A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorTakeda
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial population
Pediatric and adult participants who have received Adzynma for the treatment of cTTP, either prophylactically or as on-demand therapy for acute episodes-including those who became pregnant during Adzynma treatment.
Trial design
Design model
Cohort
Time perspective
Retrospective
Treatments tested in this trial
No Intervention
Other interventionThis is a non-interventional study.
Treatment groups
50 Participants
are divided into 1 treatment groupGroup A: All Participants1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
Index infusion is defined as the first Adzynma infusion date recorded in the medical record using commercially available Adzynma (excluding doses received in clinical studies or early access program).
Time frame
Up to 6 months following the index Adzynma infusion2
Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions
Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported.
Time frame
Up to 6 months following the index Adzynma infusionSecondary outcomes
1
Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion
Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported.
Time frame
Up to 6 months of follow-up time from the index Adzynma infusion2
Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion
Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported.
Time frame
Up to 7 days after each administration of Adzynma3
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product.
Time frame
From start of the study drug administration up to 5.5 years4
Gestational age at the Time of Infant Birth
Gestational age at the time of infant birth will be reported.
Time frame
At birth Source ClinicalTrials.gov