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Alpha Thalassemia Major - Healing Genes
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Alpha Thalassemia Major

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

A Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Alpha Thalassemia Major (ATM)


Phase 1

DESCRIPTION:

Doctors at the University of California San Francisco are seeking patients with a prenatal fetal diagnosis of ATM to trial an in-utero fetal stem cell transplant using bone marrow stem cells taken from the mother, with the goal of increased fetal survival. The following similar fetal diagnoses are eligible:

  • Alpha Thalassemia Major
  • Hemoglobinopathy; With Thalassemia
  • Hemoglobinopathies
  • Fetal Anemia
  • Fetal Hydrops
  • Alpha; Thalassemia
  • Thalassemia Major
  • Thalassemia Alpha
  • A-Thalassemia

Treatment will require a bone marrow aspiration from the mother’s iliac crest (ridge of the hip) to recover stem cells, which will be multiplied in the lab before being infused to the fetus in utero, via the umbilical vein, at least once but possibly more. The follow up will continue at least 1 year after delivery.


PATIENT MUST:

  • Be 18 week to 25 weeks (fetal age)
  • 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.
  • Willing to consent to fetal autopsy in the event of a fetal demise

THE STUDY INVOLVES:

  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Surgical recovery of bone marrow aspirate, an outpatient procedure.
  3. Infusion via umbilical vein (at least one treatment) of the recovered stem cells.

 

  1. Follow up will continue with physical assessments and questionnaires for ~12 months after delivery.

LOCATIONS AND CONTACTS:

Trials will take place at the University of California in San Francisco, CA. Map.

 

Contact: Tippi Mackenzie, MD  |  415-476-4086  |  [email protected]

Contact: Kristen Gosnell, RN  |  415-476-0445  |  [email protected]

 

 

SPONSOR INFORMATION:

University of California, San Francisco

California Institute for Regenerative Medicine (CIRM)

 

Or go online:

https://clinicaltrials.gov/ct2/show/NCT02986698

 

 

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