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International Registry for Alpha Thalassemia

This study is an international registry for people with alpha thalassemia, including alpha thalassemia major and minor. It aims to gather information about how the disease progresses naturally and how different treatments given before birth (fetal therapies) affect patients. The goal is to improve how alpha thalassemia is managed before birth. You can join if you have an alpha thalassemia diagnosis (before or after birth) with a specific genetic makeup (genotype) and have been referred to the University of California, San Francisco Fetal Treatment Center for diagnosis, management, or evaluation for an ongoing stem cell transplant trial. The study will look at how long patients survive after birth (measured at 6 months) and their adaptive behaviors (measured at 10-15 years). The current recruitment status is unclear.

Study design
This is an observational study, meaning researchers will collect information without giving any specific intervention. It plans to include 500 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed to see survival at 6 months after birth and adaptive behavior between 10-15 years of age.

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NCT04872179

International Registry of Patients With Alpha Thalassemia

Recruiting
Not specifiedAll AgesObservational
University of California, San Francisco
~500 participants
Updated 2025-04-04 on ClinicalTrials.gov

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Survival to birth
Measured over 6 months
+1 more outcome measured

Conditions

Alpha-Thalassemia
Alpha Thalassemia Major
Alpha Thalassemia Minor

Explore related trials

Alpha ThalassemiaHemoglobin Subunit Alpha

Where it's being run

1 sites across 1 states
California1

Study leadership

  • Tippi C MacKenzie, MD · PRINCIPAL_INVESTIGATOR · University of California, San Francisco

Who to contact

Billie Lianoglou, LCGC
[email protected]
(415) 476-2461
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Eligibility criteria

Inclusion

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

What this trial measures

  • Survival to birth6 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 Scale10-15 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.