Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Description

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.

Conditions

Sickle Cell Disease

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Study Overview

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Study Details

Study overview

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.

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

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Condition
Sickle Cell Disease
Intervention / Treatment

-

Contacts and Locations

Palo Alto

Lucile Packard Children's Hospital, Palo Alto, California, United States, 94304

St. Louis

Washington University, St. Louis, Missouri, United States, 63110

Columbus

Nationwide Children's Hospital, Columbus, Ohio, United States, 43205

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

12 Years to 40 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Kamau Therapeutics,

Mathew Porteus, MD, PhD, STUDY_DIRECTOR, Kamau Therapeutics

Study Record Dates

2027-07-31