Drug Trials Snapshots: LAMPIT
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to LAMPIT Prescribing Information for complete information.
LAMPIT (nifurtimox)
LAM-pit
Bayer HealthCare Pharmaceuticals Inc.
Approval date: August 6, 2020
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
LAMPIT is a drug used to treat Chagas disease in children less than 18 years of age.
Chagas disease is an infectious disease caused by a parasite (Trypanosoma [T.] cruzi). After years of infection, the parasites may cause serious and sometimes deadly heart conditions as well as serious gut and nerve damage.
How is this drug used?
LAMPIT is a tablet. It is taken by mouth three times a day with food for 60 days. Total daily dose is determined by the child’s weight.
What are the benefits of this drug?
The benefit of LAMPIT was measured by the number of patients who had a change in the antibodies to T. cruzi parasite (called serologic response). In the trial, 32% of patients who received LAMPIT for 60 days had serologic response after one year.
LAMPIT was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug while the company continues to conduct clinical trials to confirm that the drug works well.
What are the benefits of this drug?
The table below summarizes efficacy results conducted at the end of follow-up based on the serological response. Serological response to treatment was defined as ≥20% reduction in optical density measured by lysate and recombinant ELISA in subjects >8 months to <18 years or seroconversion to negative (defined as negative immunoglobulin G concentration in all patients) at 1-year post-treatment follow-up.
Table 1. Efficacy Results Using Lysate ELISA and Recombinant ELISA
| Lysate ELISA | Recombinant ELISA | |||
|---|---|---|---|---|
| 60-Day N=219 |
30-Day* N=111 |
60-Day N=219 |
30-Day* N=111 |
|
| Serological response | 70 (32%) | 21 (19%) | 76 (35%) | 24 (22%) |
| ≥20% decrease in optical density | 59 (27%) | 15 (14%) | 65 (30%) | 17 (15%) |
| Seroconversion | 11 (5%) | 6 (5%) | 11 (5%) | 7 (6%) |
| Difference (60 day – 30 day), 95% CI, p-value | 13% (3.5%, 22.6%), 0.007 | 13% (3.2%, 23.0%), 0.010 | ||
ELISA, enzyme-linked immunosorbent assay
CI=confidence interval
*The 30-day duration is not an approved dosing regimen.
LAMPIT Prescribing Information
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: LAMPIT worked similarly in males and females.
- Race: Most of the patients were White. Differences in how well LAMPIT worked among races could not be determined.
- Age: Differences in how well LAMPIT worked among age groups could not be determined.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
Table 2 summarizes subgroup efficacy results defined as ≥20% reduction in optical density or seroconversion to negative using the individual total purified antigen (lysate) and recombinant ELISA tests. Age subgroup differences were not assessed because of disease variability in different age groups.
Table 2. Efficacy Rates by Demographic Subgroups for the 60-Arm Group in Trial 1
| Demographic Subgroup | Lysate ELISA N=219 |
Recombinant ELISA N=219 |
|---|---|---|
| Sex | ||
| Male | 28/100 (28.0%) | 30/100 (30.0%) |
| Female | 42/119 (35.3%) | 46/119 (38.7%) |
| Race | ||
| White | 62/155 (40.0%) | 67/155 (43.2%) |
| American Indian or Alaska Native | 8/64 (12.5%) | 9/64 (14.1%) |
| Ethnicity | ||
| Hispanic or Latino | 70/217 (32.3%) | 76/217 (35.0%) |
| Not Hispanic or Latino | 0/2 (0) | 0/2 (0) |
| Region | ||
| Argentina | 51/119 (42.9%) | 43/119 (36.1%) |
| Bolivia | 4/41 (9.8%) | 6/41 (14.6%) |
| Colombia | 15/59 (25.4%) | 27/59 (45.8%) |
ELISA, enzyme-linked immunosorbent assay
Adapted from FDA Review
What are the possible side effects?
LAMPIT can cause serious reactions including worsening of neurologic and mental conditions, severe allergic reactions, weight loss, and possible harm to the developing fetus.
Common side effects of LAMPIT are vomiting, abdominal pain, headache, decreased appetite, nausea, fever, and rash.
What are the possible side effects?
Adverse reactions that occurred in patients in Trial 1 are summarized in the table below.
