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

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ClinicalTrials.gov Identifier: NCT02917291
Recruitment Status : Recruiting
First Posted : September 28, 2016
Last Update Posted : August 21, 2018
Sponsor:
Collaborator:
Histocell S.L.
Information provided by (Responsible Party):
Ferrer Internacional S.A.

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.

Condition or disease Intervention/treatment Phase
Acute Traumatic Spinal Cord Injury Drug: FAB117-HC Other: Control group Phase 1 Phase 2

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 between 48 and 72 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): 6 AIS A patients with lesion located between D1 and D12 will be included in 2 sequential cohorts.

Phase 2 (randomized, controlled, double-blind): 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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 46 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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.
Study Start Date : December 2016
Estimated Primary Completion Date : April 2019
Estimated Study Completion Date : January 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: FAB117-HC (Ph 1)
Patients with acute traumatic spinal cord injury grading AIS A (6 patients)
Drug: FAB117-HC
(Ph 1) Intramedullary administration. Open label dose escalation, 3 patients in cohort 1 (20 million cells) and 3 patients in cohort 2 (40 million cells)

Control group (Ph 2)
Patients with acute traumatic spinal cord injury grading AIS A or B (20 patients)
Other: Control group
(Ph 2) No treatment will be administered

Experimental: FAB117-HC (Ph 2)
Patients with acute traumatic spinal cord injury grading AIS A or B (20 patients)
Drug: FAB117-HC
(Ph 2) Intramedullary administration of the maximum tolerated dose (20 or 40 million cells)




Primary Outcome Measures :
  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 [ Time Frame: One year ]

Secondary Outcome Measures :
  1. Changes from baseline 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 [ Time Frame: One year ]
  2. Changes from baseline in the functional assessment of Spinal Cord Independence Measure (SCIM III) [ Time Frame: Day 28 and Day 90 ]
  3. Changes from baseline in Somatosensory-Evoked Potentials (SSEP) electrophysiological assessment test. [ Time Frame: Day 28 and Day 90 ]
  4. Changes from baseline in Motor-Evoked Potentials (MEP) electrophysiological assessment test [ Time Frame: Day 28 and Day 90 ]
  5. Changes from baseline in nerve conduction velocities electrophysiological assessment test [ Time Frame: Day 28 and Day 90 ]


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Phase 1 (2 Cohorts)

  1. Male or female subjects ≥ 18 to ≤ 65 years.
  2. ASIA impairment grade A.
  3. Either a level of injury between D1-D11 both inclusive, and D12 without motor zone of partial preservation (ZPP) (cohorts 1 and 2).
  4. Single traumatic spinal cord injury as defined by MRI.
  5. Injury occurred between 72 and 120h before undergoing DSS and treatment.
  6. Clinically and haemodynamically stable enough to undergo DSS.
  7. Able to give informed consent either in writing or orally in the presence of a witness.

Phase 2 (2 Groups)

  1. Male or female subjects ≥ 18 to ≤ 65 years.
  2. ASIA impairment grade A or B.
  3. An injury between D1 and D11, both inclusive, and D12 without motor zone of partial preservation (ZPP).
  4. Single traumatic spinal cord injury as defined by MRI.
  5. Injury occurring between 24 and 72 h before undergoing DSS and treatment.
  6. Clinically and haemodynamically stable enough to undergo DSS.
  7. Able to give informed consent either in writing or orally in the presence of a witness.

Exclusion Criteria:

  1. 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).
  2. Radiological or MRI or DSS evidence of complete spinal cord transection or equivalent severe lesion or abnormality.
  3. Inability to unequivocally identify the injection sites.
  4. Multiple injuries to the neurological spinal cord at different levels.
  5. Patients with any of these additional conditions:

    1. Penetrating spinal cord injuries.
    2. Associated trauma or injury to the brachial and / or lumbosacral plexus.
  6. Active infection in the surgical area.
  7. Haemodynamic instability contraindicating DSS procedure in the time frame defined for inclusion in the trial.
  8. Multiple organ failure.
  9. 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.
  10. Patients undergoing mechanical ventilation that does not allow a prior clinical examination.
  11. Inability to communicate with the neurological examiner so that the validity of patient data could be unreliable.
  12. 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.
  13. 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.
  14. Background or acute episode of Guillain-Barre syndrome.
  15. History of meningitis or meningoencephalitis.
  16. History of current autoimmune disease.
  17. Patients with history of severe thrombophilia or under anticoagulant or antiplatelet pharmacological therapy.
  18. Presence of any psychiatric illness, as defined by the DSM-IV-TR, or medically unstable illness which can reasonably be expected to pose an increased risk to on participating in the study, or which causes a significant deterioration of the clinical course of the patient. Secondary depression is expressly excluded.
  19. Drug abuse as defined by DSM-IV-TR during the 6 months prior to SCI.
  20. Pregnant women or women of childbearing age who are not using an appropriate method of contraception and, moreover, are not willing to continue to use it for the duration of the trial. If the patient is menopausal or sterile, it must be documented in the medical record.
  21. Women who are breastfeeding if unwilling to stop at the time of recruitment.
  22. History of allergy with anaphylactic shock.
  23. Patients with known hypersensitivity to any of the excipients of FAB117-HC.
  24. Patients with known hypersensitivity to penicillin, streptomycin, enzymes (trypsin or collagenase), bovine serum or DMSO.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02917291


Contacts
Contact: Andrés G Fernández, PhD ferreradvancedbiotherapeutics@ferrer.com

Locations
Spain
Complexo Hospitalario Universitario A Coruña, Unidad de Lesionados Medulares Recruiting
A Coruña, Spain, 15006
Principal Investigator: Antonio Rodríguez Sotillo, MD, PhD         
Hospital Vall d'Hebron, Unidad de Lesionados Medulares Recruiting
Barcelona, Spain, 08035
Principal Investigator: Miguel Ángel González Viejo, MD, PhD         
Hospital Universitario Virgen del Rocío, Unidad de Lesionados Medulares Recruiting
Sevilla, Spain, 41013
Principal Investigator: Juana María Barrera Chacón, MD, PhD         
Sponsors and Collaborators
Ferrer Internacional S.A.
Histocell S.L.

Responsible Party: Ferrer Internacional S.A.
ClinicalTrials.gov Identifier: NCT02917291     History of Changes
Other Study ID Numbers: FAB117-CT-01
First Posted: September 28, 2016    Key Record Dates
Last Update Posted: August 21, 2018
Last Verified: February 2017

Keywords provided by Ferrer Internacional S.A.:
Trauma Spinal Cord
Thoracic Acute Spinal Cord Injury
Neurosurgery
Cell Therapy
ATMP
HC016 adipose mesenchymal stem cell
SPINE

Additional relevant MeSH terms:
Wounds and Injuries
Spinal Cord Injuries
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Trauma, Nervous System