An observational study in Thrombotic Thrombocytopenic Purpura (TTP), sponsored by Takeda. Recruiting at 7 sites in 2 countries. Per ClinicalTrials.gov, last updated 2026-11-06.
Sponsored by Takeda · Observational
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.
67 studies on the registry are indexed under Purpura, Thrombotic Thrombocytopenic; 18 are open to participants now.
This study's planned enrollment of 50 is below the median of 156 across 27 observational studies indexed under Purpura, Thrombotic Thrombocytopenic.
Browse Purpura, Thrombotic Thrombocytopenic studies →Takeda is the lead sponsor of 1,026 studies on the registry; 89 are open to participants now.
Of its 178 completed or terminated interventional studies of FDA-regulated products, 154 (87%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Data of participants who have received Adzynma for the treatment of cTTP will be collected retrospectively from medical records for approximately 5 years.
Other: No Intervention
This is a non-interventional study.
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 infusion
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 infusion
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 infusion
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 Adzynma
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 years
Gestational age at the Time of Infant Birth
Gestational age at the time of infant birth will be reported.
Time frame: At birth
Number of Participants With Spontaneous Abortion
Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation.
Time frame: Up to 5.5 years
Number of Participants With Stillbirth
Stillbirth is defined as a fetal death after 20 weeks of gestation.
Time frame: Up to 5.5 years
Number of Participants With Induced Abortion
Number of participants with induced abortion will be reported.
Time frame: Up to 5.5 years
Number of Participants With Live Birth
Number of participants with live birth will be reported.
Time frame: Up to 5.5 years
Number of Participants With any Pregnancy Related Complications
Number of participants with any pregnancy related complications will be reported.
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
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 will be reported.
Time frame: At birth
Number of Infants Categorized According to Year of Birth
Number of infants categorized according to year of birth will be reported.
Time frame: Up to 5.5 years
Number of Infants With Normal Birth Weight
Number of infants with normal birth weight will be reported.
Time frame: Up to 5.5 years
Number of Infants With Small for Gestational Age (SGA)
Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age.
Time frame: Up to 5.5 years
Number of Infants With Congenital Anomaly Detected at the Time of Birth
Number of infants with congenital anomaly detected at the time of birth will be reported.
Time frame: At birth
Number of Participants by Breastfeeding Status While Receiving Adzynma
Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown).
Time frame: Up to 5.5 years
Number of Infant Categorized by Growth and Development Outcomes
Number of infants categorized by growth and development outcomes (normal, abnormal \[specify\], or unknown) will be reported.
Time frame: Up to 5.5 years
Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Data sharing details ↗ · vivli.org
Started May 2026 (actual). Primary completion is estimated for Apr 2030, 46 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Purpura, Thrombotic Thrombocytopenic →