Actively Recruiting
Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura: Multicentric Non-inferiority Single-arm Study
Led by University Hospital, Rouen · Updated on 2026-02-17
131
Participants Needed
30
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Immune thrombotic thrombocytopenic purpura (iTTP) is a serious condition caused by a severe deficiency of ADAMTS13, leading to the buildup of large von Willebrand factor multimers and formation of blood vessel clots. The current standard treatment combines daily plasma exchange (PEX), immunosuppressive drugs, and caplacizumab, which has shown to reduce death, complications, and the number of PEX sessions. However, PEX is invasive, time-consuming, and linked to complications, so researchers are exploring treatments without PEX to ease patient care. This study aims to evaluate the safety and effectiveness of a regimen without PEX using plasma infusion, corticosteroids/rituximab, and caplacizumab. The study replaces daily PEX with daily plasma infusions at a dose of 15 mL per kilogram of body weight, using different types of plasma such as quarantine fresh frozen plasma or inactivated plasma. This PEX-free regimen is combined with immunosuppression using corticosteroids and rituximab alongside the anti-adhesive drug caplacizumab. This approach is being tested as a single experimental treatment group for adults diagnosed with iTTP. Participants will be monitored for 30 days after starting plasma therapy to evaluate treatment outcomes. Researchers will assess the proportion of participants who experience death, treatment failure, disease worsening, or low ADAMTS13 activity below 20%. The study includes clinical and laboratory evaluations to track safety and effectiveness. The overall goal is to determine if this PEX-free regimen can provide a feasible alternative treatment for iTTP with fewer complications.
CONDITIONS
Brief Title
Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adult patient 18 years or older
- Clinical diagnosis of iTTP based on standard clinical and laboratory criteria (French Score 2), including thrombotic microangiopathy with platelet count 30 G/L and serum creatinine 200 bcmol/L
- Patient has read and understood the information letter and signed the Informed Consent Form or consent provided by representative if patient unable
- Patient affiliated with or beneficiary of a social security (national health insurance) plan
- For women of childbearing potential: effective contraception for at least 1 month and negative blood pregnancy test
- Women surgically sterile or postmenopausal for at least 12 months prior to inclusion visit
You will not qualify if you...
- Platelet count greater than 100 G/L before plasma treatment
- Patients with a French score less than 2 (unless previous TTP flare) or serum creatinine level greater than 200 bcmol/L
- Other known causes of cytopenias or organ failure such as uncontrolled cancer, chemotherapy, transplant, drugs, or AIDS stage HIV
- Severe neurological disorder such as seizure, coma, focal deficiency, or consciousness trouble
- Pregnant women, breastfeeding women, or those planning pregnancy
- Weight over 100 kilograms
- Congenital TTP
- Clinically significant active bleeding or high risk of bleeding (excluding thrombocytopenia)
- Chronic anticoagulant treatment that cannot be safely interrupted
- Malignant hypertension
- Contraindications to caplacizumab, plasma treatment, corticosteroids, rituximab, or their excipients
- Persons deprived of liberty or under judicial protection
- Participation in another drug interventional clinical trial within 30 days prior to inclusion and during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - 30 days post plasma therapy
Participants receive a regimen combining daily plasma infusions, immunosuppressive agents, and caplacizumab without therapeutic plasma exchange.
Daily visits for plasma infusions during treatment period
Trial Site Locations
Total: 30 locations
1
Chu Amiens
Amiens, France, 80054
Actively Recruiting
2
Chu Angers
Angers, France, 49933
Actively Recruiting
3
Chru Besanon
Besançon, France, 25030
Actively Recruiting
4
Chu Bobigny
Bobigny, France, 93000
Actively Recruiting
5
Hopital Jean Verdie
Bondy, France, 93140
Actively Recruiting
6
Chu Bordeaux
Bordeaux, France, 33076
Actively Recruiting
7
Chu Clermont-Ferrand
Clermont-Ferrand, France, 63003
Actively Recruiting
8
Chu Lille
Lille, France, 59037
Actively Recruiting
9
Chu Limoges
Limoges, France, 87042
Not Yet Recruiting
10
Chu Edouard Herriot
Lyon, France, 69003
Actively Recruiting
11
Ap-Hm La Conception
Marseille, France, 13005
Actively Recruiting
12
Chu Montpellier
Montpellier, France, 34295
Actively Recruiting
13
Chu Nancy
Nancy, France, 54500
Actively Recruiting
14
Chu Nantes
Nantes, France, 44093
Actively Recruiting
15
CHU NICE
Nice, France, 06200
Actively Recruiting
16
Chu Nimes
Nîmes, France, 30029
Actively Recruiting
17
Ap-Hp Saint Louis
Paris, France, 75010
Actively Recruiting
18
Ap-Hp St Antoine
Paris, France, 75571
Actively Recruiting
19
Ap-Hp Pitie Salpetriere
Paris, France, 75651
Not Yet Recruiting
20
CH PAU
Pau, France, 64046
Not Yet Recruiting
21
Chu Reims
Reims, France, 51092
Actively Recruiting
22
Chu Rouen
Rouen, France, 76031
Actively Recruiting
23
Ch Saint-Nazaire
Saint-Nazaire, France, 44606
Not Yet Recruiting
24
Chu Strasbourg
Strasbourg, France, 67091
Actively Recruiting
25
Chu Toulouse
Toulouse, France, 31000
Actively Recruiting
26
Chu Tours
Tours, France, 37044
Not Yet Recruiting
27
Ch Valenciennes
Valenciennes, France, 59322
Actively Recruiting
28
Chu Martinique
Fort-de-France, Martinique, 97261
Not Yet Recruiting
29
Reunion Nord
Saint-Denis, Reunion, 97400
Actively Recruiting
30
Chu Reunion Sud
Saint-Pierre, Reunion, 97448
Actively Recruiting
Research Team
Y
Ygal Benhamou, Pr
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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