International Registry of Patients With Alpha Thalassemia

Alpha-Thalassemia Clinical Trial

— ATM Registry  

Official title:

International Prospective Registry of Patients With Alpha Thalassemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04872179
• Other study ID #: 16-21157-B
• Status: Recruiting
• Start date: January 2017
• Est. completion date: January 2037

Study information

• Verified date: December 2023
• Source: University of California, San Francisco
• Contact: Billie Lianoglou, LCGC
• Phone: (415) 476-2461
• Email: billie.lianoglou[@]ucsf.edu
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: January 2037
• Est. primary completion date: January 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Alpha Thalassemia Major
• Alpha-Thalassemia
• beta-Thalassemia
• Thalassemia

Locations

Country	Name	City	State
United States	University of California San Francisco	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
University of California, San Francisco

Country where clinical trial is conducted

United States, 

References & Publications (1)

Kreger EM, Singer ST, Witt RG, Sweeters N, Lianoglou B, Lal A, Mackenzie TC, Vichinsky E. Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature. Prenat Diagn. 2016 Dec;36(13):1242-1249. doi: 10.1002/pd.4966. Epub 2016 Dec 7. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Survival to birth	Number of fetuses diagnosed with alpha thalassemia who survive to birth, compared to number of fetuses diagnosed with alpha thalassemia who have fetal demise or are terminated in utero. This is measured in number of fetuses alive at birth divided by number of all fetuses.	6 months
Primary	Vineland-3 Adaptive Behavior Scale	Results of neurodevelopmental testing using the Vineland Adaptive Behavior Scale version 3. The Vineland-3 scoring system is based on scores for three specific adaptive behavior domains: Communication, Daily Living Skills, and Socialization. The domain scores are expressed as standard scores with a mean of 100 and standard deviation of 15.	10-15 years
Secondary	Gestational age at birth	Gestational age of the child at birth. This is measured in weeks.	6 months
Secondary	Mechanical ventilation	Duration (if any) of requiring mechanical ventilation after birth. This is measured in days.	1 year
Secondary	Length of hospitalization	Duration of the child's hospitalization after birth. This is measured in days.	6 months-1 year
Secondary	Resolution of hydrops	Evaluate whether receiving fetal therapy leads hydrops fetalis to resolve. This is measured by ultrasound findings.	6 months