Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01590628|
Recruitment Status : Active, not recruiting
First Posted : May 3, 2012
Last Update Posted : June 4, 2019
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease & Thalassemia||Drug: NiCord||Phase 1 Phase 2|
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.
In Part 1 of this study, NiCord® will be administered to the patient in conjunction with a second, unmanipulated CBU. In Part 2 of this study, NiCord® will be administered to the patient without a second, unmanipulated CBU. The study duration per patient is approximately 270 days from signing of informed consent to last visit on day 180 post-transplant.
The overall study objectives of part 1 of this study are to evaluate the safety and efficacy of co-transplantation of NiCord® and an unmanipulated CBU in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy. The overall study objectives of part 2 of this study are to evaluate the safety and efficacy of transplantation of NiCord® in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy.
The study hypothesis for part 1 of this study is that the co-transplantation of NiCord® and an unmanipulated unrelated cord blood graft in patients with hemoglobinopathies (SCD, or thalassemia major) following myeloablative preparative therapy will be safe and will enable cord blood engraftment. The study hypothesis for part 2 of this study is that transplantation of NiCord® in patients with hemoglobinopathies (SCD, or thalassemia major) following myeloablative preparative therapy will be safe and will enable cord blood engraftment.
Up to fifteen (15) evaluable patients recruited for part 1 of the study and up to five (5) patients for part 2 of the study should be 2-45 years of age, at least 10 kg in weight, have symptomatic SCD or thalassemia major and should be considered as candidates for allogeneic myeloablative HSCT for the treatment of SCD.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies|
|Study Start Date :||April 2012|
|Estimated Primary Completion Date :||September 2019|
|Estimated Study Completion Date :||January 2020|
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells
- Safety and Tolerability will be measured by acute NiCord® infusional toxicity. [ Time Frame: 24 hours post-infusion ]
- Assessment of cumulative incidence of donor-derived neutrophil engraftment. [ Time Frame: By Day 42 ]
- Proportion of transplant-related mortality. [ Time Frame: at 100 days ]
- Event-free survival. [ Time Frame: at 100 days ]
- Overall survival. [ Time Frame: at 180 days. ]
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
|United States, New York|
|Steven & Alexandra Cohen Children's Medical Center, New York|
|New York, New York, United States, 11040|
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27705|
|United States, Texas|
|The University Of Texas M. D. Anderson Cancer Center|
|Houston, Texas, United States, 77030-4009|
|Principal Investigator:||Joanne Kurtzberg, MD||Duke University Medical Center, NC, USA|
|Principal Investigator:||Joel Brochstein, MD||Steven & Alexandra Cohen Children's Medical Center, New York|