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

NCT ID: NCT04074187

Last Updated: 2025-09-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-21
• Study Completion Date: 2021-05-19

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

Detailed Description

Study duration per participant is approximately 2 months up to approximately 6 months in case of treatment extension and recurrence during the study drug treatment period.

Conditions

Thrombotic Thrombocytopenic Purpura

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Caplacizumab

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)

Group Type: EXPERIMENTAL

Caplacizumab (ALX-0081)

Intervention Type: DRUG

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

Plasma exchange (PE)

Intervention Type: DRUG

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

Corticosteroid treatment (Methylprednisolone or prednisolone)

Intervention Type: DRUG

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

Immunosuppressive treatment (eg, rituximab)

Intervention Type: DRUG

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

Interventions

Caplacizumab (ALX-0081)

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

Intervention Type: DRUG

Plasma exchange (PE)

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

Intervention Type: DRUG

Corticosteroid treatment (Methylprednisolone or prednisolone)

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

Intervention Type: DRUG

Immunosuppressive treatment (eg, rituximab)

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

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational Site Number 3920009

Iruma-Gun, , Japan

Investigational Site Number 3920014

Kanazawa, , Japan

Investigational Site Number 3920007

Kashihara-shi, , Japan

Investigational Site Number 3920013

Kawasaki-Shi, , Japan

Investigational Site Number 3920001

Kitakyushu-Shi, , Japan

Investigational Site Number 3920002

Kumamoto, , Japan

Investigational Site Number 3920003

Kurashiki-Shi, , Japan

Investigational Site Number 3920010

Kyoto, , Japan

Investigational Site Number 3920005

Maebashi, , Japan

Investigational Site Number 3920015

Nagoya, , Japan

Investigational Site Number 3920011

Osaka, , Japan

Investigational Site Number 3920006

Sendai, , Japan

Countries

Japan

References

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.

Reference Type: BACKGROUND

PMID: 36427162 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 39095866 (View on PubMed)

Related Links

https://www.trialsummaries.com/Study/StudyDetails?id=25244&tenant=MT_SNY_9011

EFC16297 Plain Language Results Summary

Other Identifiers

U1111-1223-4914

Identifier Type: OTHER

Identifier Source: secondary_id

EFC16297

Identifier Type: -

Identifier Source: org_study_id