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Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Recruiting
I'm InterestedTrial ID: NCT04819841
Purpose
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Official Title
A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
Stanford Investigator(s)
May Chien
Clinical Assistant Professor, Medicine - Hematology Clinical Assistant Professor, Pediatrics - Hematology & Oncology
Robert Lowsky
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Eligibility
Inclusion Criteria:
* ≥12 to ≤ 40 years
* Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures:
* recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
* ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
* Lansky/Karnofsky performance status of ≥ 80
Exclusion Criteria:
* Available 10/10 HLA-matched sibling donor
* Prior HSCT or gene therapy
* Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
* Clinically significant and active bacterial, viral, fungal or parasitic infection
* Pregnancy or breastfeeding in a postpartum female
* Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment
Intervention(s):
genetic: nula-cel Drug Product
Recruiting
I'm InterestedContact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Elisabeth Merkel, RN