305) 243-9128
Fax: (305) 243-4975
E-mail: Amuzumde@mednet.med.miami.edu
Disease/Condition: Achalasia
Description: This is a randomized, double-blind dose-response trial of botulinum toxin Type A (Botox) injection in 56 patients with achalasia. The efficacy of two doses will be compared (80 units versus 160 units).
Contact Person: Laura Steinam
Telephone: (409) 772-8209
Fax:
E-mail: lsteinma@utmb.edu
Disease/Condition: 5-Oxoprolinuria, Glutathione Synthetase Deficiency
Description: Treatment of patients with low tissue glutathione (GSH) levels with glutathione mono-and diethyl esters. Confirmed enzyme deficiency is only inclusion criterium; ingestion of drug via nasogastric or oral routes or via intravenous infusion with daily to weekly blood and urine samples necessary.
Contact Person: William Rhead, M.D.
University of Iowa Hospitals and Clinics
Pediatrics
Iowa City, IA 52242
Telephone: (319) 356-2674
Fax: (319) 356-3347
E-mail: william-rhead@uiowa.edu
Disease/Condition: Delayed Puberty, Gonadotropin Deficiency, Normal Volunteer Children
Description: This study is for normal child volunteers or children with delayed puberty. We are studying 9 to 13 year old girl or boy healthy volunteers and 14 to 18 year olds who have not entered puberty by 14 to 18 years old. The study requires two admissions to our Clinical Research Center and the University of Chicago. The first study will examine the hormonal responses to Lupron, a long-acting modified form of gonadotropin releasing hormone (GnRH). GnRH is the natural hormone that regulated puberty. The second study will examine sleep because the pubertal hormone secretion begins at night, and Factrel, a man-made form of natural GnRH. Chronic systemic, metabolic, and endocrine disease will be excluded. The research study is done at no cost. Normal volunteers will be reimbursed $100 for their participation.
Contact Person: Nancy Perovic RN, BSN or Dr. Robert Rosenfield
University
of Chicago Children's Hospital
Department of Pediatric Endocrinology
5841 S. Maryland Avenue (MC-5053)
Chicago, Illinois 60637
Telephone: (773) 702-6432
Fax: (773) 702-0443
E-mail: nperovic@mcis.bsd.uchicago.edu
Disease/Condition: Lambert-Eaton Myasthenic Syndrome
Description: This is a study to evaluate the safety and efficacy of 3,4-Diaminopyridine (3,4-DAP) in the Lambert-Eaton myasthenic syndrome (LEMS). 3,4-DAP is an orphan drug and has been used for the treatment of LEMS in Europe for over 20 years. Patients with LEMS who are over age 18 and do not have seizures, cardiac arrhythmia or significant liver, kidney or hematologic disease are qualified for inclusion in this study. The study involves a 10 day hospitalization on the Clinical Research Unit at Duke University Medical Center during which patients receive blinded administration of 3,4-DAP or placebo. On discharge from hospital, patients are give open label 3,4-DAP for 6 months during which time the dose is adjusted to optimal levels. All costs of the study, except for travel, are provided by a grant from the Orphan Products Program of the US FDA.
Contact Person: Donald B. Sanders, M.D.
Box 3403, DUMC
Durham, NC 27710
Telephone: (919) 684-6078
Fax: (919) 660-3853
E-mail: sande007@mc.duke.edu
Disease/Condition: Peroxisomal Disorders
Description:
Contact Person: Kenneth D.R. Setchell, M.D.
Clinical Mass Spectrometer
Center
Children's Hospital Medical Center
Cincinnati, OH 45229
Telephone: (513) 559-4548
Fax: (513) 559-7853
E-mail: setck0@chmcc.org
Disease/Condition: Human Corneal Neovascularization
Description: Corneal neovascularization (CNV) is a deleterious response to ocular injury or infection in which abnormal vessels invade the normally clear cornea, predisposing to corneal allograft rejection, and visually disabling corneal opacification. Topical dihematoporphyrin (DHP) produces photothrombosis of CNV with low power laser during photodynamic therapy (PDT). This is a prospective randomized placebo controlled study; both groups will use topical steroids for 90 days following laser or sham treatment. Study parameters included visual acuity, glare, and contrast sensitivity, color vision, slit lamp biomicroscopy with clinical drawings by a masked ophthalmologist observer, dilated funduscopy, fluorescin angiography, clinical photography, digital corneal pachymetry, digital corneal topography to measure corneal astigmatism, and quantification of CNV length utilizing digital planimetry. Patients will be closely monitored for laser, DHP, and steroid toxicity. Patients 18 and over with CNV due to any etiology, with 20/400 vision or better in the contralateral eye are eligible.
