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Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

This study is currently recruiting participants.
Verified September 2016 by Gamida Cell ltd
Sponsor:
ClinicalTrials.gov Identifier:
NCT02504619
First Posted: July 22, 2015
Last Update Posted: August 8, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Gamida Cell ltd
  Purpose
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 Intervention Phase
Sickle Cell Disease Thalassemia Biological: CordIn Phase 1 Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Gamida Cell ltd:

Primary Outcome Measures:
  • The acute toxicities associated with the infusion of CordIn, within 24 hours post-infusion. [ Time Frame: 24 hours ]
  • The proportion of patients with donor-derived engraftment at 42 days following transplantation [ Time Frame: 42 days ]

Secondary Outcome Measures:
  • Overall survival at 365 days after transplantation [ Time Frame: 365 days ]

Estimated Enrollment: 15
Study Start Date: May 2015
Estimated Study Completion Date: September 2018
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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
  Contacts and Locations
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


Contacts
Contact: Iddo Peleg, MSc., MBA +972-2-659-5666 clinicaltrials@gamida-cell.com

Locations
United States, California
UCSF Benioff Children's Hospital Recruiting
Oakland, California, United States, 94609
Contact: Mark Walters, MD         
Principal Investigator: Mark Walters, MD         
United States, District of Columbia
Children's National Recruiting
Washington, D.C., District of Columbia, United States, 20010
Contact: Allistair Abraham, MD    202-476-3634      
France
Hôpital Robert Debré Recruiting
Paris, France
Contact: Jean-Hugues Dalle, MD    +33 1 40 03 53 88    jhugues.dalle@gmail.com   
Principal Investigator: Jean-Hugues Dalle, MD         
Sponsors and Collaborators
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
  More Information

Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT02504619     History of Changes
Other Study ID Numbers: GC P#01.01.030
First Submitted: January 21, 2015
First Posted: July 22, 2015
Last Update Posted: August 8, 2017
Last Verified: September 2016

Additional relevant MeSH terms:
Anemia, Sickle Cell
Thalassemia
Hemoglobinopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn