International Registry of Patients With Alpha Thalassemia

a study on Alpha Thalassemia Alpha Thalassemia Major Beta Thalassemia

Summary

Location
at San Francisco, California
Dates
study started
completion around
Principal Investigator
by Tippi C MacKenzie, MD
Headshot of Tippi C MacKenzie
Tippi C MacKenzie

Description

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.

Official Title

International Prospective Registry of Patients With Alpha Thalassemia

Details

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.

Keywords

Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor, Thalassemia, beta-Thalassemia

Eligibility

You can join if…

  • 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

You CAN'T join if...

  • none

Location

  • UCSF accepting new patients
    San Francisco California 94143 United States

Lead Scientist at UCSF

  • Tippi C MacKenzie, MD
    Tippi MacKenzie is a pediatric and fetal surgeon who is focused on developing better ways to diagnose and treat genetic diseases before birth. She leads a translational research lab examining the unique biology between the mother and her fetus, with the idea that pregnancy complications such as preterm labor arise from a breakdown in maternal-fetal tolerance.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
University of California, San Francisco
ID
NCT04872179
Study Type
Observational [Patient Registry]
Participants
Expecting 500 study participants
Last Updated
Contact us about this trial