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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02878603




Registration number
NCT02878603
Ethics application status
Date submitted
4/08/2016
Date registered
25/08/2016
Date last updated
28/03/2022

Titles & IDs
Public title
Follow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES)
Scientific title
Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)
Secondary ID [1] 0 0
2016-001503-23
Secondary ID [2] 0 0
LTS16371
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acquired Thrombotic Thrombocytopenic Purpura 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Blood 0 0 0 0
Other blood disorders
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Caplacizumab
Other interventions - Standard of Care

Experimental: caplacizumab - Participants who completed study ALX0681-C301 (NCT02553317) with standard of care (plasma exchange [PE], corticosteroid and other immunosuppressive agents) or caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 milligrams (mg) intravenous dose followed by a daily 10 mg subcutaneous injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.


Other interventions: Caplacizumab


Other interventions: Standard of Care
• PE with plasma (e.g., fresh frozen plasma, solvent detergent/viral-inactivated plasma, cryosupernatant), • Corticosteroid treatment and • Use of other immunosuppressive agents (e.g., rituximab).
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events
Timepoint [1] 0 0
From Baseline up to 36 months
Primary outcome [2] 0 0
Number of Acquired Thrombotic Thrombocytopenic Purpura-related Events
Timepoint [2] 0 0
From Baseline up to 36 months
Primary outcome [3] 0 0
Time to First Acquired Thrombotic Thrombocytopenic Purpura-related Events
Timepoint [3] 0 0
From Baseline up to 36 months
Primary outcome [4] 0 0
Number of Participants With aTTP Related Deaths Reported During the Study
Timepoint [4] 0 0
From Baseline up to 36 months
Primary outcome [5] 0 0
Percentage of Participants With Recurrence of Disease (aTTP)
Timepoint [5] 0 0
From Baseline up to 36 months
Primary outcome [6] 0 0
Number of Disease (aTTP) Recurrence Reported During the Study
Timepoint [6] 0 0
From Baseline up to 36 months
Primary outcome [7] 0 0
Time to Recurrence of Disease (aTTP)
Timepoint [7] 0 0
From Baseline up to 36 months
Primary outcome [8] 0 0
Percentage of Participants With Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura (TTP)
Timepoint [8] 0 0
From Baseline up to 36 months
Primary outcome [9] 0 0
Number of Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura
Timepoint [9] 0 0
From Baseline up to 36 months
Primary outcome [10] 0 0
Time to First Major Thromboembolic Event
Timepoint [10] 0 0
From Baseline up to 36 months
Primary outcome [11] 0 0
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
Timepoint [11] 0 0
Baseline, 36 Months follow-up visit
Primary outcome [12] 0 0
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
Timepoint [12] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [13] 0 0
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
Timepoint [13] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [14] 0 0
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
Timepoint [14] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [15] 0 0
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
Timepoint [15] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [16] 0 0
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
Timepoint [16] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [17] 0 0
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
Timepoint [17] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [18] 0 0
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
Timepoint [18] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [19] 0 0
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
Timepoint [19] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [20] 0 0
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
Timepoint [20] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [21] 0 0
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
Timepoint [21] 0 0
Baseline, Month 12, 24, and 36 Follow-up visits
Primary outcome [22] 0 0
Percentage of Participants With Drug-induced Treatment-emergent (TE) Anti-drug Antibodies (ADA) Positive Response
Timepoint [22] 0 0
From Baseline up to 36 months
Primary outcome [23] 0 0
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [23] 0 0
From Baseline up to 36 months

Eligibility
Key inclusion criteria
1. Completed the Final (28 day) follow-up visit in Study ALX0681-C301.

2. >= 18 years of age at the time of signing the informed consent form.

3. Provided informed consent prior to initiation of any study specific
activity/procedure.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Not being able/willing to comply with the study protocol procedures.

2. Currently enrolled in a clinical study with another investigational drug or device.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/10/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/10/2020
Sample size
Target
Accrual to date
Final
104
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Missouri
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Oklahoma
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
South Carolina
Country [8] 0 0
Austria
State/province [8] 0 0
Vienna
Country [9] 0 0
Belgium
State/province [9] 0 0
Antwerp
Country [10] 0 0
Belgium
State/province [10] 0 0
Bruxelles
Country [11] 0 0
Belgium
State/province [11] 0 0
La Louviere
Country [12] 0 0
Belgium
State/province [12] 0 0
Leuven
Country [13] 0 0
Canada
State/province [13] 0 0
Halifax
Country [14] 0 0
Canada
State/province [14] 0 0
Quebec
Country [15] 0 0
Canada
State/province [15] 0 0
Toronto
Country [16] 0 0
Czechia
State/province [16] 0 0
Brno
Country [17] 0 0
Czechia
State/province [17] 0 0
Olomouc
Country [18] 0 0
France
State/province [18] 0 0
Caen
Country [19] 0 0
France
State/province [19] 0 0
Lille
Country [20] 0 0
France
State/province [20] 0 0
Marseille
Country [21] 0 0
France
State/province [21] 0 0
Paris
Country [22] 0 0
Hungary
State/province [22] 0 0
Budapest
Country [23] 0 0
Hungary
State/province [23] 0 0
Debrecen
Country [24] 0 0
Israel
State/province [24] 0 0
Haifa
Country [25] 0 0
Israel
State/province [25] 0 0
Jerusalem Region
Country [26] 0 0
Israel
State/province [26] 0 0
Nahariya
Country [27] 0 0
Italy
State/province [27] 0 0
Catania
Country [28] 0 0
Italy
State/province [28] 0 0
Milan
Country [29] 0 0
Italy
State/province [29] 0 0
Pesaro
Country [30] 0 0
Italy
State/province [30] 0 0
Rome
Country [31] 0 0
Italy
State/province [31] 0 0
Vicenza
Country [32] 0 0
Spain
State/province [32] 0 0
Barcelona
Country [33] 0 0
Spain
State/province [33] 0 0
Sevilla
Country [34] 0 0
Spain
State/province [34] 0 0
Valencia
Country [35] 0 0
Switzerland
State/province [35] 0 0
Bern
Country [36] 0 0
Turkey
State/province [36] 0 0
Ankara
Country [37] 0 0
Turkey
State/province [37] 0 0
Istanbul
Country [38] 0 0
Turkey
State/province [38] 0 0
Kayseri
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Bristol
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Liverpool
Country [41] 0 0
United Kingdom
State/province [41] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Sanofi
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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).
Trial website
https://clinicaltrials.gov/ct2/show/NCT02878603
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Medical Director Ablynx, MD
Address 0 0
Ablynx NV
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries