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Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

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ClinicalTrials.gov Identifier: NCT02504619
Recruitment Status : Terminated (lack of recruitment)
First Posted : July 22, 2015
Results First Posted : June 26, 2019
Last Update Posted : June 26, 2019
Sponsor:
Information provided by (Responsible Party):
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™.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Thalassemia Biological: CordIn Phase 1 Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Actual Study Start Date : April 4, 2016
Actual Primary Completion Date : August 2016
Actual Study Completion Date : December 2017

Resource links provided by the National Library of Medicine


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.




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02504619


Locations
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United States, California
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
United States, District of Columbia
Children's National
Washington, District of Columbia, United States, 20010
France
Hôpital Robert Debré
Paris, France
Sponsors and Collaborators
Gamida Cell ltd
Investigators
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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

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Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT02504619     History of Changes
Other Study ID Numbers: GC P#01.01.030
First Posted: July 22, 2015    Key Record Dates
Results First Posted: June 26, 2019
Last Update Posted: June 26, 2019
Last Verified: June 2019
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Thalassemia
Hemoglobinopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn