An observational study in Alpha-Thalassemia, Alpha Thalassemia Major and Alpha Thalassemia Minor, sponsored by University of California, San Francisco. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-09-02.
Sponsored by University of California, San Francisco · Observational
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with Alpha thalassemia.
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:
University of California, San Francisco is the lead sponsor of 2,132 studies on the registry; 375 are open to participants now.
Of its 262 completed or terminated interventional studies of FDA-regulated products, 196 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients will be either self-enrolled, or enrolled through their prenatal provider (obstetrician, genetic counselor) or postnatal provider (hematologist, pediatrician).
Exclusion Criteria:
- none
Survival to birth
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.
Time frame: 6 months
Vineland-3 Adaptive Behavior Scale
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.
Time frame: 10-15 years
Gestational age at birth
Gestational age of the child at birth. This is measured in weeks.
Time frame: 6 months
Mechanical ventilation
Duration (if any) of requiring mechanical ventilation after birth. This is measured in days.
Time frame: 1 year
Length of hospitalization
Duration of the child's hospitalization after birth. This is measured in days.
Time frame: 6 months-1 year
Resolution of hydrops
Evaluate whether receiving fetal therapy leads hydrops fetalis to resolve. This is measured by ultrasound findings.
Time frame: 6 months
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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alpha-Thalassemia
University of California, San Francisco