| ||||
| |||
A new DRUG TRIALS SNAPSHOT is now available.:
Drug Trials Snapshots: CABLIVI
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.
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 the CABLIVI Package Insert for complete information.
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 the CABLIVI Package Insert for complete information.
CABLIVI (caplacizumab-yhdp)
cab-LIV-ee
Ablynx N.V.
Approval date: February 6, 2019
cab-LIV-ee
Ablynx N.V.
Approval date: February 6, 2019
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
CABLIVI is a drug used to treat adults with an episode of acquired thrombotic thrombocytopenic purpura (aTTP).
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life-threatening blood clotting disorder. During an episode of aTTP, clots form throughout the body and lead to low platelets count, destroy red blood cells, and can damage organs (especially the brain and heart).
How is this drug used?
CABLIVI is used in combination with standard treatment of plasma exchange and medications that suppresses the immune system.
- The first dose of CABLIVI is injected into a vein (intravenous) by a healthcare provider at least 15 minutes before the first plasma exchange.
- Treatment is continued with an injection under the skin (subcutaneous) after daily plasma exchange and for 30 days after daily plasma exchange is stopped.
What are the benefits of this drug?
In patients with aTTP who required plasma exchange to treat their condition, platelet count returned to the normal range more quickly in patients treated with CABLIVI than in patients treated with placebo. Patients treated with CABLIVI also had fewer deaths, repeat episodes of aTTP and major blood clotting episodes when compared to patients that received placebo.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: CABLIVI works similarly in both men and women.
- Race: The majority of patients were White. The number of patients in other races were limited; therefore, differences in how CABLIVI worked among races could not be determined.
- Age: CABLIVI works similarly in patients younger and older than 50 years of age. The majority of patients were less than 65 years of age; therefore, differences in how CABLIVI worked among patients older than 65 years of age could not be determined.
What are the possible side effects?
CABLIVI may cause serious side effects such as increased bleeding.
The most common side effects are nosebleeds, headache, and bleeding gums.
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar among men and women.
- Race: The majority of patients were White. The number of patients in other races was limited; therefore, differences in side effects among races could not be determined.
- Age: The majority of patients were less than 65 years of age; therefore, differences in side effects among age groups could not be determined.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved CABLIVI based on evidence from two clinical trials (Trial 1/NCT02553317 and Trial 2/NCT01151423) of 216 adults with aTTP. The trials were conducted in Asia, Canada, Europe, Great Britain and the United States.
Demographics of the patients who provided data for evaluation of benefits (efficacy population) are presented in Table 6, under the MORE INFO section.
The population that provided data for side effects of CABLIVI (safety population) is presented below.
Figure 1 summarizes how many men and women were in the clinical trials used to evaluate safety.
Figure 1. Baseline Demographics by Sex (safety population)
FDA Review
Figure 2 summarizes the percentage of patients by race in the clinical trials used to evaluate safety.
Figure 2. Baseline Demographics by Race
Table 1. Demographics of Safety Trials by Race
| Race | Number of Patients | Percentage of Patients |
|---|---|---|
| White | 160 | 74% |
| Black or African American | 36 | 17% |
| Asian | 4 | 2% |
| Native Hawaiian or Other Pacific Islander | 1 | 1% |
| Other | 15 | 6% |
Clinical Trial Data
Figure 3. Baseline Demographics by Age
Clinical Trial Data
How were the trials designed?
The benefit and side effects of CABLIVI were evaluated in two clinical trials.
Trial 1 enrolled adults 18 to 79 years old who had an episode of aTTP. Patients in both groups received standard of care treatment with plasma exchange and immunosuppressive therapy. Patients were randomized to receive CABLIVI or placebo daily in addition to plasma exchange and for 30 days after their last plasma exchange.
The benefit of CABLIVI was assessed by measuring the time needed for a patient’s platelet count to return to normal and comparing it with patients treated with placebo. The trial also evaluated the number of patients who died due to aTTP, had a second episode of aTTP, or had at least one major blood clotting event.
Trial 2 had a similar design as Trial 1, but healthcare providers knew which treatment was given before the trial was completed. The data were used primarily to assess side effects.
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.
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.
.png)
No hay comentarios:
Publicar un comentario