Contact Person: John Sheppard, M.D., MMSc
Medical College of Hampton Roads
403 Medical Tower
Norfolk, VA 23507
Telephone: (757) 622-2200
Fax: (757) 622-9136
E-mail:
Disease/Condition: Small Bowel Crohn's Disease
Description: Double-blind placebo controlled trial of 4ASA in small bowel Crohn's disease. This is a multicenter 3 month trial. Patients must have at least active small bowel Crohn's disease, not be on other 5ASA, prednisone 20 mg or less, no 6 MP, azathioprine, Flagyl, Cipro; women must be on birth control pills if child bearing age.
Contact Person: James Vecchio, M.D.
University of Vermont
Given, C-317
Burlington, VT 05401
Telephone: (802) 656-2554
Fax:
E-mail: vecchio@moose.uvm.edu
Disease/Condition: Pancreatic Cancer
Description: Cancer of the pancreas which is beyond the scope of surgical resection. This includes patients with metastatic disease and those with involvement of the mesenteric vessels.
Contact Person: Scott Wadler, M.D.
Montefiore Medical Center
Department of Oncology, Hofheimer One
111 East 210th Street
Bronx, NY 10467
Telephone: (718) 920-4830
Fax: (718) 798-7474
E-mail: wadler@jimmy.harvard.edu
Disease/Condition: Hereditary Angiodema
Description: This study is to treat persons with hereditary angiodema. Study is for patients at least 8 years of age who have been diagnosed with hereditary angiodema. We are exploring 2 studies at this time. One treatment for emergency attacks - throat, stomach, facial swelling, and treatment on a daily basis to help reduce attacks. One study is 6 months in length and the other is 4 months. Persons who are interested or who have questions about hereditary angiodema may contact our site.
Contact Person: Teresa Layton
UIHC
SE 302GH
200 Hawkins Drive
Iowa City, IA 52242
Telephone: (319) 356-1659
Fax: (319) 356-8222
E-mail: TLayton@ictg.uiowa.edu
Disease/Condition: Actinic Keratoses, Xeroderma Pigmentosum
Description: An eighteen month clinical study to compare the safety and efficacy of T4N5 Liposome Lotion versus placebo lotion in the protection against actinic keratoses in patients with xeroderma pigmentosum. All actinic keratoses must be removed and histologically confirmed prior to initiation of the study and every visit thereafter. Hemogram, liver and kidney function tests; amylase, total protein, albumin, cholesterol, triglycerides, skin examination, pregnancy test as applicable, questionnaires, photographs of the exposed skin quarterly for one year. Application of the lotion daily for 12 months and then follow up at 1 month and 6 months is post applications. Inclusion criteria: Confirmed diagnosis of xeroderma pigmentosum, at least one histologically confirmed actinic keratoses. Between 5-60 years of age. Exclusion criteria: Associated syndromes such as Cockayne's Syndrome or trichothiodystrophy, use of illegal drugs during the study, pregnancy or breast feeding, poor health or mental incapacity.
Contact Person: Jonathan D. Klein
Applied Genetics Inc.
205 Buffalo Avenue
Freeport, NY 11520
Telephone: (516) 868-9026
Fax: (516) 888-9143
E-mail: AGI@tribeca.jos.com
Disease/Condition: Cutaneous T-Cell Lymphoma (mycosis fungoides, Sezary Syndrome)
Description: Phase I trial testing T-cell receptor (TCR) DNA vaccination in patients with cutaneous T-Cell lymphoma (mycosis fungoides, Sezary Syndrome). Screened patients that express TCR-Vb3.1, 5.1, 6.1, or 13.1 on their malignant clone qualify for entry.
