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Stem Cell Transplantation

In Utero Stem Cell Transplant for Thalassemia

Phase 1

Waitlist Available

Led By Tippi Mackenzie, MD

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Adequate bone marrow harvest from maternal participant is a condition for inclusion

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

Must not have

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)

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 year recruitment phase to include time of bone marrow harvest through 30 days after delivery

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether it is safe to give hematopoietic stem cells to fetuses with alpha-thalassemia major during in utero transfusions of red blood cells.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not explicitly listed, but risks may include complications from in utero procedures such as infection or preterm labor, and issues related to bone marrow harvesting from the mother.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 year recruitment phase to include time of bone marrow harvest through 30 days after delivery

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 year recruitment phase to include time of bone marrow harvest through 30 days after delivery

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 year recruitment phase to include time of bone marrow harvest through 30 days after delivery for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Maternal participant tolerance of bone marrow harvest

Blood Transfusion

Secondary study objectives

Adequate bone marrow harvest from the maternal participant

successful engraftment

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: in utero hematopoietic stem cell transplantationExperimental Treatment1 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.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of California, San FranciscoLead Sponsor

2,593 Previous Clinical Trials

14,887,940 Total Patients Enrolled

California Institute for Regenerative Medicine (CIRM)OTHER

69 Previous Clinical Trials

3,346 Total Patients Enrolled

Tippi Mackenzie, MD3.73 ReviewsPrincipal Investigator - University of California, San Francisco

University of California, San Francisco

5Patient Review

Dr. Mckenzie is an incredible physician. She performed my daughter's Nissen surgery with the utmost professionalism and expertise. My daughter's quality of life has improved dramatically since the surgery, and I couldn't be more pleased. I would highly recommend her to anyone seeking a qualified doctor.