Clinical trial NCT02878603: Follow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES)
Official title: Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Acquired Thrombotic Thrombocytopenic Purpura
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2016-08-25
Enrollment: 104
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: 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.

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.

Primary outcomes:
measure: Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events
timeFrame: From Baseline up to 36 months
measure: Number of Acquired Thrombotic Thrombocytopenic Purpura-related Events
timeFrame: From Baseline up to 36 months
measure: Time to First Acquired Thrombotic Thrombocytopenic Purpura-related Events
timeFrame: From Baseline up to 36 months
measure: Number of Participants With aTTP Related Deaths Reported During the Study
timeFrame: From Baseline up to 36 months
measure: Percentage of Participants With Recurrence of Disease (aTTP)
timeFrame: From Baseline up to 36 months
measure: Number of Disease (aTTP) Recurrence Reported During the Study
timeFrame: From Baseline up to 36 months
measure: Time to Recurrence of Disease (aTTP)
timeFrame: From Baseline up to 36 months
measure: Percentage of Participants With Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura (TTP)
timeFrame: From Baseline up to 36 months
measure: Number of Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura
timeFrame: From Baseline up to 36 months
measure: Time to First Major Thromboembolic Event
timeFrame: From Baseline up to 36 months
measure: 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
timeFrame: Baseline, 36 Months follow-up visit
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: 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
timeFrame: Baseline, Month 12, 24, and 36 Follow-up visits
measure: Percentage of Participants With Drug-induced Treatment-emergent (TE) Anti-drug Antibodies (ADA) Positive Response
timeFrame: From Baseline up to 36 months
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From Baseline up to 36 months
Secondary outcomes:
Other outcomes:
Locations:
city: Saint Louis
country: United States
facility: Investigator site
state: Missouri
city: Valhalla
country: United States
facility: Investigator site
state: New York
city: Durham
country: United States
facility: Investigator Site
state: North Carolina
city: Columbus
country: United States
facility: Investigator Site
state: Ohio
city: Oklahoma City
country: United States
facility: Investigator Site
state: Oklahoma
city: Pittsburgh
country: United States
facility: Investigator site
state: Pennsylvania
city: Charleston
country: United States
facility: Investigator Site
state: South Carolina
city: Greenville
country: United States
facility: Investigator site
state: South Carolina
city: Vienna
country: Austria
facility: Investigator site
city: Antwerp
country: Belgium
facility: Investigator site
city: Bruxelles
country: Belgium
facility: Investigator site
city: La Louviere
country: Belgium
facility: Investigator site
city: Leuven
country: Belgium
facility: Investigator site
city: Halifax
country: Canada
facility: Investigator Site
city: Quebec
country: Canada
facility: Investigator Site
city: Toronto
country: Canada
facility: Investigator Site
city: Brno
country: Czechia
facility: Investigator Site
city: Olomouc
country: Czechia
facility: Investigator Site
city: Caen
country: France
facility: Investigator site
city: Lille
country: France
facility: Investigator site
city: Marseille
country: France
facility: Investigator site
city: Paris
country: France
facility: Investigator site 1
city: Paris
country: France
facility: Investigator site 2
city: Budapest
country: Hungary
facility: Investigator Site
city: Debrecen
country: Hungary
facility: Investigator Site
city: Haifa
country: Israel
facility: Investigator Site
city: Jerusalem Region
country: Israel
facility: Investigator Site
city: Nahariya
country: Israel
facility: Investigator Site
city: Catania
country: Italy
facility: Investigator Site
city: Milan
country: Italy
facility: Investigator Site
city: Pesaro
country: Italy
facility: Investigator site
city: Rome
country: Italy
facility: Investigator Site
city: Vicenza
country: Italy
facility: Investigator Site
city: Barcelona
country: Spain
facility: Investigator Site 1
city: Barcelona
country: Spain
facility: Investigator Site 2
city: Sevilla
country: Spain
facility: Investigator site
city: Valencia
country: Spain
facility: Investigator Site 2
city: Valencia
country: Spain
facility: Investyigator Site 1
city: Bern
country: Switzerland
facility: Investigator site
city: Ankara
country: Turkey
facility: Investigator site
city: Istanbul
country: Turkey
facility: Investigator site
city: Kayseri
country: Turkey
facility: Investigator site
city: Bristol
country: United Kingdom
facility: Investigator Site
city: Liverpool
country: United Kingdom
facility: Investigator site
city: London
country: United Kingdom
facility: Investigator Site
Study officials:
affiliation: Ablynx NV
name: Medical Director Ablynx, MD
role: STUDY_DIRECTOR
References: