Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

NCT02504619 | Terminated Phase 1 Results DMC

• 1 other identifier: GC P#01.01.030
• Study Type: interventional
• Target: 1
• Locations: 2 countries
• Sites: 3

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™.

Conditions

Sickle Cell Disease; Thalassemia

Outcome Measures

Primary Outcomes (2)

• Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.

  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

  One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.

  42 days

Secondary Outcomes (1)

• Percentage of Overall Survival at 365 Days After Transplantation

  365 days

Study Arms (1)

CordIn

EXPERIMENTAL

Transplantation of CordIn

Biological: CordIn

Interventions

CordIn BIOLOGICAL

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

CordIn

Eligibility Criteria

• Age: 2 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Gamida Cell ltd: lead

Study Sites (3)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

Children's National

Washington D.C., District of Columbia, 20010, United States

Location

Hôpital Robert Debré

Paris, France

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Kelly Myers
• Organization: Gamida Cell

kelly@gamida-cell.com

026595631

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2015
• First Posted: July 22, 2015
• Study Start: April 4, 2016
• Primary Completion: August 1, 2016
• Study Completion: December 1, 2017
• Last Updated: June 26, 2019
• Results First Posted: June 26, 2019

Record last verified: 2019-06

Locations

US (2); FR (1)