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

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.

Condition

  • Sickle Cell Disease

Eligibility

Eligible Ages
Between 12 Years and 40 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
nula-cel Drug Product 		nula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.
  • Genetic: nula-cel Drug Product
    nula-cel is administered via IV infusion following a myeloablative conditioning regimen

Recruiting Locations

Washington University in St. Louis and nearby locations

Washington University
St Louis 4407066, Missouri 4398678 63110
Contact:
Maggie Nash
314-273-5936
nashm@wustl.edu

More Details

NCT ID
NCT04819841
Status
Recruiting
Sponsor
Kamau Therapeutics

Study Contact

Restore Clinical Study Support
650-442-2283
RestoreStudySupport@kamautx.com

Detailed Description

Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting.