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

NCT02917291 | Unknown Phase 1 DMC

• 1 other identifier: FAB117-CT-01
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Acute Traumatic Spinal Cord Injury

Keywords

Trauma Spinal Cord; Thoracic Acute Spinal Cord Injury; Neurosurgery; Cell Therapy; ATMP; HC016 adipose mesenchymal stem cell; SPINE

Outcome Measures

Primary Outcomes (1)

• Number of adverse events as a measure of safety and tolerability of a single dose of FAB117-HC when administered by intramedullary injection into the injured spinal cord

  One year

Secondary Outcomes (4)

• Changes in neurological function using the International Standards for Neurological Classification of SCI (ISNCSCI) scale, examinations at 24h, 72h, 7d, 14d, 28d, 90d and 360 days after injection of FAB117-HC

  One year

• Changes in the functional assessment of Spinal Cord Independence Measure (SCIM III)

  Day 28 and Day 90

• Changes in Somatosensory-Evoked Potentials (SSEP) electrophysiological assessment test.

  Day 28 and Day 90

• Changes in Motor-Evoked Potentials (MEP) electrophysiological assessment test

  Day 28 and Day 90

Study Arms (3)

FAB117-HC (Ph 1)

EXPERIMENTAL

Patients with acute traumatic spinal cord injury grading AIS A (8 patients)

Drug: FAB117-HC

Control group (Ph 2)

OTHER

Patients with acute traumatic spinal cord injury grading AIS A or B (up to 20 patients)

Other: Control group

FAB117-HC (Ph 2)

EXPERIMENTAL

Patients with acute traumatic spinal cord injury grading AIS A or B (up to 20 patients)

Drug: FAB117-HC

Interventions

FAB117-HC DRUG

(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)

FAB117-HC (Ph 1)

Control group OTHER

(Ph 2) No treatment will be administered

Control group (Ph 2)

Eligibility Criteria

• Age: 16 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Phase 1 (2 Cohorts)
• Male or female subjects ≥ 16 to ≤ 70 years.
• ASIA impairment grade A.
• Either a level of injury between D1-D12 both inclusive (cohorts 1 and 2).
• Single traumatic spinal cord injury as defined by MRI.
• Injury occurred between 72 and 120h before undergoing DSS and treatment.
• Clinically and haemodynamically stable, under medical criteria, enough to undergo DSS.
• Able to give informed consent either in writing or orally in the presence of a witness.
• Phase 2 (2 Groups)
• Male or female subjects ≥ 16 to ≤ 70 years.
• ASIA impairment grade A or B.
• An injury between D1 and D12, both inclusive.
• Single traumatic spinal cord injury as defined by MRI.
• Injury occurring up to 96 h before undergoing DSS and treatment.
• Clinically and haemodynamically stable enough, under medical criteria, to undergo DSS.

You may not qualify if:

• Participated in a previous clinical study and received an investigational product within 28 days of SCI (within 5 years of SCI if the investigational product is a cell-based medicine).
• Radiological or MRI or DSS evidence of complete or partial spinal cord transection.
• Inability to unequivocally identify the injection sites.
• Multiple injuries to the neurological spinal cord at different levels.
• Patients with any of these additional conditions:
• Penetrating spinal cord injuries.
• Associated trauma or injury to the brachial and / or lumbosacral plexus.
• Active infection in the surgical area.
• Multiple organ failure.
• Significant head injury (Score on the Glasgow scale less than or equal to 13 and / or abnormal MRI/CT, meaning oedema, axonal lesion and/or haemorrhage) or other injury that in the investigator's opinion is sufficient to interfere with the assessment of spinal cord function or compromise the validity of patient data.
• Patients undergoing mechanical ventilation that does not allow a prior clinical examination.
• Inability to communicate with the neurological examiner so that the validity of patient data could be unreliable.
• Coma or significant impairment in the level of consciousness, including unconsciousness due to sedative-analgesic medications, that interferes with the performance or interpretation of assessments specific in the protocol.
• Preexisting or current significant diseases such as hepatitis C, HIV, epilepsy, neoplastic disease or other diseases that could cause neurological deficits including syphilis, myelopathy, and polyneuropathy.
• Background or acute episode of Guillain-Barre syndrome.

Sponsors & Collaborators

• Ferrer Internacional S.A.: lead
• Histocell, S.L.: collaborator

Study Sites (9)

Complexo Hospitalario Universitario A Coruña

A Coruña, 15006, Spain

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

RECRUITING

Hospital Universitario Virgen de las Nieves

Granada, 18014, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

RECRUITING

Hospital Universitario Virgen del Rocío

Seville, 41013, Spain

RECRUITING

Complejo Hospitalario de Toledo (HNP y VS)

Toledo, 45071, Spain

RECRUITING

Hospital Universitari La Fe

Valencia, 46009, Spain

RECRUITING

Hospital Clínico Universitario Lozano Blesa

Zaragoza, 50009, Spain

RECRUITING

Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain

RECRUITING

MeSH Terms

Conditions

Spinal Cord Injuries

Interventions

Control Groups

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Wounds and Injuries

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Central Study Contacts

Andrés G Fernández, PhD

CONTACT

ferreradvancedbiotherapeutics@ferrer.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2016
• First Posted: September 28, 2016
• Study Start: December 1, 2016
• Primary Completion: July 1, 2022
• Study Completion: July 1, 2023
• Last Updated: September 16, 2021

Record last verified: 2021-09

Locations

ES (9)