Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

June 3, 2019 updated by: Gamida Cell ltd

Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

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™.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France
        • Hôpital Robert Debré
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National

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.

Eligibility Criteria

Ages Eligible for Study

2 years to 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CordIn
Transplantation of CordIn
Biological: CordIn
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.
Time Frame: 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.
24 hours
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
Time Frame: 42 days
One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
42 days

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gamida Cell ltd

Investigators

  • Principal Investigator: Jean-Hugues Dalle, MD, Robert Debre, Paris, France
  • Principal Investigator: Franco Locatelli, MD, IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy
  • Principal Investigator: Allistair Abraham, MD, Children's National, Washington DC

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 Major Dates

Study Start (Actual)

April 4, 2016

Primary Completion (Actual)

August 1, 2016

Study Completion (Actual)

December 1, 2017

Study Registration Dates

First Submitted

January 21, 2015

First Submitted That Met QC Criteria

July 20, 2015

First Posted (Estimate)

July 22, 2015

Study Record Updates

Last Update Posted (Actual)

June 26, 2019

Last Update Submitted That Met QC Criteria

June 3, 2019

Last Verified

June 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GC P#01.01.030

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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