Contact Person: Stuart R. Lessin, M.D.
University of Pennsylvania
Department of Dermatology
415 Curie Blvd.
212 Clinical Research Building
Philadelphia, PA 19104
Telephone: (215) 573-3130
Fax: (215) 898-0201
E-mail: lessin@mail.med.upenn.edu
Disease/Condition: Severe childhood osteopetrosis
Description: Phase III placebo-controlled trial in 30 patients with severe childhood osteopetrosis. All patients receive calcitriol.
Contact Person: Mary Ann Hanes
Charleston, SC
Telephone: (803) 792-6807
Fax: (803) 793-2033
E-mail: KEYL@MUSC.EDU
Disease/Condition: Spinal Cord Injury, Tetraplegia, Paraplegia, Quadriplegia
Description: The objective of this project is to evaluate the safety and efficacy of a functional neuromuscular stimulation (FNS) system that allows individuals with low cervical or high thoracic spinal cord injuries to stand and transfer with minimal assistance. The overall goal is to increase the independence and personal mobility of people with incomplete tetraplegia or paraplegia via a surgically implanted FNS system. FNS can successfully restore standing and facilitate transfers in selected volunteers with injuries between the levels of C6 and T4 through the activation of the paralyzed knee, hip and trunk muscles. The implant is installed in a single surgical procedure and is powered by a wearable controller that coordinates the delivery of stimulation through a transcutaneous radio-frequency (RF) link. Post-surgical exercise and rehabilitation lead to independent use of the system in the home and community.
Contact Person: Ronald Triolo, M.D.
Cleveland FES Center
University West Building, Suite 207
11000 Cedar Avenue
Cleveland, OH 44106
Telephone: (216) 791-3800
Fax: (216) 231-3258
E-mail: rxt24@po.cwru.edu
Disease/Condition: Neonatal Adrenoleukodystrophy, Infantile Refsum Peroxisome Biogenesis Disorder
Description: This study aims to evaluate therapy of disorders of peroxisome biogenesis (neonatal adrenoleukodystrophy and infantile refsum disease) with the fatty acid, docasohexaenoic acid (DHA). Children with these disorders between the ages of 3-36 months with useful vision are sought to evaluate DHA in improving visual and cognitive function.
Contact Person: Gerald V. Raymond, M.D.
Kennedy Krieger Institute
Neurogenetics
707 N. Broadway
Baltimore, MD 21205
Telephone: (410) 550-9405
Fax: (410) 550-9839
E-mail: Raymond@kennedykrieger.org
Disease/Condition: Chronic Graft vs Host Disease
Description: The goal of this study is to develop an effective regimen to treat high risk and refractory chronic graft vs host disease. We are studying the combination of cyclosporine, thalidomide, and PUVA (ultraviolet light). Thalidomide and cyclosporine are effective in treating chronic GVHD in the laboratory and we have found PUVA to be very effective in treating skin disease. Patient eligibility-chronic GVHD with progression of disease on therapy at any time point or with stable disease after 6 months of treatment or high risk chronic GVHD at presentation (having two or more of the following: bilirubin greater than or equal to 1.2., lichenoid histology, and/or progressive onset). Ineligible patients: life threatening medical problems with a life expectancy of less than one month; unable to take oral medications; unwilling or unable to return to the transplant center for the necessary follow-up; peripheral neuropathy; pregnant or intend to become pregnant; renal failure, as shown by a creatinine of greater than or equal to 4.
Contact Person: Georgia B. Vogelsang, M.D.
