International Registry of Patients With Alpha Thalassemia (ATM Registry)
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|ClinicalTrials.gov Identifier: NCT04872179|
Recruitment Status : Recruiting
First Posted : May 4, 2021
Last Update Posted : October 4, 2021
|Condition or disease|
|Alpha-Thalassemia Alpha Thalassemia Major Alpha Thalassemia Minor|
The aim of this registry is to prospectively and retrospectively collect data on patients who are diagnosed with alpha thalassemia major and other alpha thalassemia mutations. Data collected will be used to:
- Identify patient outcomes of therapies.
- Improve clinical management of patients with ATM.
- Improve medical decision making.
- Improve quality of care.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||500 participants|
|Target Follow-Up Duration:||30 Years|
|Official Title:||International Prospective Registry of Patients With Alpha Thalassemia|
|Actual Study Start Date :||January 2017|
|Estimated Primary Completion Date :||January 2027|
|Estimated Study Completion Date :||January 2037|
- Survival to birth [ Time Frame: 6 months ]Number of fetuses diagnosed with alpha thalassemia who survive to birth, compared to number of fetuses diagnosed with alpha thalassemia who have fetal demise or are terminated in utero. This is measured in number of fetuses alive at birth divided by number of all fetuses.
- Vineland-3 Adaptive Behavior Scale [ Time Frame: 10-15 years ]Results of neurodevelopmental testing using the Vineland Adaptive Behavior Scale version 3. The Vineland-3 scoring system is based on scores for three specific adaptive behavior domains: Communication, Daily Living Skills, and Socialization. The domain scores are expressed as standard scores with a mean of 100 and standard deviation of 15.
- Gestational age at birth [ Time Frame: 6 months ]Gestational age of the child at birth. This is measured in weeks.
- Mechanical ventilation [ Time Frame: 1 year ]Duration (if any) of requiring mechanical ventilation after birth. This is measured in days.
- Length of hospitalization [ Time Frame: 6 months-1 year ]Duration of the child's hospitalization after birth. This is measured in days.
- Resolution of hydrops [ Time Frame: 6 months ]Evaluate whether receiving fetal therapy leads hydrops fetalis to resolve. This is measured by ultrasound findings.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04872179
|Contact: Billie Lianoglou, LCGC||(415) firstname.lastname@example.org|
|United States, California|
|University of California San Francisco||Recruiting|
|San Francisco, California, United States, 94143|
|Principal Investigator: Tippi C MacKenzie, MD|
|Principal Investigator:||Tippi C MacKenzie, MD||University of California, San Francisco|