Clinical Trials Logo

Clinical Trial Summary

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.


Clinical Trial Description

Alpha thalassemia major (ATM) is almost universally fatal in utero and represents an orphan disease with an unmet need for effective therapies. The only current treatment to allow the fetus to be born is to perform in utero transfusions (IUT) of red blood cells to treat the anemia and avoid the complications of hydrops and fetal demise. Often, affected pregnancies undergo elective termination after diagnosis. Cases with prenatal diagnosis of ATM who receive IUT and survive to birth will ultimately require lifelong monthly blood transfusions or bone marrow transplant, if a suitable donor is identified. This is a phase 1 clinical trial to demonstrate the safety, feasibility and efficacy of performing in utero stem cell transplantation on fetuses affected with ATM. The investigators aim to recruit ten participants with a prenatal diagnosis of ATM. Participants will undergo bone marrow harvest and an in utero transfusion combined with maternal stem cells. Transplanting maternal cells into the fetus takes advantage of existing maternal-fetal tolerance during pregnancy. Hematopoietic stem cell (HSC) transplantation into the fetus takes advantage of the developing fetal immune system to induce tolerance to the transplanted cells without using conditioning or immunosuppression. Performing stem cell transplantation at the same time as IUT minimizes any additional procedural risk to the fetus. The investigators hope to demonstrate that it is safe and feasible to perform in utero stem cell transplantation. Additionally, the investigators want to demonstrate postnatal chimerism of maternal cells so that, if a bone marrow transplant remains necessary after delivery, conditioning and immune suppression will not be required. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02986698
Study type Interventional
Source University of California, San Francisco
Contact
Status Enrolling by invitation
Phase Phase 1
Start date October 5, 2017
Completion date February 2026

See also
  Status Clinical Trial Phase
Terminated NCT00034528 - Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia Phase 2
Active, not recruiting NCT03655678 - A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia Phase 2/Phase 3
Completed NCT03609827 - Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Recruiting NCT06107400 - Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease Early Phase 1
Completed NCT00744692 - Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders Phase 1
Recruiting NCT05799118 - Study of the Role of Genetic Modifiers in Hemoglobinopathies
Active, not recruiting NCT03745287 - A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease Phase 2/Phase 3
Completed NCT00673608 - Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload Phase 4
Recruiting NCT04644016 - Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders Phase 2
Recruiting NCT04853576 - A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) Phase 1/Phase 2
Recruiting NCT05356195 - Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) Phase 3
Completed NCT03609840 - Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Active, not recruiting NCT01850108 - Non-Myeloablative Conditioning and Bone Marrow Transplantation N/A
Recruiting NCT05329649 - Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) Phase 3
Active, not recruiting NCT01966367 - CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation Phase 1/Phase 2
Completed NCT00153985 - Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies Phase 2
Recruiting NCT03128996 - Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders Phase 1/Phase 2
Completed NCT03149289 - Hepatitis C Virus Infection in Patients With Hemoglobinopathies N/A
Completed NCT00000588 - Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone Phase 2
Recruiting NCT00427661 - A Pilot Study of HSCT for Patients With High-Risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen N/A