Date: 2013-02-14
Type of information: Results
phase: 1-2
Announcement: results
Company: Gamida Cell (Israel)
Product: NiCord® (expanded cell graft derived from an entire unit of umbilical cord blood enriched with stem cells)
Action
mechanism: cell therapy
Disease: sickle cell disease
Therapeutic area: Genetic diseases - Blood diseases
Country: USA
Trial
details: The official name of the study is 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. (NCT01590628)
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 cord blood unit (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.
Latest
news: * On February 14, 2013, Gamida Cell announced the successful results of the Phase I/II study of its second pipeline product NiCord, umbilical cord derived stem cells expanded using the company’s proprietary NAM technology. Study data was announced during an oral presentation titled "Nicord® Expanded Hematopoietic Progenitor Cells (HPC) Are Capable of Outcompeting the Unmanipulated (UM) Cord Blood Unit and of Prolonged Myeloid and Lymphoid Engraftment Following Myeloablative Dual Umbilical Cord Blood (UCB) Transplantation". The presentation was delivered at the prestigious 2013 BMT Tandem Meetings in Salt Lake City by Dr. Mitchell E. Horwitz, associate professor of medicine at Duke Medicine and a principal investigator of the NiCord study for hematological malignancies (HM).
Eleven patients, ages 21-61, with high-risk hematological malignancies received NiCord and an un-manipulated graft of umbilical cord blood. Eight patients engrafted with NiCord. The median time to neutrophil engraftment was 10.5 (7-18) days for those engrafting with NiCord. Two patients engrafted with the un-manipulated UCB and one patient experienced primary graft failure. There were no cases of Grade III/IV acute GvHD. No safety concerns surrounding the use of NiCord were raised. With a median follow-up of 8 months, the progression-free and overall survival are both 90%.
Dr. Horwitz said, “The results from this early study suggest that NiCord expanded hematopoietic progenitor cells (HPCs) reduce the time to hematopoietic recovery and are capable of long term (>22 months) neutrophil and T-cell engraftment. Co-transplantation of NiCord and an un-manipulated cord blood unit is feasible and safe. Further studies are planned to confirm that the NiCord product can be transplanted as a single expanded unit, without co-infusion of un-manipulated cells. This would mean that only one cord blood unit would be used for transplantation, rather than two units, as are used today in most adult umbilical cord blood transplantations.”
Gamida Cell is now continuing to develop NiCord for other indications including sickle cell disease, thalassemia, severe autoimmune diseases and genetic metabolic diseases. A phase I/II study is currently enrolling pediatric patients with sickle cell disease at Duke Medicine and at the Steven and Alexandra Cohen Children's Medical Center of New York.
* On November 12, 2012, Gamida Cell, a leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine, has announced that the first patient, enrolled in its pilot study of NiCord® as an investigational treatment for sickle cell disease (SCD), has been transplanted at Duke University in North Carolina.
* On September 19, 2012, Gamida Cell has announced that the company is also actively enrolling for a Phase I/II study of NiCord® as an experimental treatment for sickle cell disease.
