Congenital thrombotic thrombocytopenic purpura

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At a Glance

Live from database

1FDA treatments

2Recruiting trials

Regulatory Watchboard

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

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for Congenital thrombotic thrombocytopenic purpura.
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Approved Treatments

1 FDA-approved

Adzynma

(APADAMTASE ALFA)Orphan drugstandard

TAKEDA PHARMACEUTICALS AMERICA, INC.

12.1 Mechanism of Action ADZYNMA is a recombinant form of the endogenous ADAMTS13. ADAMTS13 is a plasma zinc metalloprotease that regulates the activi...

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Clinical Trial Landscape

2 active trials

2Unknown

2Total recruiting

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Recent News & Research

No recent news articles indexed yet for Congenital thrombotic thrombocytopenic purpura.
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Specialist Network

Top 6 by expertise

Zhanguo Li

Peking University Institute of Rheuamotology and Immunology

Score 427 trials819 pubs

David KLATZMANN, MD, PhD

Assistance Publique - Hôpitaux de Paris

Score 62 trials

Houman Hemmati, MD, M.D., Ph.D

Sanguine Biosciences

📍 LOS ANGELES, CA

Score 63 trials

Wolfgang Frenzel

International Medical Director

Score 64 trials

Xiao-Hui Zhang, MD

Peking University Institute of Hematology, Peking University People's Hospital

Score 316 trials

Frédéric Rieux-Laucat

Institut Imagine

Score 31 trial12 pubs

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