Congenital thrombotic thrombocytopenic purpura

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ORPHA:93583OMIM:274150M31.1
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1FDA treatments2Active trials31Specialists8Treatment centers1Financial resources

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

Also known as:

Congenital ADAMTS-13 deficiencyCongenital TTPcTTPFamilial TTPMoschcowitz diseaseTTPUpshaw-Schulman syndrome
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
May 2026TGD001 Treatment in Thrombotic Microangiopathies

TargED Biopharmaceuticals B.V. — PHASE1, PHASE2

TrialNOT YET RECRUITING
Apr 2026A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

Takeda

TrialNOT YET RECRUITING
Apr 2026A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program

Takeda

TrialNOT YET RECRUITING
Dec 2024Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors

US Thrombotic Microangiopathy Alliance

TrialNOT YET RECRUITING
May 2024A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Takeda

TrialRECRUITING
Apr 2024Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura

University Hospital, Rouen — NA

TrialRECRUITING
Mar 2024Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry

Fundación Española de Hematología y Hemoterapía

TrialRECRUITING
Oct 2022Aspirin for Prophylaxis of TTP

The First Affiliated Hospital of Soochow University — PHASE2, PHASE3

TrialNOT YET RECRUITING
Feb 2021A Neurocognitive Study in Patients With Brain Oligometastases Receiving Hypofractionated SRT

Chang Gung Memorial Hospital

TrialRECRUITING
Oct 2006Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)

Insel Gruppe AG, University Hospital Bern

TrialRECRUITING

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

Treatments

1 available

Adzynma

APADAMTASE ALFA· TAKEDA PHARMACEUTICALS AMERICA, INC.

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

View Details →FDA Label ↗Patient Assistance ↗

Clinical Trials

2 recruitingView all trials with filters →
Other2 trials
A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Actively Recruiting
PI: Study Director (Takeda) · Sites: Tokyo
View on ClinicalTrials.gov ↗I'm interested →
Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
Actively Recruiting
PI: Johanna A Kremer Hovinga, MD (University Clinic of Hematology and Central Hemato) · Sites: Oklahoma City, Oklahoma; Vienna +5 more
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 31View all specialists →
ZL
Zhanguo Li
Specialist
PI on 7 active trials819 Congenital thrombotic thrombocytopenic purpura publications
HM
Houman Hemmati, MD
LOS ANGELES, CA
Specialist
PI on 3 active trials
DP
David KLATZMANN, MD, PhD
Specialist
PI on 2 active trials
WF
Wolfgang Frenzel
Specialist
PI on 4 active trials
FP
Flora Peyvandi, MD, PhD
Specialist
PI on 3 active trials
XM
Xiao-Hui Zhang, MD
Specialist
PI on 16 active trials
DM
David T. Teachey, MD
PHILADELPHIA, PA
Specialist
PI on 1 active trial
FR
Frédéric Rieux-Laucat
Specialist
PI on 1 active trial12 Congenital thrombotic thrombocytopenic purpura publications
AR
Anat Rabinovich
Specialist
3 Congenital thrombotic thrombocytopenic purpura publications
OE
Offer Erez
Specialist
3 Congenital thrombotic thrombocytopenic purpura publications
JW
Jerzy Windyga
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
LW
Linda T Wang
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
AT
Alice Taylor
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
BL
Bernhard Lämmle
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
NG
Nathalie Gouge-Biebuyck
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
YD
Yahsou Delmas
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
IS
Ilan Shapira
NEW YORK, NY
Specialist
PI on 2 active trials
HT
Howard Trachtman
NEW YORK, NY
Specialist
PI on 1 active trial
SC
Spero R Cataland
COLUMBUS, OH
Specialist
PI on 1 active trial
EM
Elaine M Majerus
SAINT LOUIS, MO
Specialist
PI on 1 active trial
AJ
Adrien Joseph
HOUSTON, TX
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
MS
Marie Scully
WEST HARTFORD, CT
Specialist
6 Congenital thrombotic thrombocytopenic purpura publications
BK
Barbara A Konkle
SEATTLE, WA
Specialist
PI on 2 active trials
MP
Munjal Patel
PLYMOUTH, MI
Specialist
2 Congenital thrombotic thrombocytopenic purpura publications
SA
Susan F. Assmann
Specialist
PI on 2 active trials

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

1 resources
Adzynma(APADAMTASE ALFA)— TAKEDA PHARMACEUTICALS AMERICA, INC.

Travel Grants

No travel grants are currently matched to Congenital thrombotic thrombocytopenic purpura.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Congenital thrombotic thrombocytopenic purpura

Disease timeline:

New recruiting trial: A Neurocognitive Study in Patients With Brain Oligometastases Receiving Hypofractionated SRT

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Turkey Thrombotic Thrombocytopenic Purpura Disease Registry: National Multicenter Study

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Italian iTTP Registry

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Construction of a Database for TTP

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Immunological Aspect of Thrombotic Thrombocytopenic Purpura (TTP)

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Association of TNFAIP3 With Immune-mediated TTP

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP

A new clinical trial is recruiting patients for Congenital thrombotic thrombocytopenic purpura

New recruiting trial: Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry

A new clinical trial is recruiting patients for Congenital 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 Congenital thrombotic thrombocytopenic purpura

Are there clinical trials for Congenital thrombotic thrombocytopenic purpura?

Yes — 2 recruiting clinical trials are currently listed for Congenital 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 Congenital thrombotic thrombocytopenic purpura?

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