RECRUITING

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

Talk to us

Conditions

Sickle Cell Diseasesickle cell anemiagene correctiongene therapyCRISPR

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2021-11-15
Study Completion:2027-07-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04819841

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.

Ages Eligible for Study:12 Years to 40 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * ≥12 to ≤ 40 years
  2. * Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures:
  3. * recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
  4. * ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
  5. * Lansky/Karnofsky performance status of ≥ 80
  1. * Available 10/10 HLA-matched sibling donor
  2. * Prior HSCT or gene therapy
  3. * Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
  4. * Clinically significant and active bacterial, viral, fungal or parasitic infection
  5. * Pregnancy or breastfeeding in a postpartum female
  6. * Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment

Contacts and Locations

Study Contact

Restore Clinical Study Support
CONTACT
650-416-6562
RestoreStudySupport@kamautx.com

Principal Investigator

Mathew Porteus, MD, PhD
STUDY_DIRECTOR
Kamau Therapeutics

Study Locations (Sites)

Lucile Packard Children's Hospital
Palo Alto, California, 94304
United States
Washington University
St. Louis, Missouri, 63110
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States

Collaborators and Investigators

Sponsor: Kamau Therapeutics

  • Mathew Porteus, MD, PhD, STUDY_DIRECTOR, Kamau Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-11-15
Study Completion Date2027-07-31

Study Record Updates

Study Start Date2021-11-15
Study Completion Date2027-07-31

Terms related to this study

Keywords Provided by Researchers

  • sickle cell disease
  • sickle cell anemia
  • gene correction
  • gene therapy
  • CRISPR

Additional Relevant MeSH Terms

  • Sickle Cell Disease