Industry Update: Clinical trials
Latestdevelopments compiled from October 01 — November 30 2015
Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions October 01 — November 30 2015, scheduled to be published in Volume 11 Issue 2 of Regenerative Medicine.
Clinical trials
Asterias
Asterias Biotherapeutics (CA, USA; www.asteriasbiotherapeutics.com), subsidiary of BioTime (CA, USA; www.biotimeinc.com), announced that its Data Monitoring Committee has reviewed the initial safety data from all three newly injured patients with sensory and motor complete cervical spinal cord injury in the first cohort and has recommended dose escalation to the second cohort. Recruitment for the second cohort will begin immediately, with a planned enrollment of five patients who will each receive 10 million of oligodendrocyte progenitor AST-OPC1 cells. The three patients in the first cohort were administered a low dose of 2 million AST-OPC1 cells and have completed at least 30 days of follow up for safety. The results of the trial continue to support a positive safety profile for AST-OPC1, with no serious adverse events observed to date in any of the three treated patients. Additional information on the Phase 1/2a study, including trial sites, can be found at www.clinicaltrials.gov (ID: NCT02302157) and at the SCiStar Study Website www.scistarstudy.com
Cytori
Cytori Therapeutics (CA, USA; www.cytori.com) has enrolled and treated the first patient in the SCLERADECII trial, a late phase, follow on European clinical trial designed to determine the efficacy and safety of Cytori Cell Therapyâ„¢ in patients with hand dysfunction associated with scleroderma. SCLERADECII is a multicenter, double blind, placebo controlled trial of a single administration of Cytori Cell Therapy (ECCS50). The trial will enroll up to 40 patients randomized in a 1:1 fashion to either active or placebo control. If the trial endpoint is successfully achieved, this trial may be used to seek European marketing approval of Cytori Cell Therapy for this indication, which has been designated by the European Commission as an orphan medicinal product for treatment of scleroderma. Additional information can be found at www.clinicaltrials.gov (ID: NCT02558543).
Osiris
Osiris Therapeutics (MD, USA; www.osiris.com) initiated a multicenter, randomized, blinded study with an open-label extension option to evaluate the safety and efficacy of OTI-15-01 for the treatment of chronic diabetic foot ulcers. OTI-15-01 is comprised of several components known to be critical for effective wound healing including a collagen rich matrix, growth factors and neonatal fibroblasts, epithelial cells, endothelial cells, and MSC. These cell types mediate their functions in response to the local tissue microenvironment and secrete a variety of growth factors and extracellular matrix proteins that orchestrate wound healing. The phase III trial is designed as a randomized, controlled, blinded study, and is expected to enroll up to 224 patients in approximately 20 sites. This protocol for OTI-15-01 is conducted under the Investigational New Drug (IND) application IND 16383. Additional information can be found at www.clinicaltrials.gov (ID: NCT02571738).
SanBio
SanBio Inc. (CA, USA; www.san-bio.com) has opened patient recruitment for its Phase II clinical trial study to evaluate the clinical efficacy and safety of its proprietary cell therapy following traumatic brain injury. The “Stem cell therapy for traumatic brain injury” STEMTRA trial will examine the effects of genetically modified adult bone marrow derived stem cells SB623, specifically in patients with chronic motor deficits resulting from focal traumatic brain injury. The study will be conducted across approximately 25 clinical trial sites throughout the U.S. Total enrolment is expected to reach 52 patients. Additional information can be found at www.clinicaltrials.gov (ID: NCT02416492) and at the SanBio website at http://www.san-bio.com/clinical/clinical_trials.php
StemCells
StemCells (CA, USA; www.stemcellsinc.com) announced that the six-month interim results for the first cohort in its ongoing Phase II Pathwayâ„¢ Study in cervical spinal cord injury (SCI) showed motor improvements in both strength and function. The patients were treated with proprietary Human Central Nervous System Stem Cells (HuCNS-SC). The assessment of motor function involved using tests of dexterity. Patients eligible for the study have complete loss of motor control below the level of injury, the most severe degree of SCI as defined by the American Spinal Injury Association Impairment Scale. Clinicians used both International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) and Graded Assessment of Strength Sensibility and Prehension (GRASSP) measures to establish a pre-transplant baseline for each patient and to assess post-transplant progress. This first cohort of the Pathway Study was designed to assess the safety, and preliminary signs of efficacy, of cell administration into the cervical cord and select the dose level for the 40-patient second cohort, a randomized, controlled and single-blinded arm of the trial, which is already underway. Based on six-month follow-up, for the first cohort, an overall pattern of motor improvement was detected in four of the six patients as measured by gains in both strength and function on the collective ISNCSCI and GRASSP outcomes. Additional information can be found at www.clinicaltrials.gov (ID: NCT02163876)
StemCutis
StemCutis (CA, USA; www.stemcutis.com), a subsidiary of Stemedica Cell Technologies, (CA, USA; www.stemedica.com), has enrolled the first cohort of three subjects in its Phase I/II clinical trial for subjects with cutaneous photoaging. The study entitled “A Phase I/II, Open-Label Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Mesenchymal Bone Marrow Cells to Subjects for Cutaneous Photoaging” is the first clinical trial in the U.S. to use allogeneic stem cells systemically for the treatment of photoaged skin. Additional information can be found at www.clinicaltrials.gov (ID: NCT01771679).