International registry for patients with Alpha Thalassemia
International Prospective Registry of Patients With Alpha Thalassemia
University of California, San Francisco · NCT04872179
This study is collecting information from people with alpha thalassemia to see how the condition affects them and how different treatments work, with the goal of improving care for patients.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Sex | All |
| Sponsor | University of California, San Francisco (other) |
| Locations | 1 site (San Francisco, California) |
| Trial ID | NCT04872179 on ClinicalTrials.gov |
What this trial studies
This registry aims to collect both prospective and retrospective data on patients diagnosed with alpha thalassemia, including alpha thalassemia major and other mutations. The focus is on understanding the natural history of the disease and evaluating the outcomes of fetal therapies. By gathering this information, the registry seeks to enhance clinical management, improve medical decision-making, and ultimately elevate the quality of care for patients with alpha thalassemia.
Who should consider this trial
Good fit: Ideal candidates for this registry include individuals diagnosed with alpha thalassemia who are referred for evaluation at the University of California, San Francisco Fetal Treatment Center.
Not a fit: Patients without a diagnosis of alpha thalassemia or those not referred to the specified treatment center may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could lead to improved prenatal management and treatment outcomes for patients with alpha thalassemia.
How similar studies have performed: Other studies focusing on registries for genetic conditions have shown success in improving patient outcomes and management strategies.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
San Francisco, California
- University of California San Francisco — San Francisco, California, United States (RECRUITING)
Study contacts
- Principal investigator: Tippi C MacKenzie, MD — University of California, San Francisco
- Study coordinator: Billie Lianoglou, LCGC
- Email: billie.lianoglou@ucsf.edu
- Phone: (415) 476-2461
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor