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

An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura

Sponsors

Lead Sponsor: Sanofi

Source Sanofi
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.

Overall Status Recruiting
Start Date November 2, 2019
Completion Date January 2021
Primary Completion Date January 2021
Phase Phase 2/Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP) Approximately 2 months up to approximately 6 months
Secondary Outcome
Measure Time Frame
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
Time to normalization of 3 organ damage marker levels Approximately 2 months up to approximately 6 months
Time to stop of daily plasma exchnage (PE) Approximately 2 months up to approximately 6 months
Number of days to plasma exchange Approximately 2 months up to approximately 6 months
Total volume of plasma Approximately 2 months up to approximately 6 months
Number of days in ICU and in hospital Approximately 2 months up to approximately 6 months
Change from baseline in the standardized mini mental state exam (SMMSE) total score Approximately 2 months up to approximately 6 months
Proportion of participants with at least one treatment emergent thromboembolic event Approximately 2 months up to approximately 6 months
Number of patients with treatment emergent adverse events Approximately 2 months up to approximately 6 months
Pharmacodynamic (PD) markers Approximately 2 months up to approximately 6 months
Pharmacokineticks: plasma concentration Approximately 2 months up to approximately 6 months
Immunogenicity of caplacizumab Approximately 2 months up to approximately 6 months
Enrollment 18
Condition
Intervention

Intervention Type: Drug

Intervention Name: Caplacizumab (ALX-0081)

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

Arm Group Label: Caplacizumab

Intervention Type: Drug

Intervention Name: Plasma exchange (PE)

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

Arm Group Label: Caplacizumab

Intervention Type: Drug

Intervention Name: Corticosteroid treatment (Methylprednisolone or prednisolone)

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

Arm Group Label: Caplacizumab

Intervention Type: Drug

Intervention Name: Immunosuppressive treatment (eg, rituximab)

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

Arm Group Label: Caplacizumab

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.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Clinical Sciences & Operations Study Director Sanofi
Overall Contact

Last Name: Trial Transparency email recommended (Toll free number for US & Canada)

Phone: 800-633-1610

Phone Ext.: option 6

Email: [email protected]

Location
Facility: Status:
Investigational Site Number 3920008 | Akita-Shi, Japan Recruiting
Investigational Site Number 3920009 | Iruma-Gun, Japan Recruiting
Investigational Site Number 3920014 | Kanazawa-Shi, Japan Recruiting
Investigational Site Number 3920007 | Kashihara-Shi, Japan Recruiting
Investigational Site Number 3920013 | Kawasaki-Shi, Japan Recruiting
Investigational Site Number 3920001 | Kitakyushu-Shi, Japan Recruiting
Investigational Site Number 3920002 | Kumamoto-Shi, Japan Recruiting
Investigational Site Number 3920003 | Kurashiki-Shi, Japan Recruiting
Investigational Site Number 3920010 | Kyoto-Shi, Japan Recruiting
Investigational Site Number 3920005 | Maebashi-Shi, Japan Recruiting
Investigational Site Number 3920015 | Nagoya, Japan Recruiting
Investigational Site Number 3920011 | Osaka-Shi, Japan Recruiting
Investigational Site Number 3920004 | Sapporo-Shi, Japan Recruiting
Investigational Site Number 3920006 | Sendai-Shi, Japan Recruiting
Investigational Site Number 3920012 | Wakayama-Shi, Japan Recruiting
Location Countries

Japan

Verification Date

November 2019

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Caplacizumab

Type: Experimental

Description: 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)

Patient Data Yes
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov