Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult aTTP

NCT04720261 | Terminated Phase 2 DMC

• 1 other identifier: 2019/0408/HP
• Study Type: interventional
• Target: 58
• Locations: 2 countries
• Sites: 24

Brief Summary

The aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study.

Conditions

Thrombotic Thrombocytopenic Purpura, Acquired

Outcome Measures

Primary Outcomes (1)

• To evaluate the feasibility of a personalized caplacizumab regimen in aTTP based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-PE treatment

  composite endpoint defined by the occurrence of at least one the following events during the 30 days post-PE treatment: death, refractoriness or exacerbation.

  30 days post-PE treatment

Secondary Outcomes (12)

• Response to treatment (platelet count recovery)

  30 days post-PE treatment

• Durable remission achievement

  90 days post-PE treatment

• Mortality at D90 post-PE treatment

  90 days post-PE treatment

• Refractoriness at D30 post-PE treatment

  Day 30 post-PE treatment

• Exacerbation at D30 post-PE treatment

  Day 30 post-PE treatment

Study Arms (1)

Caplacizumab

OTHER

All patients in the study are aTTP and needs to be treated by caplacizumab. The duration of this treatment will be evaluated through the ADAMTS 13 activity.

Drug: Caplacizumab

Interventions

Caplacizumab DRUG

Analyse of ADAMTS13 activity in patients with aTTP treated with Caplacizumab in order to help adapting the treatment with caplacizumab in TTP patients

Caplacizumab

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patients ≥ 18 years;
• Patient having read and understood the information letter and signed the Informed Consent Form. If the patient is unable to express his consent, the consent will be signed by his representative ((1) the trusted person, or failing that, (2) a family member, or (3) a close relative of the person concerned). In this case, consent to continue the study will subsequently be requested from the patient (article L1122-1-1 of the CSP);
• Patient affiliated with, or beneficiary of a social security (national health insurance) plan;
• For women:
• Women of childbearing potential :
• Effective contraception according to WHO definition (estrogen-progestin or intrauterine device or tubal ligation) since at least 1 month and;
• Negative blood pregnancy test;
• Women surgically sterile (absence of ovaries and/or uterus);

You may not qualify if:

• Platelet count \> 100 G/L;
• Patients with a French score \< 2 (a serum creatinine level \> 200 μmol/L +/- associated with a platelet count \> 30 G/L), in order to exclude possible cases of atypical hemolytic uremic syndrome;
• Known other causes of cytopenias and/or organ failure including but not limited to: uncontrolled cancer, chemotherapy, transplant, drugs, HIV at AIDS stage;
• Pregnant women (positive result from a blood pregnancy test) or patients with an imminent project of pregnancy; breastfeeding women (due to lack of pharmacological data for caplacizumab during pregnancy and breastfeeding);
• Congenital TTP;
• Clinically significant active bleeding or high risk of bleeding (excluding thrombocytopenia);
• Chronic treatment with anticoagulant that cannot be interrupted safely, including but not limited to: vitamin K antagonists, direct oral anticoagulant, low molecular weight heparin or heparin;
• Malignant hypertension;
• Contra-indication to CABLIVI 10 mg powder and solvent for solution for injection: hypersensitivity to caplacizumab or to any of the excipients;
• Contra-indication to PE treatment;
• Contra-indication to corticosteroid (= ((methyl)prednisone or (methyl)prednisolone)) or excipients;
• Contra-indication to rituximab or excipients and to its premedication;
• Person deprived of liberty by administrative or judicial decision or placed under judicial protection (guardianship or supervision);

Sponsors & Collaborators

• University Hospital, Rouen: lead

Study Sites (24)

Chu Amiens

Amiens, 80054, France

Location

Chu Angers

Angers, 49933, France

Location

Chu Besancon

Besançon, 25030, France

Location

Chu Bordeaux

Bordeaux, 33404, France

Location

CHU CAEN

Caen, 14033, France

Location

Chu Clermont-Ferrand

Clermont-Ferrand, 63003, France

Location

Chu Lille

Lille, 59037, France

Location

Chu Limoges

Limoges, 87042, France

Location

Ch Edouard Herriot

Lyon, 69003, France

Location

Ap-Hm Hopital de La Conception

Marseille, 13005, France

Location

Chu Montpellier

Montpellier, 34295, France

Location

Chu Nancy

Nancy, 54035, France

Location

Chu Nantes

Nantes, 44093, France

Location

Ah-Hp Hopital Saint Louis

Paris, 75010, France

Location

Ap-Hp Hopital de Cochin

Paris, 75014, France

Location

Ap-Hp Hopital de Necker

Paris, 75015, France

Location

Ap-Hp Hopital de St Antoine

Paris, 75571, France

Location

Chu Reims

Reims, 51092, France

Location

Chu Rennes

Rennes, 35033, France

Location

Chu Rouen

Rouen, 76031, France

Location

Reunion Sud

Saint-Paul, 97448, France

Location

Chu Tours

Tours, 37044, France

Location

Ch Valenciennes

Valenciennes, 59300, France

Location

Fort de France

Fort-de-France, 97261, Martinique

Location

MeSH Terms

Conditions

Thrombotic thrombocytopenic purpura, acquired

Interventions

caplacizumab

Study Design

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

Model Details: Experimental, prospective, non-comparative, multicentric national study

Study Record Dates

• First Submitted: January 12, 2021
• First Posted: January 22, 2021
• Study Start: April 26, 2021
• Primary Completion: May 16, 2023
• Study Completion: May 16, 2023
• Last Updated: February 20, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

FR (23); MA (1)