Phase 3
N=104
Follow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES)
Acquired Thrombotic Thrombocytopenic Purpura
Bottom Line
View on ClinicalTrials.gov: NCT02878603 ↗Enrolled (actual)
104
Serious AEs
42.3%
Results posted
Nov 2021
Primary outcome: Primary: Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events — 37.9; 8.2 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Caplacizumab (Biological); Standard of Care (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sanofi
- Primary completion
- Oct 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events |
37.9; 8.2 | — |
| PRIMARY Number of Acquired Thrombotic Thrombocytopenic Purpura-related Events |
11; 4 | — |
| PRIMARY Time to First Acquired Thrombotic Thrombocytopenic Purpura-related Events |
NA; NA | — |
| PRIMARY Number of Participants With aTTP Related Deaths Reported During the Study |
1; 0 | — |
| PRIMARY Percentage of Participants With Recurrence of Disease (aTTP) |
27.6; 8.2 | — |
| PRIMARY Number of Disease (aTTP) Recurrence Reported During the Study |
8; 4 | — |
| PRIMARY Time to Recurrence of Disease (aTTP) |
NA; NA | — |
| PRIMARY Percentage of Participants With Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura (TTP) |
37.9; 8.2 | — |
| PRIMARY Number of Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura |
11; 4 | — |
| PRIMARY Time to First Major Thromboembolic Event |
NA; NA | — |
| PRIMARY Cognitive Function: Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Absolute Scores at Baseline, 36 Months Follow-up Visit, and Change From Baseline in RBANS Total Score at 36 Months Follow-up Visit |
89.7; 92.7; 98.0; 96.5; 2.1; 4.2 | — |
| PRIMARY Health-Related Quality of Life (HRQoL): Change From Baseline in Headache Impact Test (HIT-6) Total Scores at Month 12, 24, and 36 Follow-up Visits |
45.0; 48.2; 0.9; 0.1; 1.2; -0.6 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire (SF-36) Health Survey - Physical Functioning Domain Scores at Month 12, 24, and 36 Follow-up Visits |
68.3; 74.1; 1.3; 1.5; -0.8; 5.7 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Role Functioning/Physical Domain Scores at Month 12, 24, and 36 Follow-up Visits |
60.1; 65.9; -5.3; 7.7; 4.1; 7.1 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Role Functioning/Emotional Domain Scores at Month 12, 24, and 36 Follow-up Visits |
75.3; 75.0; -13.2; 1.6; -1.3; 0.9 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Energy/Fatigue Domain Scores at Month 12, 24, and 36 Follow-up Visits |
50.9; 52.4; -0.3; 2.0; 5.9; 0.6 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Emotional Well-being Domain Scores at Month 12, 24, and 36 Follow-up Visits |
72.1; 70.1; -9.3; -2.5; -3.3; -2.0 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Social Functioning Domain Scores at Month 12, 24, and 36 Follow-up Visits |
74.6; 74.0; -10.6; 1.6; 0.0; -0.8 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Pain Domain Scores at Month 12, 24, and 36 Follow-up Visits |
68.4; 68.0; -1.8; 0.3; -9.4; 5.0 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - General Health Domain Scores at Month 12, 24, and 36 Follow-up Visits |
66.4; 53.1; -4.0; 4.9; -1.5; 4.8 | — |
| PRIMARY Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Change in Health Status Scores at Month 12, 24, and 36 Follow-up Visits |
45.7; 53.6; 30.0; 20.1; 21.3; 10.6 | — |
| PRIMARY Percentage of Participants With Drug-induced Treatment-emergent (TE) Anti-drug Antibodies (ADA) Positive Response |
0; 10.7 | — |
| PRIMARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
26; 68; 16; 28; 1; 0 | — |
Summary
The objectives of this study were to evaluate long-term safety and efficacy of caplacizumab, to evaluate safety and efficacy of repeated use of caplacizumab and to characterize long-term impact of acquired thrombotic thrombocytopenic purpura (aTTP).
Eligibility Criteria
Inclusion Criteria
- Completed the Final (28 day) follow-up visit in Study ALX0681-C301.
- >= 18 years of age at the time of signing the informed consent form.
- Provided informed consent prior to initiation of any study specific activity/procedure.
Exclusion Criteria
- Not being able/willing to comply with the study protocol procedures.
- Currently enrolled in a clinical study with another investigational drug or device.
Data sourced from ClinicalTrials.gov (NCT02878603). 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.