In Utero Stem Cell Transplant for Thalassemia

Overseen By Tippi Mackenzie, MD

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: University of California, San Francisco

Disqualifiers: Morbid obesity, Cardiac disease, Mirror syndrome, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 5

6 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Tippi Mackenzie, MD

Principal Investigator

University of California, San Francisco

Eligibility Criteria

This trial is for fetuses between 18 to 26 weeks old diagnosed with alpha-thalassemia major, showing signs of anemia or hydrops. Parents must be genetic carriers and agree to in utero treatments and a possible autopsy if the fetus doesn't survive. The mother must be healthy enough for bone marrow harvest without conditions like obesity or heart disease.

Inclusion Criteria

My bone marrow harvest is sufficient for the study's needs.

Parents must consent to fetal autopsy in the event of a fetal demise

My unborn child has alpha-thalassemia major and we've chosen in utero treatment.

Exclusion Criteria

I do not have conditions like severe obesity, heart disease, or anemia that would prevent safe surgery.

My unborn child does not have major health issues unrelated to thalassemia.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

In Utero Treatment

In utero hematopoietic stem cell transplantation performed at the time of in utero transfusion of red blood cells

Immediate procedure

1 visit (in-person)

Postnatal Follow-up

Participants are monitored for successful engraftment and safety, including chimerism and survival

1 year

Weekly visits for the first 4 weeks, then monthly visits for 1 year

Long-term Follow-up

Monitoring of long-term outcomes and stability of engraftment

5 years

Treatment Details

Interventions

• In Utero Hematopoietic Stem Cell Transplantation

Trial Overview The trial tests the safety of giving hematopoietic stem cells (cells that can form blood cells) to a fetus during an existing treatment where they get red blood cell transfusions inside the womb.

Participant Groups

Group I: in utero hematopoietic stem cell transplantation Experimental Treatment 1 Intervention

Perform in utero hematopoietic stem cell transplantation at the time of intrauterine transplantation in fetuses with alpha-thalassemia major. The cellular product is: Semi-allogeneic, Related, Maternal Bone Marrow-Derived, Miltenyi CliniMACS Plus enriched CD34+ hematopoietic stem cells administered in utero at a dose of 1 x 10\^7-10\^9 cells/kg fetal weight with equal to or less than 1% CD3+ T cells (equivalent to 10\^5-10\^7 T cells/kg fetal weight) in a final volume of 2-5ml suspended in 5% human serum albumin in Normosol buffer (Hospira, Inc.). Stem cells will be administered immediately before the red blood cells intravenously via the umbilical vein during the clinically indicated IUT. All participants will receive one dose of stem cells but may receive additional transfusions as clinically indicated.

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

California Institute for Regenerative Medicine (CIRM)

Collaborator

Trials

70

Recruited

3,300+