International Registry of Patients With Alpha Thalassemia (ATM Registry)

University of California San Francisco (UCSF)

Status: Enrolling

Conditions: Alpha Thalassemia Major; Alpha Thalassemia Minor; Alpha-Thalassemia

Study type: Observational

Funder types: Other

Identifiers

NCT04872179

16-21157-B

Details and patient eligibility

About

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.

Full 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.

Enrollment: 500 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design