Phase 3
N=145
Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura
Acquired Thrombotic Thrombocytopenic Purpura
Bottom Line
View on ClinicalTrials.gov: NCT02553317 ↗Enrolled (actual)
145
Serious AEs
43.0%
Results posted
May 2019
Primary outcome: Primary: Time to Platelet Count Response — 2.69; 2.88 days — p== 0.0099
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Caplacizumab (Biological); Placebo (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ablynx, a Sanofi company
- Primary completion
- Aug 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Platelet Count Response |
2.69; 2.88 | = 0.0099 sig |
| SECONDARY Number and Percentage of Subjects With TTP-Related Death, Recurrence of TTP, or a Major Thromboembolic Event During the Study Drug Treatment Period |
9; 36 | < 0.0001 sig |
| SECONDARY Number and Percentage of Subjects With a Recurrence of TTP in the Overall Study Period |
9; 28 | = 0.0004 sig |
| SECONDARY Number and Percentage of Subjects With Refractory Disease |
0; 3 | = 0.0572 |
| SECONDARY Time to Normalization of Organ Damage Marker Levels |
2.86; 3.36 | — |
Summary
The study was a Phase III, double-blind, placebo-controlled, randomized study to evaluate the efficacy of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis
Eligibility Criteria
Inclusion Criteria
- Adult male or female ≥ 18 years of age at the time of signing the informed consent form (ICF).
- Clinical diagnosis of acquired thrombotic thrombocytopenic purpura (aTTP) (initial or recurrent), which included thrombocytopenia and microscopic evidence of red blood cell fragmentation (e.g., schistocytes).
- Required initiation of daily PE treatment and had received 1 PE treatment prior to randomization
- Others as defined in the protocol
Exclusion Criteria
- Platelet count ≥100×10E9/L.
- Serum creatinine level >200 µmol/L in case platelet count is > 30×10E9/L
- Known other causes of thrombocytopenia
- Congenital TTP (known at the time of study entry).
- Pregnancy or breast-feeding.
- Subjects who were previously enrolled in a clinical study with caplacizumab and received caplacizumab or for whom the assigned treatment arm is unknown
- Others as defined in the protocol
Data sourced from ClinicalTrials.gov (NCT02553317). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.