← Back to Search

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

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

Study 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.Check my eligibility

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.See study design

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

Select...

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

Select...

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

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Maternal participant tolerance of bone marrow harvest

Blood Transfusion

Secondary outcome measures

Adequate bone marrow harvest from the maternal participant

successful engraftment

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 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of California, San FranciscoLead Sponsor

2,500 Previous Clinical Trials

15,236,461 Total Patients Enrolled

California Institute for Regenerative Medicine (CIRM)OTHER

63 Previous Clinical Trials

3,138 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.

Media Library

Alpha-Thalassemia Research Study Groups: in utero hematopoietic stem cell transplantation

Alpha-Thalassemia Clinical Trial 2023: In Utero Hematopoietic Stem Cell Transplantation Highlights & Side Effects. Trial Name: NCT02986698 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any ongoing opportunities to participate in this investigation?

"Per the data on clinicaltrials.gov, this particular medical trial is not presently recruiting patients. It was initially listed on October 5th 2017 and last updated on October 28th 2022; yet despite its inactivity there are currently 76 other research projects enrolling candidates."

Answered by AI

Does the eligibility criterion for this research encompass people aged under 35?

"This clinical trial has been designed with a lower age limit of 18 weeks and an upper bound of 26 weeks."

Answered by AI

Is it possible for me to join this clinical research study?

"To qualify for this research project, participants must have hemoglobinopathies and fall into the 18-26 week age bracket. A total of 10 individuals will be accepted to participate in the study."

Answered by AI

Is there a high degree of security involved with in utero hematopoietic stem cell transplantation?

"Our internal assessment at Power has assigned in utero hematopoietic stem cell transplantation a safety rating of 1, as it is currently undergoing Phase 1 testing with limited evidence for its efficacy and safety."

Answered by AI

Recent research and studies

ChimerismJournal

The Maternal Immune Response Inhibits the Success of in Utero Hematopoietic Cell Transplantation

Nijagal, Amar, Marta Wegorzewska, Tom Le, Qizhi Tang, and Tippi C. MacKenzie. 2011. “The Maternal Immune Response Inhibits the Success of in Utero Hematopoietic Cell Transplantation”. Chimerism. Informa UK Limited. doi:10.4161/chim.2.2.16287.

Seminars in Pediatric SurgeryJournal

Clinical Implications of Maternal-fetal Cellular Trafficking

Nijagal, Amar, and Tippi C. MacKenzie. 2013. “Clinical Implications of Maternal-fetal Cellular Trafficking”. Seminars in Pediatric Surgery. Elsevier BV. doi:10.1053/j.sempedsurg.2012.10.011.

in UteroJournal

Favorable Outcomes After<i>in Utero</i>transfusion in Fetuses with Alpha Thalassemia Major: A Case Series and Review of the Literature

Kreger, Emily M., Sylvia T. Singer, Russell G. Witt, Nancy Sweeters, Billie Lianoglou, Ashutosh Lal, Tippi C. Mackenzie, and Elliott Vichinsky. 2016. “Favorable Outcomes Afterin Uterotransfusion in Fetuses with Alpha Thalassemia Major: A Case Series and Review of the Literature”. Prenatal Diagnosis. Wiley. doi:10.1002/pd.4966.

Current Opinion in PediatricsJournal