Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Acquired Thrombotic Thrombocytopenic Purpura
Bottom Line
View on ClinicalTrials.gov: NCT01151423 ↗Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Caplacizumab (Biological); Placebo (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ablynx, a Sanofi company
- Primary completion
- Mar 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL |
2.4; 4.3; 3; 4.9 | = 0.005 sig |
| SECONDARY Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) |
29; 18 | — |
| SECONDARY Number and Percentage of Subjects With Exacerbations of TTP |
3; 11 | — |
| SECONDARY Number and Percentage of Subjects With Relapse of TTP |
11; 3 | — |
| SECONDARY Number of Daily PE Sessions During the Initial Daily PE Period |
6.7; 8.4 | — |
| SECONDARY Total Volume of Plasma Administered During the Initial Daily PE Period |
22481.8; 28358.4 | — |
| SECONDARY Number of Days With at Least One PE Administration During the Total Course of the Study |
11.8; 12.6 | — |
| SECONDARY The Maximum Number of Consecutive Days Per Subject Where There Was no Interruption of PE During the Initial Daily PE Period |
6.6; 8.1 | — |
| SECONDARY Resolution of Non-focal Neurological Symptoms |
66.76; 49.62; 62.10; 58.36 | — |
| SECONDARY Number of Participants With Resolution of TTP-related Signs or Symptoms |
29; 29; 30; 33; 31; 27 | — |
| SECONDARY Mortality |
0; 2 | — |
| SECONDARY Number of PE Related Adverse Events |
72; 44 | — |
| SECONDARY Number and Percentage of Subjects With PE Related AEs |
20; 20 | — |
| SECONDARY Number of Treatment-emergent Adverse Events (TEAEs) by Severity |
348; 299; 154; 173; 37; 23 | — |
| SECONDARY Number and Percentage of Subjects With TEAEs by Severity |
31; 36; 27; 31; 18; 14 | — |
| SECONDARY Number of TEAEs and Their Relationship to Study Drug |
12; 6; 59; 9; 486; 524 | — |
| SECONDARY Number of Participants Who Developed Treatment-emergent Anti-Drug Antibodies (ADA) |
3; 0 | — |
| SECONDARY Plasma Concentrations of Caplacizumab |
100; 1765.9; 450.4; 562; 288; 415.8 | — |
| SECONDARY Pharmacodynamics (PD): Ristocetin Cofactor (RICO) Activity Over Time |
76.2; 82.1; 16.2; 84.4; 21.4; 94.4 | — |
| SECONDARY Pharmacodynamics: Von Willebrand Factor Antigen (vWF:Ag) Over Time |
185.1; 204.4; 120.6; 166.6; 94.6; 140.2 | — |
| SECONDARY PD: Coagulation Factor VIII Clotting Activity (FVIII:C) Over Time |
144.18; 156.8; 104; 149; 90.7; 152.9 | — |
Summary
Eligibility Criteria
Inclusion Criteria
- 18 years of age or older (adults) or aged 12 to 3 x upper limit of normal (ULN) (needed to differentiate isolated increase in indirect bilirubin due to hemolysis, this was not an exclusion parameter but disease-related)
- alanine transaminase (ALT)/ aspartate transaminase (AST) > 5 x ULN
- alkaline phosphatase (ALP) > 5 x ULN
- gamma-glutamyl transpeptidase (GGT) > 5 x ULN
- Severe chronic renal impairment, as defined by glomerular filtration rate < 30 mL/min
Note that the use of another investigational drug or device within 30 days prior to screening was not allowed. Participation in non-interventional/observational studies and registries during the study period was allowed. Participation in another clinical study was not allowed until the end of the follow-up period or within 30 days after the last study treatment in case of early subject withdrawal from the study. Subjects who had already participated in the current study and had either completed the study per protocol or had discontinued prematurely, were not allowed to be re-included.
Data sourced from ClinicalTrials.gov (NCT01151423). 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.