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Thrombotic Thrombocytopenic PurpuraPhase 2/3NCT04074187

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

A Phase 2/3 study testing Caplacizumab (ALX-0081), Plasma exchange (PE) and Corticosteroid treatment (Methylprednisolone or prednisolone) in people with Thrombotic Thrombocytopenic Purpura, run by Sanofi at 12 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Thrombotic Thrombocytopenic Purpura
What is being tested?
Caplacizumab (ALX-0081), Plasma exchange (PE) and Corticosteroid treatment (Methylprednisolone or prednisolone)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
12 sitesJapan
Study dates
21 Oct 2019to 19 May 2021
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 13Show fewer
  • 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.

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

Japan12 sites

  • Investigational Site Number 3920009

    Iruma-Gun

  • Investigational Site Number 3920014

    Kanazawa

  • Investigational Site Number 3920007

    Kashihara-shi

  • Investigational Site Number 3920013

    Kawasaki-Shi

  • Investigational Site Number 3920001

    Kitakyushu-Shi

  • Investigational Site Number 3920002

    Kumamoto

  • Investigational Site Number 3920003

    Kurashiki-Shi

  • Investigational Site Number 3920010

    Kyoto

  • Investigational Site Number 3920005

    Maebashi

  • Investigational Site Number 3920015

    Nagoya

  • Investigational Site Number 3920011

    Osaka

  • Investigational Site Number 3920006

    Sendai

Other trials in Japan

About this study

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

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.

Read more

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
Read the 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.

Being tested
Caplacizumab (ALX-0081), Plasma exchange (PE), Corticosteroid treatment (Methylprednisolone or prednisolone), Immunosuppressive treatment (eg, rituximab)
Medical subject headings
Purpura, Thrombotic Thrombocytopenic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Caplacizumab (ALX-0081)Drug

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

  • Plasma exchange (PE)Drug

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

  • Corticosteroid treatment (Methylprednisolone or prednisolone)Drug

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

  • Immunosuppressive treatment (eg, rituximab)Drug

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

Study groups in NCT04074187
Study groupRoleWhat they receive
CaplacizumabExperimentalEligible 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)
Receives: Drug: Caplacizumab (ALX-0081); Drug: Plasma exchange (PE); Drug: Corticosteroid treatment (Methylprednisolone or prednisolone); Drug: Immunosuppressive treatment (eg, rituximab)

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Approximately 2 months up to approximately 6 months

Secondary outcomes (16)

  1. Number of recurrences of TTP

    Number of recurrences of TTP during study drug treatment (including extensions) and follow-up, as well as during overall study period.

    Measured: Approximately 2 months up to approximately 6 months

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

    Proportion of participants with TTP-related death, a recurrence of TTP, or at least one treatmentemergent major thromboembolic event (eg, myocardial infarction, cerebrovascular accident, pulmonary embolism or deep venous thrombosis [DVT]) during the overall treatment period (including extensions).

    Measured: Approximately 2 months up to approximately 6 months

  3. Time to platelet count response

    Time to platelet count response, defined as initial platelet count ≥150,000/μL with subsequent stop of daily plasma exchange (PE) within 5 days.

    Measured: Approximately 2 months up to approximately 6 months

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

    Proportion of participant who have a platelet count ≥150,000/μL on Day 1, 2, 3, 4, 5 and Day 10 and end of study drug treatment (ie, last weekly visit during the overall treatment period).

    Measured: Approximately 2 months up to approximately 6 months

  5. Proportion of participants with refractory TTP

    Proportion of participant with refractory TTP, defined as persistent thrombocytopenia, lack of sustained platelet count increment or platelet counts <50,000/μL and persistently elevated LDH (>1.5 x upper limit of normal [ULN]) despite 5 PEs and steroid treatment.

    Measured: Approximately 2 months up to approximately 6 months

  6. Time to normalization of 3 organ damage marker levels

    Time to normalization of all 3 of the following organ damage marker levels: Time to LDH ≤ 1 x ULN, and cTnI ≤ 1 x ULN, and serum creatinine ≤ 1 x ULN and time to individual organ damage marker level.

    Measured: Approximately 2 months up to approximately 6 months

  7. Time to stop of daily plasma exchnage (PE)

    Time to stop of daily PE

    Measured: Approximately 2 months up to approximately 6 months

  8. Number of days to plasma exchange

    The endpoint will be assessed in 4 time periods: daily PE period (the first daily PE period only), overall treatment period, follow-up period (of 4 weeks after stop of study drug treatment), and overall study period

    Measured: Approximately 2 months up to approximately 6 months

  9. Total volume of plasma

    The endpoint will be assessed in 4 time periods: daily PE period (the first daily PE period only), overall treatment period, follow-up period (of 4 weeks after stop of study drug treatment), and overall study period

    Measured: Approximately 2 months up to approximately 6 months

  10. Number of days in ICU and in hospital

    Number of days in ICU and in hospital in 4 time periods: daily PE period (the first daily PE period only), overall treatment period, in the Follow-up period (of 4 weeks after stop of study drug treatment) and overall study period.

    Measured: Approximately 2 months up to approximately 6 months

  11. Change from baseline in the standardized mini mental state exam (SMMSE) total score

    Change from baseline in SMMSE total score on Day 1, (Day 2, 3, 4 as optional) and Day 5 of daily Plasma Exchange (PE) period, and Weeks 1 and 5 of the 30-day postdaily PE period, and the first (7 days after last dosing) and final follow-up (28 days after last dosing) visit.

    Measured: Approximately 2 months up to approximately 6 months

  12. Proportion of participants with at least one treatment emergent thromboembolic event

    The treatment-emergent major thromboembolic event (eg, myocardial infarction, cerebrovascular accident, pulmonary embolism or deep venous thrombosis [DVT]) during the overall treatment period (including extensions) will be evaluated.

    Measured: Approximately 2 months up to approximately 6 months

  13. Number of patients with treatment emergent adverse events

    Number of Patients with treatment emergent Adverse events (AEs) and serious adverse events (SAEs) and bleeding events

    Measured: Approximately 2 months up to approximately 6 months

  14. Pharmacodynamic (PD) markers

    PD parameters: von Willebrand factor antigen(vWF:Ag), coagulation factor VIII clotting activity (FVIII:C), von Willebrand factor ristocetin cofactor activity (vWF:RICO)

    Measured: Approximately 2 months up to approximately 6 months

  15. Pharmacokineticks: plasma concentration

    Total caplacizumab plasma concentrations

    Measured: Approximately 2 months up to approximately 6 months

  16. Immunogenicity of caplacizumab

    Anti-drug antibodies

    Measured: Approximately 2 months up to approximately 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Aug 2019
  2. Study start21 Oct 2019
  3. Primary completion19 May 2021
  4. Study completion19 May 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04074187
Sponsor's protocol ID
EFC16297
Other IDs
U1111-1223-4914
Status last verified
Sep 2025
First submitted
14 Aug 2019
Last updated
22 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Publications

  • 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.

    PubMed 36427162

  • 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.

    PubMed 39095866

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Caplacizumab (ALX-0081), Plasma exchange (PE) and Corticosteroid treatment (Methylprednisolone or prednisolone) in people with Thrombotic Thrombocytopenic Purpura, run by Sanofi at 12 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP) (time frame: Approximately 2 months up to approximately 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04074187, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2025.

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