Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

Phase 1

Terminated

• Conditions: Thalassemia; Sickle Cell Disease
• Interventions: Biological: CordIn

• Registration Number: NCT02504619
• Lead Sponsor: Gamida Cell ltd

Brief Summary

CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.

Detailed Description

CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.

The trial ends when the last patient completes their last visit. The overall study objectives are to evaluate the safety and efficacy of CordIn™: single ex-vivo expanded cord blood unit transplantation in patients with hemoglobinopathies (sickle cell disease or thalassemia major) following a preparative therapy.

Study Timeline

• Study First Submitted: 2015-01-21
• Study First Submitted QC: 2015-07-20
• Study First Posted: 2015-07-22
• Study Start: 2016-04-04
• Primary Completion: 2016-08
• Study Completion: 2017-12
• Results First Submitted: 2019-04-16
• Status Verified: 2019-06
• Results First Submitted QC: 2019-06-03
• Last Update Submitted: 2019-06-03
• Results First Posted: 2019-06-26
• Last Update Posted: 2019-06-26

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 1

Inclusion Criteria

• Patient is a candidate for allogeneic SCT for treatment of SCD or thalassemia
• Patients must have one partially HLA-matched CBUs
• Adequate Karnofsky Performance score or Lansky Play-Performance scale
• Sufficient physiological reserves
• Signed written informed consent

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Exclusion Criteria

• Prior allogeneic HSCT
• Evidence of HIV infection or HIV positive serology
• Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
• Active or uncontrolled infection
• Pregnancy or lactation

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CordIn	CordIn	Transplantation of CordIn

Primary Outcome Measures

Name	Time	Method
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation	42 days	One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.	24 hours	The number of patients with grade 4 or 5 toxicity were estimated together with 95% confidence limits based on the binomial distribution. The proportion with toxicity grades 1, 2, and 3 was also estimated.

Secondary Outcome Measures

Name	Time	Method
Percentage of Overall Survival at 365 Days After Transplantation	365 days	The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method.

Trial Locations

Locations (3)

UCSF Benioff Children's Hospital; 🇺🇸 Oakland, California, United States

Children's National; 🇺🇸 Washington, District of Columbia, United States

Hôpital Robert Debré; 🇫🇷 Paris, France