Johns Hopkins University
600 N. Wolfe Street
Oncology Room 3-121
Baltimore, MD 21287
Telephone: (410) 955-8783
Fax: (410) 955-1969
E-mail: vogelsang@wfmail.onc.jhu.edu
Disease/Condition: Amyloidosis
Description: AL amyloidosis is a disease in which an excess of monoclonal immunoglobulin light chains produced by bone marrow plasma cells, form pathologic amyloid fibrillar deposits in the kidneys, heart, liver, autonomic and peripheral nerves. For patients with AL amyloidosis, death usually occurs in 1 to 2 years from diagnosis despite standard treatment with oral melphalan and prednisone. Our current clinical trial involves the use of high dose chemotherapy (melphalan) with blood stem support at our center. To be eligible, patients must be greater than 18 years old and within 12 months of their diagnosis, must have had ho previous therapy for amyloidosis with steroids or melphalan, and must have a left ventricular ejection fraction greater than 40%. Preliminary studies have shown that patients treated intensively in this fashion can achieve remissions of their plasma cell disease and of their amyloid-related organ involvement (BLOOD 1996; 88:2801-6.
Contact Person: Ms. Serena Baqai
Boston University School of Medicine
Amyloid Treatment and Research Program
80 East Concord Street
Boston, MA 02118
Telephone: (617) 638-4317
Fax: (617) 638-5226
E-mail: buamyloid@med-med1.bu.edu
Disease/Condition: Malignant Pleural Mesothelium
Description: Phase II Study of Intrapleural Liposome-Entrapped NDDP L-NDDP is a lipophilic, non-cross resistant platinum compound, which has shown no systemic toxicity after intrapleural administration. Patients with malignant pleural mesotheliuma and free flowing pleural fluid are eligible, regardless of prior chemotherapy. L-NDDP is administered through an intrapleural catheter every 3 weeks. Tumor response will be assessed by radiographic exams and thoracoscopic exam. All pre-clinical and prior clinical data suggest that L-NDDP may be particularly effective in patients with low burden disease.
Contact Person: Dong M. Shin, M.D.
U.T. M.D. Anderson Cancer Center
1515 Holcombe Blvd., Box 80
Houston, TX 77030
Telephone: (713) 792-6363
Fax: (713) 796-8655
E-mail: dshin@notes.mdacc.tmc.edu
Disease/Condition: Facioscapulohumeral Muscular Dystrophy
Description: This is an FDA-sponsored double blind trial of proventil in fascioscapulohumeral muscular dystrophy, a 52 week study of albuterol (a B2 agonist) to see if Proventil increases muscle strength. Ambulatory patients, ages 18-60 without other complications (e.g. other diseases or medications) may be eligible.
Contact Person: Lynn Cos, R.N.
University of Rochester Medical Center
Department of Neurology
601 Elmwood Avenue, Box 673
Rochester, NY 14642
Telephone: (716) 275-7680
Fax: (716) 256-1423
E-mail: LCOS@mail.Neurolofy.rochester.edu
Disease/Condition: Gonadotropin Deficiency - Adults
Description: This study is to evaluated a new, simplified form of treatment for 18 to 39 year old males and females with gonadotropin deficiency (GnD). Patients will be treated on an outpatient basis with intermittent injections of Lupron, a long-acting modified form of gonadotropin releasing hormone (GnRH). Intermittent inpatient and outpatient evaluations are required. The research study is done at no cost and free parking stickers are provided.
Contact Person: Nancy Perovic RN, BSN or Dr. Robert Rosenfield
University
of Chicago Children's Hospital
Department of Pediatric Endocrinology
5841 S. Maryland Avenue (MC-5053)
Chicago, Illinois 60637,
Telephone: (773) 702-6432
Fax: (773) 702-0443
E-mail: nperovic@mcis.bsd.uchicago.edu
Disease/Condition: Gaucher Disease
Description: A Phase II clinical trial to study the effect of Fosamax (alendronate disodium) on bone manifestations seen in people with Gaucher disease type 1 (non-neuronopathic) who are receiving enzyme replacement therapy (ERT). Individuals must be between the ages of 18 and 50 years of age. Study participants will take alendronate disodium or a placebo for two years and will return for evaluation every six months. Evaluation will consist of a physical exam, DEXA, X-rays of the long bones, and blood and urine studies to examine bone turnover. Transportation to Cincinnati and lodging for the six-month evaluations will be paid for each participant. The principal investigator for this study is Richard J Wenstrup, M.D.