Table 3. Adverse Reactions Reported in (≥1%) Pediatric Patients with Chagas Disease in the 60-Day Arm in Trial 1 Treated with LAMPIT
| System Organ Class | Adverse Reactions | Incidence |
|---|---|---|
| Blood and lymphatic system disorders | Anemia Eosinophilia |
2.7% 2.3% |
| Gastrointestinal disorders | Vomiting Abdominal paina Nausea Diarrhea |
14.6% 13.2% 8.2% 4.6% |
| General disorders and administration site conditions | Pyrexia | 7.3% |
| Investigations | Weight decreased | 2.7% |
| Metabolism and nutrition disorders | Decreased appetite | 10.5% |
| Nervous system disorders | Headache Dizziness |
12.8% 2.7% |
| Skin and subcutaneous tissue disorders | Rashb Urticaria |
5.5% 2.3% |
a Abdominal pain includes abdominal pain and abdominal pain upper
b Rash includes rash, rash macular, rash maculo-papular, rash morbilliform, and rash papular.
LAMPIT Prescribing Information
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar in males and females.
- Race: Most of the patients were White. Differences in side effects by race could not be determined.
- Age: The occurrence of side effects was similar in tested age groups.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
The table below summarizes adverse events during the clinical trial by demographic subgroups.
Table 4. Subgroup Analysis of Treatment Emergent Adverse Events in the 60-Day Arm in Trial 1
| Demographic Subgroup | LAMPIT n/N (%) |
|---|---|
| All patients | 147/219 (67.1) |
| Sex | |
| Female | 77/119 (64.7) |
| Male | 70/100 (70.0) |
| Race | |
| White | 105/155 (67.7) |
| American Indian or Alaska Native | 42/64 (65.6) |
| Age | |
| 0 to 27 days | 3/4 (75.0) |
| 28 days to younger than 8 months | 4/8 (50.0) |
| 8 months to younger than 2 years | 15/17 (88.2) |
| 2 years to younger than 18 years | 125/190 (65.8) |
| Country | |
| Argentina | 78/119 (65.5) |
| Bolivia | 26/41 (63.4) |
| Colombia | 43/59 (72.9) |
Abbreviations: TEAE, treatment-emergent adverse event
FDA Review
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved LAMPIT based on evidence from the one clinical trial (Trial 1/NCT02625974) of 219 children with Chagas disease. Trial was conducted at 25 sites in Argentina, Bolivia and Colombia. Demographics of this trial are presented in the figures below.
Figure 1 summarizes by sex how many children were in the clinical trial.
Figure 1. Baseline Demographics by Sex
FDA Review
Figure 2 summarizes by race how many children were in the clinical trial.
Figure 2. Baseline Demographics by Race
FDA Review
Figure 3 summarizes how many patients of certain age were in the clinical trial.
Figure 3. Baseline Demographics by Age
FDA Review
Figure 4 summarizes by ethnicity how many children were in the clinical trial.
Figure 4. Baseline Demographics by Ethnicity
FDA Review
Who participated in the trials?
The table below summarizes demographics of patients in the clinical trial.
Table 5. Baseline Demographics of Patients in the 60-Day Arm in Trial 1
| Demographic Parameter | LAMPIT N=219 n (%) |
|---|---|
| Sex | |
| Male | 100 (45.7) |
| Female | 119 (54.3) |
| Race | |
| White | 155 (70.8) |
| American Indian or Alaska Native | 64 (29.2) |
| Age | |
| Median (years) | 10 |
| Min, max (years) | 0.02, 17 |
| Age Group | |
| 0 to 27 days | 4 (1.8) |
| 28 days to younger than 8 months | 8 (3.7) |
| 8 months to younger than 2 years | 17 (7.8) |
| 2 years to younger than 18 years | 190 (86.8) |
| Ethnicity | |
| Hispanic or Latino | 217 (99.1) |
| Not Hispanic or Latino | 2 (0.9) |
| Region | |
| Argentina | 119 (54.3) |
| Bolivia | 41 (18.7) |
| Colombia | 59 (26.9) |
FDA Review
How were the trials designed?
There was one trial that provided data for the assessment of benefit and side effects of LAMPIT.
In the trial, children less than 18 years of age with Chagas disease and without symptoms of heart or gastrointestinal disease were randomly treated with LAMPIT tablets three times a day for 60 days. Patients were followed for 1 year.
The benefit of LAMPIT was evaluated at the end of follow-up by measuring antibodies to the parasite in the blood.
How were the trials designed?
The safety and efficacy of LAMPIT were established in one randomized, double-blind, parallel group, historically controlled trial in children with Chagas disease.
Children less than 18 years of age with serologic evidence of T. cruzi infection and without Chagas disease-related cardiac or gastrointestinal symptoms were randomly assigned to receive LAMPIT three times a day for either 30 or 60 days and were followed for 1 year.
Serological response to treatment was defined as ≥20% reduction in optical density measured by lysate and recombinant ELISA in subjects >8 months to <18 years or seroconversion to negative (defined as negative immunoglobulin G concentration in all patients) at 1-year post-treatment follow-up.
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
PRESCRIBING INFORMATION