Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02504619
• Other study ID #: GC P#01.01.030
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: April 4, 2016
• Est. completion date: December 2017

Study information

• Verified date: June 2019
• Source: Gamida Cell ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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™.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: December 2017
• Est. primary completion date: August 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 25 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Hemoglobinopathies
• Sickle Cell Disease
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
France	Hôpital Robert Debré	Paris
United States	UCSF Benioff Children's Hospital	Oakland	California
United States	Children's National	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Gamida Cell ltd

Countries where clinical trial is conducted

United States,  France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Percentage of Overall Survival at 365 Days After Transplantation	The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method.	365 days