NCT02986698TerminatedPhase 1
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
A Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Alpha Thalassemia Major
Source: ClinicalTrials.gov · Last updated 27 Jan 2025
Brief summary (registry, in English)
The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
- Status (registry)
- Terminated
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 6 (actual)
- First submitted
- 5 Dec 2016
- Start date
- 5 Oct 2017
- Primary completion date
- 30 Apr 2024
- Lead sponsor
- University of California, San Francisco
- Diseases
- Thalassaemia
- NCT number
- NCT02986698
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Maternal Subject Exclusion Criteria: Maternal participants will be excluded if they have one or more morbidities that would preclude bone marrow harvest and fetal intervention including, but not limited to, morbid obesity with BMI > 35, maternal cardiac disease, mirror syndrome, symptomatic maternal anemia, or if they develop preterm premature rupture of membranes (PPROM) or active preterm labor (PTL).
Other
- Fetal Subject Exclusion Criteria: Fetal participants will be excluded if they have a second major anatomic anomaly (not related to the underlying thalassemia) that contributes a significant morbidity or mortality risk, or echocardiogram or ultrasound findings that indicate a high risk of fetal demise after fetal intervention.
Who the study is looking for
- Male or female fetuses from 18 weeks and 0/7 days to 26 weeks 0/7 days gestation with a diagnosis of alpha-thalassemia major by chorionic villus sampling (CVS), amniocentesis, cordocentesis or by identification of parents as genetic carriers, and identification of fetal anemia or signs of impending hydrops, for whom parents elect to pursue in utero transfusion, and are willing to undergo subsequent IUT for the remainder of gestation.
- parents must consent to fetal autopsy in the event of a fetal demise
- adequate bone marrow harvest from maternal participant is a condition for inclusion
Age in the registry: 18 weeks to 26 weeks · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of California Coordinator of this site? Confirm its status | San Francisco | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.