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

Launched by KAMAU THERAPEUTICS · Mar 25, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called nula-cel for people with severe Sickle Cell Disease (SCD). The main goal is to see how safe this treatment is and to gather early information on how well it works. The trial will involve about 15 participants between the ages of 12 and 40 who have experienced serious complications from their SCD, like frequent painful crises or acute chest syndrome, despite receiving standard care.

To be eligible for the study, participants need to have severe SCD and cannot have a fully matched donor available or a history of certain previous treatments. During the trial, participants will receive the nula-cel treatment and will be monitored closely for any side effects or changes in their condition. This is an important step in developing new therapies for Sickle Cell Disease, and participants will be contributing to valuable research that may help others in the future.

Gender

ALL

Eligibility criteria

• 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