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

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ClinicalTrials.gov Identifier: NCT01590628
Recruitment Status : Recruiting
First Posted : May 3, 2012
Last Update Posted : January 17, 2018
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
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 Hemoglobinopathies.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease & Thalassemia Drug: NiCord Phase 1 Phase 2

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 2-45 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.


Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (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 Hemoglobinopathies
Study Start Date : April 2012
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: NiCord Drug: NiCord
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells


Outcome Measures

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

Secondary Outcome Measures :
  1. Proportion of transplant-related mortality. [ Time Frame: at 100 days ]
  2. Event-free survival. [ Time Frame: at 100 days ]
  3. Overall survival. [ Time Frame: at 180 days. ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must be 2 - 45 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01590628


Contacts
Contact: Kelly Myers 972-2-6595631 clinicaltrials@gamida-cell.com

Locations
United States, New York
Steven & Alexandra Cohen Children's Medical Center, New York Completed
New York, New York, United States, 11040
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:
Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT01590628     History of Changes
Other Study ID Numbers: GC P#02.01.020
First Posted: May 3, 2012    Key Record Dates
Last Update Posted: January 17, 2018
Last Verified: January 2018

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

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