A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

Plain-language summary

This study observationally follows children and adults with congenital thrombotic thrombocytopenic purpura (cTTP) who are treated with Adzynma; no additional intervention is given as part of the study. It can enroll people diagnosed with cTTP who have previously received commercially available Adzynma treatment. It is a study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Takeda

Intervention

No Intervention

Start date

5/26/2026

Estimated completion

4/1/2030

Countries

Austria, Germany

Related genes

Locations

• Vienna, Austria
• Bonn, Germany
• Cologne, Germany
• Jena, Germany
• Leipzig, Germany
• München, Germany
• München, Germany

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion criteria:

1. Is diagnosed with cTTP.
2. Had received commercially available Adzynma treatment for cTTP within the eligibility period of which there must be a recorded date and dose of at least one Adzynma administration in their medical chart.
3. Provides a signed informed consent form (informed consent form \[ICF\]; or assent and consent forms, if applicable), in accordance with local ethical and institutional requirements.

Exclusion criteria:

1. Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13).
2. Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.