A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No Intervention.
- Who it may be relevant to
- Registry conditions: Thrombotic Thrombocytopenic Purpura (TTP). Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Austria, Germany
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Overview
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13. The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant. Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.
Interventions
- Other No Intervention
This is a non-interventional study.
Primary outcome measures
- Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions [Time frame: Up to 6 months following the index Adzynma infusion]
- Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions [Time frame: Up to 6 months following the index Adzynma infusion]
Secondary outcome measures (12)
- Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion [Time frame: Up to 6 months of follow-up time from the index Adzynma infusion]
- Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion [Time frame: Up to 7 days after each administration of Adzynma]
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) [Time frame: From start of the study drug administration up to 5.5 years]
- Gestational age at the Time of Infant Birth [Time frame: At birth]
- Number of Participants With Spontaneous Abortion [Time frame: Up to 5.5 years]
- Number of Participants With Stillbirth [Time frame: Up to 5.5 years]
- Number of Participants With Induced Abortion [Time frame: Up to 5.5 years]
- Number of Participants With Live Birth [Time frame: Up to 5.5 years]
- Number of Participants With any Pregnancy Related Complications [Time frame: Up to 5.5 years]
- Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth [Time frame: At birth]
- Number of Infants Categorized According to Year of Birth [Time frame: Up to 5.5 years]
- Number of Infants With Normal Birth Weight [Time frame: Up to 5.5 years]
Eligibility criteria
Inclusion criteria
- Is diagnosed with cTTP.
- 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.
- 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
- Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13).
- Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Germany · 6 centers
- Universitatsklinikum Bonn — Bonn
- Universitaetsklinikum Koeln — Cologne
- Universitaetsklinikum Jena — Jena
- Universitaetsklinikum Leipzig — Leipzig
- Klinikum Rechts der Isar der Technischen Universitaet Muenchen — München
- LMU Klinikum — München
Austria · 1 center
- AKH - Medizinische Universitaet Wien — Vienna
Identifiers
NCT: NCT07353099 · TAK-755-4007 · EUPAS1000000870