Acute Traumatic Spinal Cord InjuryPhase 1/2SPINENCT02917291
Safety and Preliminary Efficacy of FAB117-HC in Patients With Acute Traumatic Spinal Cord Injury
A Phase 1/2 study testing FAB117-HC in people with Acute Traumatic Spinal Cord Injury, run by Ferrer Internacional S.A. at 9 sites in Spain.
Trial 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… Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- Acute Traumatic Spinal Cord Injury
- What is being tested?
- FAB117-HC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 9 sitesSpain
- Study dates
- Dec 2016to Jul 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
- Able to give informed consent either in writing or orally in the presence of a witness.
Who cannot join
- 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.
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- Associated trauma or injury to the brachial and / or lumbosacral plexus.
- Active infection in the surgical area.
- Haemodynamic instability contraindicating DSS procedure in the time frame defined for inclusion in the trial.
- Multiple organ failure.
- Severe multiple trauma that hampers the stabilization procedure in the defined term for the inclusion in the trial.
- 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.
- History of meningitis or meningoencephalitis.
- Current autoimmune disease treated with immunosuppressant therapy.
- Patients with history of severe thrombophilia or under anticoagulant pharmacological therapy which long elimination half-live prevents a rapid transition to heparin (like dabigatran and rivaroxaban).
- Presence of any psychiatric illness, as defined by the DSM-IV-TR, or medically unstable illness that means a hindrance to the adherence to rehabilitation and/or to the informed consent signature.
- 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.
- Women who are breastfeeding if unwilling to stop at the time of recruitment.
- History of allergy with anaphylactic shock.
- Patients with known hypersensitivity to any of the excipients of FAB117-HC.
- Patients with known hypersensitivity to penicillin, streptomycin, enzymes (trypsin or collagenase), bovine serum or DMSO.
Trial locations and contacts
9 sites in 1 country.
Andrés G Fernández, PhD contact
9 sites in 1 country
Spain9 sites, 9 recruiting
- Recruiting
Complexo Hospitalario Universitario A Coruña
A Coruña, 15006
Antonio Rodríguez Sotillo, MD, PhD
- Recruiting
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Miguel Ángel González Viejo, MD, PhD
- Recruiting
Hospital Universitario Virgen de las Nieves
Granada, 18014
Inmaculada García Montes, MD PhD
- Recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
Igor Paredes Sansinenea, MD, PhD
- Recruiting
Hospital Universitario Virgen del Rocío
Seville, 41013
Juana María Barrera Chacón, MD, PhD
- Recruiting
Complejo Hospitalario de Toledo (HNP y VS)
Toledo, 45071
Ángel Gil Agudo, MD, PhD
- Recruiting
Hospital Universitari La Fe
Valencia, 46009
Teresa Bas, MD, PhD
- Recruiting
Hospital Clínico Universitario Lozano Blesa
Zaragoza, 50009
Isabel Villarreal, MD PhD
- Recruiting
Hospital Universitario Miguel Servet
Zaragoza, 50009
Begoña Hidalgo, MD PhD
About this study
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.
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.
Read the 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
- Being tested
- FAB117-HC, Control group
- Keywords
- Trauma Spinal Cord, Thoracic Acute Spinal Cord Injury, Neurosurgery, Cell Therapy, ATMP, HC016 adipose mesenchymal stem cell, SPINE
- Medical subject headings
- Spinal Cord Injuries
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FAB117-HCDrug
(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)
Control groupOther
(Ph 2) No treatment will be administered
FAB117-HCDrug
(Ph 2) Intramedullary administration of the maximum tolerated dose (20 or 40 million cells)
| Study group | Role | What they receive |
|---|---|---|
| FAB117-HC (Ph 1) | Experimental | Patients with acute traumatic spinal cord injury grading AIS A (8 patients) Receives: Drug: FAB117-HC |
| Control group (Ph 2) | Other | Patients with acute traumatic spinal cord injury grading AIS A or B (up to 20 patients) Receives: Other: Control group |
| FAB117-HC (Ph 2) | Experimental | Patients with acute traumatic spinal cord injury grading AIS A or B (up to 20 patients) Receives: Drug: FAB117-HC |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: One year
Changes in the functional assessment of Spinal Cord Independence Measure (SCIM III)
Measured: Day 28 and Day 90
Changes in Somatosensory-Evoked Potentials (SSEP) electrophysiological assessment test.
Measured: Day 28 and Day 90
Changes in Motor-Evoked Potentials (MEP) electrophysiological assessment test
Measured: Day 28 and Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2016 (estimated)
- Study startDec 2016
- Primary completionJul 2022 (estimated)
- Study completionJul 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Traumatic Spinal Cord InjuryStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ferrer Internacional S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Histocell, S.L.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02917291
- Sponsor's protocol ID
- FAB117-CT-01
- Acronym
- SPINE
- Status last verified
- Sep 2021
- First submitted
- 20 Sep 2016
- Last updated
- 16 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT02917291
- Acronym / sponsor ID
- SPINE / FAB117-CT-01
- 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
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Ferrer Internacional S.A.Industry (pharma, biotech, devices)
- Collaborators
- Histocell, S.L.
- Study start
- Dec 2016
- Primary completion
- Jul 2022 (estimated)
- Study completion
- Jul 2023 (estimated)
- First posted
- 28 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| FAB117-HC (Ph 1) | Experimental | Drug: FAB117-HCPatients with acute traumatic spinal cord injury grading AIS A (8 patients) |
| Control group (Ph 2) | Other | Other: Control groupPatients with acute traumatic spinal cord injury grading AIS A or B (up to 20 patients) |
| FAB117-HC (Ph 2) | Experimental | Drug: FAB117-HCPatients with acute traumatic spinal cord injury grading AIS A or B (up to 20 patients) |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- 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.
Key exclusion criteria (26 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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) | Drug | FAB117-HC (Ph 1) |
| Control group(Ph 2) No treatment will be administered | Other | Control group (Ph 2) |
| FAB117-HC(Ph 2) Intramedullary administration of the maximum tolerated dose (20 or 40 million cells) | Drug | FAB117-HC (Ph 2) |
Primary outcome
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
4 secondary outcomes
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
Time frame: One year
Changes in the functional assessment of Spinal Cord Independence Measure (SCIM III)
Time frame: Day 28 and Day 90
Changes in Somatosensory-Evoked Potentials (SSEP) electrophysiological assessment test.
Time frame: Day 28 and Day 90
Changes in Motor-Evoked Potentials (MEP) electrophysiological assessment test
Time frame: Day 28 and Day 90
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrés G Fernández, PhD contact
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FAB117-HC in people with Acute Traumatic Spinal Cord Injury, run by Ferrer Internacional S.A. at 9 sites in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Andrés G Fernández, PhD (ferreradvancedbiotherapeutics@ferrer.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2021.