Trials Sponsors Conditions Drugs Pricing

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

CordInBiological

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

CordIn

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age2 Years - 25 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (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

Sponsors & Collaborators

Gamida Cell ltd
Industrylead

Study Sites (3)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Children's National

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

Hôpital Robert Debré

Paris, France

MeSH Terms

Conditions

Anemia, Sickle CellThalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title: Kelly Myers
Organization: Gamida Cell

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

Enrollment

1 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1 2
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

Actual (reported)

Primary Completion

Study Stopped

Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (1)

Study Arms (1)

CordIn

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations

Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (1)

Study Arms (1)

CordIn

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations