Washington University in St. Louis

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

Arm	Description	Assigned 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.