Thrombotic thrombocytopenic purpura

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:54057OMIM:274150M31.1
Who is this for?
Show terms as
2FDA treatments8Active trials22Specialists8Treatment centers1Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Thrombotic thrombocytopenic purpura is treated with 1 medication in our database, including ACD-A. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Fresenius Kabi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Thrombotic thrombocytopenic purpura treatment below.

Also known as:

Clinical phenotype terms— hover any for plain English:

Microangiopathic hemolytic anemiaHP:0001937Abnormal circulating lactate dehydrogenase concentrationHP:0045040
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

9 events
Apr 2025Association of TNFAIP3 With Immune-mediated TTP

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico — NA

TrialRECRUITING
Dec 2024Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center

Assistance Publique - Hôpitaux de Paris

TrialRECRUITING
Jun 2024Italian iTTP Registry

Fondazione Luigi Villa

TrialRECRUITING
May 2023Immunological Aspect of Thrombotic Thrombocytopenic Purpura (TTP)

University Hospital, Rouen

TrialRECRUITING
Mar 2023A Study of TAK-755 (rADAMTS13) With Little to No Plasma Exchange (PEX) Treatment in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

Takeda — PHASE2

TrialACTIVE NOT RECRUITING
Dec 2022Turkey Thrombotic Thrombocytopenic Purpura Disease Registry: National Multicenter Study

Turkish Hematology Association

TrialRECRUITING
Oct 2022Construction of a Database for TTP

The First Affiliated Hospital of Soochow University

TrialRECRUITING
Apr 2021A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Takeda — PHASE3

TrialRECRUITING
Feb 2019Cablivi: FDA approved

CABLIVI is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

2 available

Cablivi

CAPLACIZUMAB· Genzyme CorporationOrphan Drug

CABLIVI is indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

ACD-A

Anticoagulant Citrate Dextrose Solution Formula A· Fresenius Kabi

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Clinical Trials

8 recruitingView all trials with filters →
Phase 31 trial
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Phase 3
Actively Recruiting
PI: Study Director (Takeda) · Sites: Atlanta, Georgia; Buffalo, New York +25 more · Age: 070 yrs
Phase 21 trial
A Study of TAK-755 (rADAMTS13) With Little to No Plasma Exchange (PEX) Treatment in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Phase 2
Active
PI: Study Director (Takeda) · Sites: Gainesville, Florida; Boston, Massachusetts +20 more · Age: 1899 yrs
N/A1 trial
Association of TNFAIP3 With Immune-mediated TTP
N/A
Actively Recruiting
PI: Ilaria Mancini, PhD (Fondazione IRCCS Ca' Granda, Ospedale Maggiore Pol) · Sites: Milan · Age: 1299 yrs
Other5 trials
Construction of a Database for TTP
Actively Recruiting
· Sites: Suzhou, Jiangsu
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
Actively Recruiting
PI: Paul COPPO, MD, PHD (Assistance Publique - Hôpitaux de Paris) · Sites: Paris
Turkey Thrombotic Thrombocytopenic Purpura Disease Registry: National Multicenter Study
Actively Recruiting
PI: ilker kurkcu (Sentez-CRO) · Sites: Ankara · Age: 1899 yrs
Immunological Aspect of Thrombotic Thrombocytopenic Purpura (TTP)
Actively Recruiting
· Sites: Rouen · Age: 1899 yrs
Italian iTTP Registry
Actively Recruiting
PI: Flora Peyvandi, MD, PhD (Fondazione IRCCS Ca' Granda Ospedale Maggiore Poli) · Sites: Milan, MI · Age: 1299 yrs

Specialists

22 foundView all specialists →
IP
Ilaria Mancini, PhD
Specialist
PI on 1 active trial
LM
Lucas Kühne, MD
Specialist
PI on 1 active trial1 Thrombotic thrombocytopenic purpura publication
LM
lei zhang, MD
Specialist
PI on 1 active trial1 Thrombotic thrombocytopenic purpura publication
CB
Charlotte Bradbury
Specialist
PI on 2 active trials1 Thrombotic thrombocytopenic purpura publication
VM
Victor Blanchette, MD
Specialist
PI on 1 active trial
MM
Manuel Carcao, MD
Specialist
PI on 1 active trial
FZ
Francesco Zaja
Specialist
PI on 2 active trials2 Thrombotic thrombocytopenic purpura publications
MD
Mengtao Li, Dr.
Specialist
PI on 1 active trial
AM
Alexandra Schifferli, Dr. med.
Specialist
PI on 1 active trial
GM
Gregory Cheng, PhD, MD
Specialist
PI on 1 active trial1 Thrombotic thrombocytopenic purpura publication
XM
Xiao-Hui Zhang, MD
Specialist
PI on 16 active trials
TG
Tomás González
Specialist
PI on 1 active trial10 Thrombotic thrombocytopenic purpura publications

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

1 grants

Accessia Health: Thrombotic Thrombocytopenic Purpura (TTP) - Public Insurance

Accessia Health

Apply ↗

Community

Open Thrombotic thrombocytopenic purpuraForum →

No community posts yet. Be the first to share your experience with Thrombotic thrombocytopenic purpura.

Start the conversation →

Latest news about Thrombotic thrombocytopenic purpura

No recent news articles for Thrombotic thrombocytopenic purpura.

Follow this condition to be notified when news becomes available.

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Thrombotic thrombocytopenic purpura

What is Thrombotic thrombocytopenic purpura?

Thrombotic thrombocytopenic purpura is treated with 1 medication in our database, including ACD-A. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Fresenius Kabi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Thrombotic thrombocytopenic purpura treatment below.

Are there clinical trials for Thrombotic thrombocytopenic purpura?

Yes — 8 recruiting clinical trials are currently listed for Thrombotic thrombocytopenic purpura on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Thrombotic thrombocytopenic purpura?

22 specialists and care centers treating Thrombotic thrombocytopenic purpura are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Thrombotic thrombocytopenic purpura?

1 FDA-approved treatment and 1 patient support program are currently tracked on UniteRare for Thrombotic thrombocytopenic purpura. See the treatments and support programs sections for copay assistance, eligibility, and contact details.