In-utero stem cell transplantation for fetuses with Bart's Hydrops Fetalis Syndrome
Feasibility of In-utero Hematopoietic Stem Cell Transplantation as a Conjunctive Treatment During In-utero Blood Transfusion for Hemoglobin Bart's Hydrops Fetalis Syndrome in Hong Kong
This study is testing if giving stem cells to unborn babies with Bart's Hydrops Fetalis Syndrome can help improve their health before they are born.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 18 Years and up |
| Sex | Female |
| Sponsor | Chinese University of Hong Kong Academic / other |
| Locations | 1 site (Shatin) |
| Trial ID | NCT05797272 on ClinicalTrials.gov |
What this trial studies
This pilot study invites pregnant women diagnosed with Bart's Hydrops Fetalis Syndrome (BHFS) to consider in-utero hematopoietic stem cell transplantation (HSCT) as a treatment option. The study aims to assess the feasibility of performing HSCT on fetuses diagnosed with BHFS during in-utero transfusion of red blood cells. Participants will undergo harvesting of bone marrow or peripheral blood stem cells, followed by an in-utero transfusion combined with maternal stem cells. The goal is to evaluate the potential for this intervention to improve outcomes for affected fetuses.
Who should consider this trial
Good fit: Ideal candidates are pregnant women under 26 weeks of gestation with a confirmed diagnosis of BHFS who choose to continue their pregnancy.
Not a fit: Patients with fetuses that have additional major anatomical anomalies or genetic abnormalities unrelated to BHFS may not benefit from this intervention.
Why it matters
Potential benefit: If successful, this approach could significantly improve survival rates and health outcomes for fetuses diagnosed with BHFS.
How similar studies have performed: While this approach is novel, other studies have explored in-utero interventions for similar conditions, but the specific application of HSCT for BHFS is largely untested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Pregnancy before 26 weeks and 0 day of gestational age with a diagnosis of BHFS confirmed by chorionic villus sampling, amniocentesis or cordocentesis; * The parents elected to pursue IUT and are willing to undergo subsequent IUT for the remainder of gestation. Exclusion Criteria: A) Fetal subjects: * Fetuses having a second major anatomic anomaly (not related to the underlying thalassemia) that contributes a significant morbidity or mortality risk; * Fetuses having a genetic or chromosomal abnormalities other than BHFS that contributes a significant morbidity or mortality risk; * Echocardiogram or ultrasound findings that indicate a high risk of fetal demise after fetal intervention; * Fetuses diagnosed with in-utero death prior to the actual intervention. B) Maternal subjects: * Maternal age \< 18 years, mentally handicapped or severely ill; * Maternal participants having one or more morbidities that would preclude bone marrow or peripheral blood stem cells harvest and fetal intervention including, but not limited to, bleeding disorder, maternal cardiac disease, maternal mirror syndrome, symptomatic maternal anemia, or if they develop preterm premature rupture of membranes or active preterm labor; * Unable to understand English or Chinese to give consent.
Where this trial is running
Shatin
- The Chinese University of Hong Kong — Shatin, Hong Kong (Recruiting)
Study contacts
- Principal investigator: Tak Yeung LEUNG, MD — Chinese University of Hong Kong
- Study coordinator: Tak Yeung LEUNG, MD
- Email: tyleung@cuhk.edu.hk
- Phone: 852-35052806
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.