Date: 2014-07-29
Type of
information: Clinical trial authorisation
phase: 1b
Announcement: clinical trial authorization
Company: Fate Therapeutics (USA - CA)
Product: PROHEMA® (16, 16-dimethyl prostaglandin E2, or dmPGE2, modulated cord blood)
Action
mechanism:
- stem cell therapy. PROHEMA® is a pharmacologically-modulated, cord blood-derived hematopoietic stem cell (HSC) therapeutic. Ir is produced through a proprietary, two-hour, ex vivo cell modulation process that results in rapid activation of key biological pathways involved in homing, proliferation and survival of HSCs. In preclinical testing, PROHEMA has demonstrated the potential to accelerate engraftment and to drive durable hematopoietic reconstitution, without the need for multi-week expansion protocols. In an initial Phase 1b clinical trial in adult patients with hematologic malignancies undergoing double umbilical cord blood transplant (dUCBT), the median time to neutrophil recovery ( > 500 cells/µL) with PROHEMA was 17.5 days, which compares favorably to historical norms for patients undergoing dUCBT. In that trial, 100-day survival with PROHEMA was 100%, and PROHEMA provided the dominant source of hematopoiesis in 10 of 12 evaluable subjects, suggesting that treatment with PROHEMA may accelerate engraftment and drive durable and preferential hematologic reconstitution.
Disease: treatment of pediatric patients undergoing hematopoietic stem cell (HSC) transplantation for the treatment of inherited metabolic disorders (IMDs)
Therapeutic
area: Metabolic diseases - Trasplantation - Rare diseases
Country: USA
Trial
details:
- The PROVIDE trial is designed to enroll up to 12 patients with various forms of IMDs, between the ages of 1 and 18, at up to three leading U.S. pediatric HSC transplant centers. The trial\'s inclusion criteria allows for the enrollment of patients with different types of lysosomal and peroxisomal storage diseases such as Hurler and Hunter syndromes, Krabbe disease and various other leukodystrophies, among others. The primary endpoint of the PROVIDE trial is safety as assessed by neutrophil engraftment.
Latest
news:
- • On July 29, 2014, Fate Therapeutics a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators to treat orphan diseases, announced that the FDA has cleared its Investigational New Drug Application (IND) for the clinical development of PROHEMA® in pediatric patients undergoing hematopoietic stem cell (HSC) transplantation for the treatment of inherited metabolic disorders (IMDs). The FDA\'s clearance of the IND allows the Company to begin expanding its clinical investigation of PROHEMA into rare, non-malignant disorders. The Company plans to initiate enrollment of the \"PROVIDE\" trial (PROHEMA eValuation for the treatment of Inherited metabolic DisordErs) in pediatric patients with IMDs in the fourth quarter of 2014.
- PROHEMA® is currently in Phase 2 clinical development for the treatment of adult patients with hematologic malignancies undergoing allogeneic HSC transplantation, and the Company intends to initiate enrollment of a Phase 1b clinical trial in pediatric patients with hematologic malignancy in mid-2014. In 2010, the FDA granted PROHEMA orphan designation for the enhancement of stem cell engraftment in patients undergoing allogeneic cord blood HSC transplantation. In in vivo murine models of allogeneic HSC transplantation, Fate Therapeutics demonstrated that the use of PROHEMA, as compared to unmodulated HSCs, led to a significant increase both in the engraftment of donor HSCs and in the donor-derived expression of enzyme in the brain.
Is
general: Yes
