Immune-mediated thrombotic thrombocytopenic purpura

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:93585M31.1
Who is this for?
Show terms as
3FDA treatments3Active trials34Specialists8Treatment centers2Financial resources

Where are you in your journey?

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

Overview

Also known as:

Clinical phenotype terms— hover any for plain English:

Microangiopathic hemolytic anemiaHP:0001937Anti-ADAMTS13 antibody positivityHP:6000462SchistocytosisHP:0001981
Orphanet ↗NORD ↗

FDA & Trial Timeline

4 events
Nov 2025An Open-label Study of Intravenous Immunoglobulin (5%) for the Treatment of Primary Immune Thrombocytopenia

Grand Shuyang Life Sciences (Chengdu) Co., Ltd. — PHASE3

TrialRECRUITING
May 2025A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

University of Minnesota — PHASE2

TrialRECRUITING
Oct 2022Improvement of Immunologic and Molecular Techniques for the Diagnosis and Follow-up of Patients With Thrombotic Thrombocytopenic Purpura

Fundación Española de Hematología y Hemoterapía

TrialACTIVE NOT RECRUITING
Mar 2021Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP

University of Cologne

TrialRECRUITING

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

Treatments

3 available

Cablivi

CAPLACIZUMAB· Genzyme Corporation
CABLIVI is indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immun

CABLIVI is indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy

Doptelet

avatrombopag· Sobi, Inc.Orphan Drug

treatment of thrombocytopenia in pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia who have had an insufficient response to a previous treatment

Adzynma

APADAMTASE ALFA· TAKEDA PHARMACEUTICALS AMERICA, INC.Orphan Drug

prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP)

Clinical Trials

3 recruitingView all trials with filters →
Phase 21 trial
A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Phase 2
Actively Recruiting
PI: Marshall Mazepa, MD (University of Minnesota) · Sites: Minneapolis, Minnesota · Age: 1899 yrs
Other2 trials
Improvement of Immunologic and Molecular Techniques for the Diagnosis and Follow-up of Patients With Thrombotic Thrombocytopenic Purpura
Active
· Sites: Coimbra, Coimbra District; A Coruña, A Coruña +4 more · Age: 099 yrs
Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP
Actively Recruiting
PI: Linus Völker, MD (Department II of Internal Medicine, University of ) · Sites: Cologne · Age: 1899 yrs

Specialists

Showing 25 of 34View all specialists →
PC
Paul Coppo
Specialist
15 Immune-mediated thrombotic thrombocytopenic purpura publications
PP
Pascale Poullin
Specialist
7 Immune-mediated thrombotic thrombocytopenic purpura publications
KS
Kazuya Sakai
Specialist
7 Immune-mediated thrombotic thrombocytopenic purpura publications
RB
Raïda Bouzid
Specialist
7 Immune-mediated thrombotic thrombocytopenic purpura publications
AV
Agnès Veyradier
Specialist
10 Immune-mediated thrombotic thrombocytopenic purpura publications
JW
Julia Weisinger
Specialist
3 Immune-mediated thrombotic thrombocytopenic purpura publications
BJ
Bérangère Joly
Specialist
3 Immune-mediated thrombotic thrombocytopenic purpura publications
AW
Alain Wynckel
Specialist
4 Immune-mediated thrombotic thrombocytopenic purpura publications
EA
Elie Azoulay
Specialist
4 Immune-mediated thrombotic thrombocytopenic purpura publications
FP
Flora Peyvandi
Specialist
4 Immune-mediated thrombotic thrombocytopenic purpura publications
PA
Pasquale Agosti
Specialist
4 Immune-mediated thrombotic thrombocytopenic purpura publications
BJ
Bérangère S Joly
Specialist
5 Immune-mediated thrombotic thrombocytopenic purpura publications
MM
Masanori Matsumoto
Specialist
5 Immune-mediated thrombotic thrombocytopenic purpura publications
YB
Ygal Benhamou
Specialist
6 Immune-mediated thrombotic thrombocytopenic purpura publications
XM
Xiaohui Zhang, MD
Beijing, FL
Specialist

Rare Disease Specialist

PI on 12 active trials
IP
Ilaria Mancini, PhD
Specialist
PI on 1 active trial
MM
Marshall Mazepa, MD
MINNEAPOLIS, MN
Specialist
PI on 1 active trial
XM
Xiao-Hui Zhang, MD
Specialist
PI on 16 active trials
IM
Ilaria Mancini
Specialist
3 Immune-mediated thrombotic thrombocytopenic purpura publications
LV
Linus Alexander Völker
Specialist
2 Immune-mediated thrombotic thrombocytopenic purpura publications
CP
Christopher J Patriquin
Specialist
2 Immune-mediated thrombotic thrombocytopenic purpura publications
MR
Marienn Réti
Specialist
2 Immune-mediated thrombotic thrombocytopenic purpura publications
CP
Cristina Pascual-Izquierdo
Specialist
2 Immune-mediated thrombotic thrombocytopenic purpura publications
JW
John-Paul Westwood
Specialist
2 Immune-mediated thrombotic thrombocytopenic purpura publications
MT
Mari R Thomas
Specialist
2 Immune-mediated 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

Financial Resources

2 resources
Cablivi(CAPLACIZUMAB)Genzyme Corporation
Adzynma(APADAMTASE ALFA)TAKEDA PHARMACEUTICALS AMERICA, INC.

Travel Grants

No travel grants are currently matched to Immune-mediated thrombotic thrombocytopenic purpura.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Immune-mediated thrombotic thrombocytopenic purpuraForum →

No community posts yet. Be the first to share your experience with Immune-mediated thrombotic thrombocytopenic purpura.

Start the conversation →

Latest news about Immune-mediated thrombotic thrombocytopenic purpura

Disease timeline:

New recruiting trial: An Open-label Study of Intravenous Immunoglobulin (5%) for the Treatment of Primary Immune Thrombocytopenia

A new clinical trial is recruiting patients for Immune-mediated thrombotic thrombocytopenic purpura

New recruiting trial: Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia

A new clinical trial is recruiting patients for Immune-mediated thrombotic thrombocytopenic purpura

New recruiting trial: A Prospective, One-arm and Open Clinical Study of Obinutuzumab in the Treatment of Pediatric Primary Immune Thrombocytopenia (ITP)

A new clinical trial is recruiting patients for Immune-mediated thrombotic thrombocytopenic purpura

New recruiting trial: Short-course High-dose Prednisone and Dexamethasone in Children With ITP

A new clinical trial is recruiting patients for Immune-mediated thrombotic thrombocytopenic purpura

New recruiting trial: Phase I Study of SHR-2173 Injection in Patients With Primary Immune Thrombocytopenia

A new clinical trial is recruiting patients for Immune-mediated thrombotic thrombocytopenic purpura

New recruiting trial: Exploring the Efficacy, Safety of a Modified Starting Dosage of Avatrombopag in Immune Throbocytopenia (ITP) - a Pilot Study

A new clinical trial is recruiting patients for Immune-mediated thrombotic thrombocytopenic purpura

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 Immune-mediated thrombotic thrombocytopenic purpura

Are there clinical trials for Immune-mediated thrombotic thrombocytopenic purpura?

Yes — 3 recruiting clinical trials are currently listed for Immune-mediated 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 Immune-mediated thrombotic thrombocytopenic purpura?

25 specialists and care centers treating Immune-mediated 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 Immune-mediated thrombotic thrombocytopenic purpura?

2 patient support programs are currently tracked on UniteRare for Immune-mediated thrombotic thrombocytopenic purpura. See the treatments and support programs sections for copay assistance, eligibility, and contact details.