Recruiting

Observational Study

Sponsor:

UCLA

Code:

NCT04872179

Conditions

Alpha-Thalassemia

Alpha Thalassemia Major

Alpha Thalassemia Minor

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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.

Conditions

Alpha-Thalassemia

Alpha Thalassemia Major

Alpha Thalassemia Minor

Study ID

NCT04872179

Start date

Jan, 2017

Status verified date

Aug, 2026

Completion date

Jan, 2037

Anticipated

Primary completion date

Jan, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

500 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Survival to birth [ Time Frame: 6 months ]
  • Vineland-3 Adaptive Behavior Scale [ Time Frame: 10-15 years ]

Central Contacts and Locations

Central contacts

Billie Lianoglou, LCGC

(415) 476-2461billie.lianoglou@ucsf.edu

Locations

University of California San Francisco

Recruiting

San Francisco, California, United States, 94143

Principal Investigator:

Tippi C MacKenzie, MD

More Information

Sponsor

University of California, San Francisco

Last update posted

Sep 2, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of California, San Francisco on 2026-09-02.