NCT02986698

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

Terminated Phase 1 Last updated 27 January 2025

What this trial tests

Phase 1 trial testing in utero hematopoietic stem cell transplantation in Alpha Thalassemia Major in 6 participants. Terminated before completion.

Timeline

5 October 2017

Primary endpoint
30 April 2024

31 December 2024

Quick facts

Lead sponsor	University of California, San Francisco
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	6
Start date	5 October 2017
Primary completion	30 April 2024
Estimated completion	31 December 2024
Sites	1 location across United States

Drugs / interventions tested

• in utero hematopoietic stem cell transplantation — full drug profile →

Conditions studied

• Alpha Thalassemia Major — all drugs for Alpha Thalassemia Major →
• Hemoglobinopathy; With Thalassemia — all drugs for Hemoglobinopathy; With Thalassemia →
• Hemoglobinopathies — all drugs for Hemoglobinopathies →
• Fetal Anemia — all drugs for Fetal Anemia →

Sponsor

University of California, San Francisco

Who can join

Adults 18 Weeks to 26 Weeks, any sex, with Alpha Thalassemia Major or Hemoglobinopathy; With Thalassemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Ionizable lipid nanoparticles for in utero mRNA delivery.
   Riley RS, Kashyap MV, Billingsley MM, White B, et al · · 2021 · cited 161× · PMID 33523869 · DOI 10.1126/sciadv.aba1028
2. Forever Connected: The Lifelong Biological Consequences of Fetomaternal and Maternofetal Microchimerism.
   Bianchi DW, Khosrotehrani K, Way SS, MacKenzie TC, et al · · 2021 · cited 39× · PMID 33417673 · DOI 10.1093/clinchem/hvaa304
3. Advances in the management of α-thalassemia major: reasons to be optimistic.
   Horvei P, MacKenzie T, Kharbanda S. · · 2021 · cited 28× · PMID 34889445 · DOI 10.1182/hematology.2021000295
4. Biomaterials in fetal surgery.
   Winkler SM, Harrison MR, Messersmith PB. · · 2019 · cited 23× · PMID 31099350 · DOI 10.1039/c9bm00177h
5. Potential new approaches to the management of the Hb Bart's hydrops fetalis syndrome: the most severe form of α-thalassemia.
   King AJ, Higgs DR. · · 2018 · cited 22× · PMID 30504332 · DOI 10.1182/asheducation-2018.1.353
6. Fetal gene therapy.
   Waddington SN, Peranteau WH, Rahim AA, Boyle AK, et al · · 2024 · cited 19× · PMID 37470194 · DOI 10.1002/jimd.12659
7. Challenges in chronic transfusion for patients with thalassemia.
   Lal A. · · 2020 · cited 19× · PMID 33275743 · DOI 10.1182/hematology.2020000102
8. The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry.
   Schwab ME, Lianoglou BR, Gano D, Gonzalez Velez J, et al · · 2023 · cited 18× · PMID 36306387 · DOI 10.1182/bloodadvances.2022007823