Pilot Study Evaluating Safety & Efficacy of a DCBT: NiCord® & UNM CBU to SCD Patients After Myeloablative Therapy

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Gamida Cell ltd
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd
ClinicalTrials.gov Identifier:
NCT01590628
First received: April 15, 2012
Last updated: February 24, 2014
Last verified: February 2014
  Purpose

Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells and a Second, Unmanipulated CBU in Patients with Sickle Cell Disease (SCD).


Condition Intervention Phase
Sickle Cell Disease
Drug: NiCord
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by Gamida Cell ltd:

Primary Outcome Measures:
  • Safety and Tolerability will be measured by acute NiCord® infusional toxicity. [ Time Frame: 24 hours post-infusion ] [ Designated as safety issue: Yes ]
  • Assessment of cumulative incidence of donor-derived neutrophil engraftment. [ Time Frame: By Day 42 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Proportion of transplant-related mortality. [ Time Frame: at 100 days ] [ Designated as safety issue: Yes ]
  • Event-free survival. [ Time Frame: at 100 days ] [ Designated as safety issue: Yes ]
  • Overall survival. [ Time Frame: at 180 days. ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 10
Study Start Date: April 2012
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NiCord Drug: NiCord
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells

Detailed Description:

Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell transplantations (HSCT) and can be used for the treatment of various life-threatening diseases, such as hematological malignancies or genetic blood disorders, in such cases where a matched related stem cell donor is not available. However, the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU), which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality. To improve outcomes and extend applicability of UCB transplantation, one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable the broader application of UCB transplantation, and improve the clinical outcomes of UCB transplantation.

The study is designed as a pilot, single center, single arm study, evaluating the safety and efficacy of the co-transplantation of NiCord® with an unmanipulated CBU to patients with SCD following myeloablative therapy.

The total study duration is approximately 220 days, starting with the signing of an informed consent to the last visit on day 180 post-transplant. A long-term post-study follow-up is planned at 6 months post-study completion (1 year post-transplantation), and a long-term follow-up using the Center for International Blood and Marrow Transplant Research (CIBMTR) registry.

The study hypothesis is that the co-transplantation of NiCord® and an unmanipulated unrelated cord blood graft in patients with SCD following myeloablative preparative therapy consisting of hydroxyurea, busulfan, cyclophosphamide and ATG will be safe and will enable cord blood engraftment.

The main study objectives are assessment of the acute toxicity associated with the infusion of NiCord® within 24 hours post infusion, and assessment of cumulative incidence of donor-derived neutrophil engraftment by day 42 following co-transplantation of NiCord® and unmanipulated cord blood grafts. In addition, the proportion of transplant-related mortality at 100 days, event-free survival at 100 days and overall survival at 180 days will be assessed.

Ten evaluable patients recruited for the study should be 2-21 years of age, at least 10 kg in weight, have symptomatic SCD and should be considered as candidates for allogeneic myeloablative HSCT for the treatment of SCD.

  Eligibility

Ages Eligible for Study:   2 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must be 2 - 21 years of age and at least 10 kg
  • Must have clinically severe SCD (SS, SC or SBeta0 Thal) and eligible for myeloablative SCT
  • Must have two partially HLA-matched CBUs
  • Back-up autologous stem cells harvested from bone marrow
  • Adequate Karnofsky Performance score or Lansky Play-Performance scale
  • Sufficient physiological reserves
  • Signed written informed consent

Exclusion Criteria:

  • HLA-matched related donor able to donate
  • Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors
  • Prior allogeneic hematopoietic SCT within the last 12 months or reduced-intensity transplant within the past 6 months
  • Human immunodeficiency virus (HIV) infection
  • Active or uncontrolled infection
  • Pregnancy or lactation
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01590628

Contacts
Contact: David Snyder, PhD 972-2-6595666 david.snyder@gamida-cell.com

Locations
United States, New York
Steven & Alexandra Cohen Children's Medical Center, New York Recruiting
New York, New York, United States, 11040
Contact: Joel Brochstein, MD    718-470-3460    JBrochstein@NSHS.edu   
Principal Investigator: Joel Brochstein, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27705
Contact: Dr. Joanne Kurtzberg, MD    919-668-1119    joanne.kurtzberg@duke.edu   
Principal Investigator: Dr. Joanne Kurtzberg, MD         
Sponsors and Collaborators
Gamida Cell ltd
Investigators
Principal Investigator: Joanne Kurtzberg, MD Duke University Medical Center, NC, USA
Study Director: David Snyder, PhD Gamida Cell ltd
Principal Investigator: Joel Brochstein, MD Steven & Alexandra Cohen Children's Medical Center, New York
  More Information

Additional Information:
No publications provided

Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT01590628     History of Changes
Other Study ID Numbers: GC P#02.01.020
Study First Received: April 15, 2012
Last Updated: February 24, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Gamida Cell ltd:
Sickle Cell Disease
Genetic disorder

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

ClinicalTrials.gov processed this record on September 18, 2014