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

Kamau Therapeutics

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

Sickle Cell Disease

Treatments

Genetic: nula-cel Drug Product

Study type

Interventional

Funder types

Industry

Identifiers

NCT04819841

KMAU-001-001

Details and patient eligibility

About

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.

Full description

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

Enrollment

15 estimated patients

Sex

All

Ages

12 to 40 years old

Volunteers

No Healthy Volunteers

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