Thrombotic Thrombocytopenic Purpura (TTP)
Conditions
Keywords
Drug Therapy
Brief summary
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
OTHERNo Intervention
This is a non-interventional study.
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions | Up to 6 months following the index Adzynma infusion | 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). |
| Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions | Up to 6 months following the index Adzynma infusion | Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion | Up to 6 months of follow-up time from the index Adzynma infusion | Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported. |
| Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion | Up to 7 days after each administration of Adzynma | Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From start of the study drug administration up to 5.5 years | 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. |
| Gestational age at the Time of Infant Birth | At birth | Gestational age at the time of infant birth will be reported. |
| Number of Participants With Spontaneous Abortion | Up to 5.5 years | Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation. |
| Number of Participants With Stillbirth | Up to 5.5 years | Stillbirth is defined as a fetal death after 20 weeks of gestation. |
| Number of Participants With Induced Abortion | Up to 5.5 years | Number of participants with induced abortion will be reported. |
| Number of Participants With Live Birth | Up to 5.5 years | Number of participants with live birth will be reported. |
| Number of Participants With any Pregnancy Related Complications | Up to 5.5 years | Number of participants with any pregnancy related complications will be reported. |
| 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 | 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. |
| Number of Infants Categorized According to Year of Birth | Up to 5.5 years | Number of infants categorized according to year of birth will be reported. |
| Number of Infants With Normal Birth Weight | Up to 5.5 years | Number of infants with normal birth weight will be reported. |
| Number of Infants With Small for Gestational Age (SGA) | Up to 5.5 years | Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age. |
| Number of Infants With Congenital Anomaly Detected at the Time of Birth | At birth | Number of infants with congenital anomaly detected at the time of birth will be reported. |
| Number of Participants by Breastfeeding Status While Receiving Adzynma | Up to 5.5 years | Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown). |
| Number of Infant Categorized by Growth and Development Outcomes | Up to 5.5 years | Number of infants categorized by growth and development outcomes (normal, abnormal \[specify\], or unknown) will be reported. |
Countries
Austria, Germany
Contacts
CONTACTTakeda Contact
STUDY_DIRECTORStudy Director
Takeda
Outcome results
None listed