International Registry of Patients With Alpha Thalassemia

Last updated: April 2, 2025
Sponsor: University of California, San Francisco
Overall Status: Active - Recruiting

Phase

N/A

Condition

Thalassemia

Treatment

N/A

Clinical Study ID

NCT04872179
16-21157-B
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • diagnosis of alpha thalassemia (prenatal or postnatal) with genotype consistent withATM or BHFS phenotype

  • referred to the University of California, San Francisco Fetal Treatment Center forfetal diagnosis, management and/or evaluation for the ongoing in utero stem celltransplantation clinical trial

Exclusion

Exclusion Criteria:

  • none

Study Design

Total Participants: 500
Study Start date:
January 01, 2017
Estimated Completion Date:
January 31, 2037

Study Description

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:

  1. Identify patient outcomes of therapies.

  2. Improve clinical management of patients with ATM.

  3. Improve medical decision making.

  4. Improve quality of care.

Connect with a study center

  • University of California San Francisco

    San Francisco, California 94143
    United States

    Active - Recruiting

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