Can a global registry map the path to better outcomes for alpha thalassemia?
NCT ID NCT04872179
First seen Sep 02, 2026 · Last updated Sep 03, 2026 · Updated 1 time
Summary
This international registry follows people with alpha thalassemia, a genetic blood disorder, to learn how the disease progresses and how well fetal treatments work. Researchers will track survival to birth, developmental milestones, and health after birth. The goal is to improve prenatal care and counseling for families facing this condition.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- This registry could improve prenatal counseling and management for alpha thalassemia, potentially increasing survival and better preparing families for outcomes.
- What could go wrong
- As a registry, it does not test a treatment, so it cannot prove what works. Data depends on voluntary participation and may not represent all patients.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 500 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2017
- Expected to finish
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Jan 2037
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Patients will be either self-enrolled, or enrolled through their prenatal provider (obstetrician, genetic counselor) or postnatal provider (hematologist, pediatrician).
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * diagnosis of alpha thalassemia (prenatal or postnatal) with genotype consistent with ATM or BHFS phenotype * referred to the University of California, San Francisco Fetal Treatment Center for fetal diagnosis, management and/or evaluation for the ongoing in utero stem cell transplantation clinical trial Exclusion Criteria: \- none
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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University of California San Francisco
RECRUITINGSan Francisco, California, 94143, United States
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