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U.S. Department of Health and Human Services

Vaccines, Blood & Biologics

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Bioresearch Monitoring Final Review Memo - Atryn

Memorandum

Date: January 12, 2009
FROM: Joseph P. Manik, Bioresearch Monitoring Branch, HFM-664
Division of Inspections and Surveillance
Office of Compliance and Biologics Quality
THROUGH: Patricia A. Holobaugh
Bioresearch Monitoring Branch Chief, HFM-664
TO: Roman T. Drews, Ph.D., Committee Chair, HFM-392
Pratibha Rana, Regulatory Project Manager, HFM 380
SUBJECT: Bioresearch Monitoring Final Review memo
STN: BLA 125284/0
Sponsor: GTC Biotherapeutics, Inc.
Product: ATryn ® (antithrombin alfa rhAT)

FINAL SUMMARY STATEMENT

Results of four bioresearch monitoring inspections of four clinical sites did not reveal problems that impact the data submitted in the application.

BACKGROUND

 Inspections of four clinical investigators were performed in support of this Biologics License Application (BLA). Information from the BLA was compared to source documents during the inspections. The inspections focused on specific questions concerning two pivotal studies.

PROTOCOL : GTC AT HD 012-04

STUDY TITLE: A Multicenter, Multinational Study to Assess the Safety and Efficacy of Antithrombin alfa in Hereditary Antithrombin (AT) Deficient Patients in High-Risk Situations for Thrombosis

Inspection of clinical sites and outcome for GTC AT HD 012-04

Study Site Number

Study Site

 

Location

Number of Subjects

Form FDA 483 Issued

Final Classification

 

01-06

Yale-New Haven Hospital

New Haven , Connecticut

01

No

NAI

01-04

Weill Medical College of Cornell University

New York , New York

01

No

NAI

NAI – No Action Indicated, VAI – Voluntary Action Indicated, EIR – Establishment Inspection Report

PROTOCOL: GTC AT III 01002

STUDY TITLE: A Multicenter, Multinational Study to Assess the Incidence of Deep Vein Thrombosis (DVT) Following Prophylactic Intravenous Administration of Recombinant Human Antithrombin (rh AT) to Hereditary Antithrombin Deficient Patients in High-Risk Situations

Inspection of clinical sites and outcome for GTC AT III 01002

Study Site Number

Study Site

 

Location

Number of Subjects

Form FDA 483 Issued

Final Classification

 

81

Northwest Georgia Oncology Centers

Marietta , Georgia

02

No

EIR Pending

83

Children’s Hospital

Medical University of South Carolina

Charleston , South Carolina

01

No

EIR Pending

NAI – No Action Indicated, VAI – Voluntary Action Indicated, EIR – Establishment Inspection Report

SPONSOR ISSUES

No sponsor or monitoring issues were noted.

INSPECTIONAL FINDINGS

Clinical Investigator (CI) issues:

Study Site # 01-06: The inspection revealed no deviations from applicable federal regulations governing investigational drugs but the FDA investigator discussed two items at the conclusion of the inspection, including the importance of recording observations in ink and the importance of properly documenting any changes that are made to recorded observations so that accurate study records are maintained.

Study Site # 01-04: The inspection revealed no deviations from applicable federal regulations governing investigational drugs.

As of this date inspections have been completed for Study Site # 81, Northwest Georgia Oncology Centers, and Study Site # 83, Children’s Hospital Medical University of South Carolina, but t he Establishment Inspection Report (EIR) is pending. T he Bioresearch Monitoring Branch has contacted the investigators who conducted each of these inspections and has obtained the following information:

Study Site # 81 : The inspection revealed n o deviations from applicable federal regulations governing investigational drugs. No FDA 483 was issued.

Study Site # 83: The inspection covered informed consent, a comparison of source documents and CRFs, a comparison of the safety and efficacy assessments provided with the assignment to the data contained in the case report forms/source documents, in addition to the C.P. 7348.811 coverage requirements of protocol adherence, IRB approval and correspondence, sponsor correspondence, and test article accountability. The inspection found only one subject screened for the study. No objectionable conditions were found during the inspection. No FDA 483 was issued.

BIMO ADMINISTRATIVE FOLLOW-UP

Close out letters were issued to Study Site # 01-04 and Study Site # 01-06. Correspondence will be issued to Study Site # 81 and Study Site # 83 after complete review of the establishment inspection reports and final classification.

Should you have any questions or comments about the contents of this memorandum or any aspect of Bioresearch Monitoring, please contact me at (301) 827-6335.

___________________________
Joseph P. Manik
Consumer Safety Officer