Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

NCT ID: NCT02504619

Last Updated: 2019-06-26

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-04-04

Study Completion Date

2017-12-31

Brief Summary

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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™.

Detailed Description

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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.

Conditions

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Sickle Cell Disease Thalassemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CordIn

Transplantation of CordIn

Group Type EXPERIMENTAL

CordIn

Intervention Type BIOLOGICAL

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

Interventions

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CordIn

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Gamida Cell ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jean-Hugues Dalle, MD

Role: PRINCIPAL_INVESTIGATOR

Robert Debre, Paris, France

Franco Locatelli, MD

Role: PRINCIPAL_INVESTIGATOR

IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy

Allistair Abraham, MD

Role: PRINCIPAL_INVESTIGATOR

Children's National, Washington DC

Locations

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UCSF Benioff Children's Hospital

Oakland, California, United States

Site Status

Children's National

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

Site Status

Hôpital Robert Debré

Paris, , France

Site Status

Countries

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United States France

Other Identifiers

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GC P#01.01.030

Identifier Type: -

Identifier Source: org_study_id

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