A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

NCT04074187 | Completed Phase 2

• 2 other identifiers: EFC16297U1111-1223-4914
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 12

Brief Summary

Primary Objective: To evaluate the effect of caplacizumab on prevention of recurrence of aTTP (proportion of participants with a recurrence of aTTP) during the overall study period. Secondary Objectives:

• To evaluate effect of caplacizumab on
• prevention of recurrence of TTP (the number of recurrences of TTP) during overall study period.
• a composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events during study drug treatment
• restoring platelet counts as a measure of prevention of further microvascular thrombosis
• refractory disease
• biomarkers of organ damage: LDH, cardiac troponin I, serum creatinine
• plasma exchange (PE) parameters (days of PE and volume of plasma used), days in intensive care unit, days in hospital
• cognitive status of Japanese patients
• To evaluate safety profile of caplacizumab in Japanese patients
• To evaluate effect of caplacizumab on pharmacodynamic (PD) markers in Japanese patients
• To evaluate pharmacokinetic (PK) profile of caplacizumab in Japanese patients
• To evaluate immunogenicity of caplacizumab in Japanese patients

Conditions

Thrombotic Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (1)

• Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP)

  Proportion of participants with a recurrence of aTTP during the overall study period. The success criterion for this study is proportion of evaluable participants (per-protocol population) with a recurrence of aTTP during the overall study period to be 20% or less.

  Approximately 2 months up to approximately 6 months

Secondary Outcomes (16)

• Number of recurrences of TTP

  Approximately 2 months up to approximately 6 months

• Proportion of participants with composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events

  Approximately 2 months up to approximately 6 months

• Time to platelet count response

  Approximately 2 months up to approximately 6 months

• Proportion of participant who have a platelet count ≥150,000/μL

  Approximately 2 months up to approximately 6 months

• Proportion of participants with refractory TTP

  Approximately 2 months up to approximately 6 months

Study Arms (1)

Caplacizumab

EXPERIMENTAL

Eligible study participants will receive caplacizumab in addition to standard of care such as daily plasma exchange (PE) and corticosteroid treatment (mandatory), immunosuppressive treatment (if needed)

Drug: Caplacizumab (ALX-0081); Drug: Plasma exchange (PE); Drug: Corticosteroid treatment (Methylprednisolone or prednisolone); Drug: Immunosuppressive treatment (eg, rituximab)

Interventions

Caplacizumab (ALX-0081) DRUG

Pharmaceutical form:Lyophilized powder for solution for injection Route of administration: IV (first dose), SC (all subsequent doses)

Caplacizumab

Plasma exchange (PE) DRUG

Pharmaceutical form:Plasma (e.g. fresh frozen plasma) Route of administration: Plasma exchange

Caplacizumab

Corticosteroid treatment (Methylprednisolone or prednisolone) DRUG

Pharmaceutical form:Solution for injection or Tablet Route of administration: IV or Oral

Caplacizumab

Immunosuppressive treatment (eg, rituximab) DRUG

Pharmaceutical form:Solution for injection (depending on product) Route of administration: IV (depending on product)

Caplacizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Japanese participant must be 18 years or older at the time of signing the informed consent.
• Participants who have a clinical diagnosis of aTTP (initial or recurrent), which includes thrombocytopenia (defined as platelet count \<100,000/µL), microangiopathic hemolytic anemia as evidenced by red blood cell fragmentation (eg, presence of schistocytes), and increased levels of LDH
• Participants who require initiation of daily PE treatment and have received a maximum of 1 PE treatment prior to enrollment in the study
• Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
• Capable of giving signed informed consent

You may not qualify if:

• Platelet count ≥100,000/µL,
• Serum creatinine level \> 2.3mg/dL in case platelet count is \> 30,000µL
• Known other causes of thrombocytopenia
• Congenital TTP
• Clinically significant active bleeding or high risk of bleeding
• Malignant arterial hypertension
• Known chronic treatment with anticoagulant treatment that cannot be stopped
• Participants who were previously enrolled in a clinical study with caplacizumab and received caplacizumab or for whom the assigned treatment arm is unknown
• Participants currently or less than 28 days prior to enrollment in this study, enrolled in a clinical study with another investigational drug or device
• Clinical condition other than that associated with TTP, with life expectancy \< 6 months, such as end-stage malignancy
• Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
• Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (12)

Investigational Site Number 3920009

Iruma-Gun, Japan

Location

Investigational Site Number 3920014

Kanazawa, Japan

Location

Investigational Site Number 3920007

Kashihara-shi, Japan

Location

Investigational Site Number 3920013

Kawasaki-Shi, Japan

Location

Investigational Site Number 3920001

Kitakyushu-Shi, Japan

Location

Investigational Site Number 3920002

Kumamoto, Japan

Location

Investigational Site Number 3920003

Kurashiki-Shi, Japan

Location

Investigational Site Number 3920010

Kyoto, Japan

Location

Investigational Site Number 3920005

Maebashi, Japan

Location

Investigational Site Number 3920015

Nagoya, Japan

Location

Investigational Site Number 3920011

Osaka, Japan

Location

Investigational Site Number 3920006

Sendai, Japan

Location

Related Publications (2)

• Miyakawa Y, Imada K, Ichikawa S, Uchiyama H, Ueda Y, Yonezawa A, Fujitani S, Ogawa Y, Matsushita T, Asakura H, Nishio K, Suzuki K, Hashimoto Y, Murakami H, Tahara S, Tanaka T, Matsumoto M. The efficacy and safety of caplacizumab in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura: an open-label phase 2/3 study. Int J Hematol. 2023 Mar;117(3):366-377. doi: 10.1007/s12185-022-03495-6. Epub 2022 Nov 24.

  PMID: 36427162 BACKGROUND

• Imada K, Miyakawa Y, Ichikawa S, Uchiyama H, Ueda Y, Hashimoto Y, Nishimi M, Tsukamoto M, Tahara S, Matsumoto M. Frontline use of rituximab may prevent ADAMTS13 inhibitor boosting during caplacizumab treatment in patients with iTTP: post hoc analysis of a phase 2/3 study in Japan. Thromb J. 2024 Aug 2;22(1):72. doi: 10.1186/s12959-024-00642-3.

  PMID: 39095866 DERIVED

Related Links

• EFC16297 Plain Language Results Summary

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

caplacizumab; Plasma Exchange; Methylprednisolone; Prednisolone; Rituximab

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Blood Transfusion; Biological Therapy; Therapeutics; Plasmapheresis; Blood Component Removal; Sorption Detoxification; Extracorporeal Circulation; Surgical Procedures, Operative; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 14, 2019
• First Posted: August 29, 2019
• Study Start: October 21, 2019
• Primary Completion: May 19, 2021
• Study Completion: May 19, 2021
• Last Updated: September 22, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

JP (12)