Phase 2 N=16 Treatment

Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

Sickle Cell Disease & Thalassemia

Bottom Line

View on ClinicalTrials.gov: NCT01590628 ↗

Enrolled (actual)

Serious AEs

100.0%

Results posted

Apr 2022

Primary outcome: Primary: Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. — 0; 0; 3; 0 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: NiCord (Drug)
Age: Pediatric, Adult · 2+ yrs
Sex: All
Sponsor: Gamida Cell ltd
Primary completion: Sep 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity.	0; 0; 3; 0	—
PRIMARY Assessment of Cumulative Incidence of Donor-derived Neutrophil Engraftment.	13; 3	—
SECONDARY Proportion of Transplant-related Mortality.	0; 0	—
SECONDARY Event-free Survival	92.3; 66.6	—
SECONDARY Overall Survival	92.3; 100	—

Summary

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies

Eligibility Criteria

Inclusion Criteria

Must be 2 - 45 years of age and at least 10 kg
Must have clinically severe SCD (SS, SC or SBeta0 Thal) or thalassemia major and be eligible for myeloablative SCT
Must have two partially HLA-matched CBUs for part 1; and one partially HLA-matched CBU for part 2
Back-up autologous stem cells harvested from bone marrow
Adequate Karnofsky Performance score or Lansky Play-Performance scale
Sufficient physiological reserves
Signed written informed consent

Exclusion Criteria

HLA-matched related donor able to donate
Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors
Prior allogeneic hematopoietic SCT within the last 12 months or reduced-intensity transplant within the past 6 months
Human immunodeficiency virus (HIV) infection
Active or uncontrolled infection
Pregnancy or lactation

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01590628). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.