Contact Person: Laurie Williams, M.S., Study Coordinator
Children's Hospital
Medical Center
3333 Burnet Ave., PAV-352
Cincinnati, OH 45229
,
Telephone: (800) 647-4805
Fax: (513) 636-7297
E-mail: will12@chmcc.org
Disease/Condition: Neoplastic Meningitis
Description: The purposes of this study are to determine the safety and side effects of a new water soluble, microcrystalline preparation of busulfan, Spartaject, administered into the CSF in the treatment of neoplastic meningitis. The role of Spartaject is unknown, and learning the proper dose and potential benefit of this drug are the goal of this study. The drug will be injected into the cerebrospinal fluid (CSF) by lumbar puncture (insertion of a needle into the lower back) or through an intraventricular reservoir ("Ommaya Reservoir") that has been or will be placed by a neurosurgeon. Spartaject will be administered twice weekly for a total of two weeks at a dose determined in part by the effects seen in previous patients enrolled on this study. If the tumor responds, further therapy with Spartaject can be provided. It is hoped that this proposed treatment will lead to remission (where no disease is detectable) and prolong survival, but this cannot be guaranteed; therefore, this treatment should not be considered a cure.
Contact Person: Henry S. Friedman, MD or Tracy Kerby, MA, CCRA
Neuro-Oncology
Duke University Medical Center, Box 3624
Room 047 Baker House, Hospital South
Trent Drive
Durham, NC 27710
Telephone: (919) 684-5301
Fax: (919) 684-6674
E-mail: fried003@mc.duke.edu or kerby001@mc.duke.edu
Disease/Condition: Active Class IV Nephritis in Lupus
Description: This study is being conducted to compare the efficacy and safety of mycophenolate mofetil (CellCept) to intravenous cyclophosphamide for the treatment of active flares of severe lupus kidney disease. The study will be carried out at approximately 20 centers with expertise in systemic lupus erythematosus throughout the United States. In this study, which is of six to nine months in duration, patients will be randomly assigned to receive one of the two regimens. After three months, an analysis of each participant's progress will be made, and those who have not had sufficient improvement will be crossed over to receive the other study regimen. This is designed to provide the best treatment for each patient.
Contact Person: Karen Orloff
SUNY Health Science Center at Brooklyn
450 Clarkson Avenue
Box 42
Brooklyn, New York 11203,
Telephone: (718) 270-1662
Fax: (718) 270-1562
E-mail:
Disease/Condition: Common Variable Immunodeficiency
Description: This study is designed to determine if a natural compound, that boosts T-cell immunity, can help stimulate better immunity in people who have common variable immunodeficiency. The investigators have been using interleukin 2 (IL-2) in this immune deficiency, for about seven years, in various studies. What the investigators have seen so far leads to this new study, since they have seen good results in the former studies. Everyone in this study is given the interleukin 2; no one is given a placebo substance. This study will last two years. The injection is a very small amount of interleukin 2, which is a substance healthy T-cells make, but which is deficient in common variable immunodeficiency. There are two groups of patients enrolled, both are given low doses of interleukin 2; the highest dose is 150,000 units twice a week for one group, and 250,000 units twice a week for the other group. Both doses are capable of enhancing immunity, but the investigators do not yet know how much interleukin 2 is optimum. This is one of the goals of this study. Both doses are still quite low, and the investigators do not expect the participants to have significant side effects. These injections will be self-administered in the particpants' home. The investigators will send the medication to the participants. The interleukin 2 will be supplied free of charge. Everyone in this study must be willing to collect diary information (about antibiotics, doctor visits, etc.). A very important part of this study is to assess how the participants' immune system is performing; for this reason, a visit to Mount Sinai for blood tests will be required at intervals of every six months. Since the study will last two years, the participants will need to count on making five trips to Mount Sinai for blood tests.
Contact Person: Charlotte Cunningham-Rundles, M.D., PhD.
1425 Madison Avenue
Mount Sinai Medical Center
New York, New York 10029,
Telephone: (212) 659-9268
Fax: (212) 987-5593
E-mail: ccunning@smtplink.mssm.edu