NCT02917291

Safety and Preliminary Efficacy of FAB117-HC in Patients With Acute Traumatic Spinal Cord Injury

Clinical Trial of Phase 1/2 to Evaluate the Feasibility, Safety, Tolerability and Preliminary Efficacy of the Administration of FAB117-HC, a Drug Whose Active Ingredient is HC016, Allogeneic Adipose Derived Adult Mesenchymal Stem Cells Expanded and Pulsed With H2O2, in Acute Traumatic SCI Patients.

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Sponsor: Ferrer Internacional S.A. · Industry
• Sex: All
• Age: 16 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

The main objective of the study is the evaluation of the safety and tolerability of FAB117-HC (a medicinal product containing human allogeneic adipose derived adult mesenchymal stem cells expanded and pulsed with H2O2, HC016 cells) administered at a single-time point to patients with acute thoracic traumatic spinal cord injury (SCI). The study will also include initial exploration of potential clinical efficacy. Dose levels of 20 million and 40 million cells will be administered.

Detailed description

FAB117-HC is an investigational medicinal product whose active substance is HC016, allogeneic adipose-derived adult mesenchymal stem cells expanded and pulsed with H2O2. The main purpose of this study is to evaluate the safety and tolerability of a single administration of FAB117-HC using: a) two sequential escalating doses administered between 72 and 120 hours post-injury, to patients with acute traumatic SCI with ASIA Impairment Scale (AIS) grade A; and b) the determined maximum tolerated dose administered up to 96 h post-injury to patients with AIS grading of A or B. The study includes also initial exploration of efficacy. Treatment is administered by intramedullary injection into the injured spinal cord, during the decompression and stabilization surgery (DSS) of the fracture. DSS is routinely performed on almost all SCI patients. The study has been divided into two phases: Phase 1 (open label): 8 AIS A patients with lesion located between D1 and D12 will be included in 2 sequential cohorts. Phase 2 (randomized, controlled, double-blind): Up to 40 AIS A or B patients with lesion located between D1 and D12, will be randomly divided into two groups (control and treated) that will be balanced in AIS grade.

Conditions

• Acute Traumatic Spinal Cord Injury

Interventions

Type	Name	Description
DRUG	FAB117-HC	(Ph 1) Intramedullary administration. Open label dose escalation, 3 patients in cohort 1 (20 million cells) and 5 patients in cohort 2 (40 million cells)
OTHER	Control group	(Ph 2) No treatment will be administered
DRUG	FAB117-HC	(Ph 2) Intramedullary administration of the maximum tolerated dose (20 or 40 million cells)

Timeline

• Start date: 2016-12-01
• Primary completion: 2022-07-01
• Completion: 2023-07-01
• First posted: 2016-09-28
• Last updated: 2021-09-16

Locations

9 sites across 1 country: Spain