Clinical Trials Register

Summary
EudraCT Number:	2013-000906-52
Sponsor's Protocol Code Number:	SC0806-A101
National Competent Authority:	Estonia - SAM
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2017-12-11
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Estonia - SAM

A.2

EudraCT number

2013-000906-52

A.3

Full title of the trial

An Open, Randomized, Rehabilitation-controlled Study to Assess Safety, Tolerability, and Efficacy of Heparin Activated Recombinant Human Fibroblast Growth Factor 1 on a Biodegradable Device in Subjects with Traumatic Spinal Cord Injury

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A.4.1

Sponsor's protocol code number

SC0806-A101

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	BioArctic AB
B.1.3.4	Country	Sweden
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	BioArctic AB
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	BioArctic AB
B.5.2	Functional name of contact point	Clinical trials information
B.5.3	Address:
B.5.3.1	Street Address	Warfvinges väg 35
B.5.3.2	Town/ city	Stockholm
B.5.3.3	Post code	SE-112 51
B.5.3.4	Country	Sweden
B.5.4	Telephone number	+46(0)734411798
B.5.5	Fax number	+46(0)86956939
B.5.6	E-mail	hans.basun@bioarctic.se

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/754
D.3 Description of the IMP
D.3.1	Product name	Heparin-activated recombinant human Fibroblast Growth Factor 1 (rhFGF1) with diluent and SCI-device
D.3.2	Product code	SC0806
D.3.4	Pharmaceutical form	Implant
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intrathecal use
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Yes
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Complete Traumatic Spinal Cord Injury (TSCI)

E.1.1.1

Medical condition in easily understood language

Complete Traumatic Spinal Cord Injury (TSCI)

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

• To assess the safety and tolerability of one implantation of SC0806 in subjects with complete Traumatic Spinal Cord Injury.
• To assess the efficacy of one implantation of SC0806 on Electrophysiology (MEP) in subjects with complete Traumatic Spinal Cord Injury.

E.2.2

Secondary objectives of the trial

To evaluate/assess
• plasma concentrations of FGF1
• changes of the neurological classification
• motor function
• effect of specific walking training
• functional improvements
• pain scores
• urinary function
• Quality of Life
• Immunogenicity (anti-FGF1 antibody)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Traumatic Spinal Cord Injury
2. Male or female subjects aged between 18 and 65 years.
3. Body Mass Index (BMI) ≤35, body weight ≤125 kg and height ≤ 195 cm at Screening.
4. Complete spinal cord injury (ASIA Impairment Scale level A, no voluntary bladder function, negative motor and sensory evoked potentials).
5. A single spinal cord lesion injury at the neurologic level between T2-T11
6. A Baseline MRI that indicates a pathology consistent with a traumatic SCI
7. Minimum of 4 months and maximum 10 years post injury with no evidence of neurological improvement prior to implantation surgery unless there is a complete anatomical cut-off of the spinal cord as judged from the MRI assessment.
8. Females must not be lactating or pregnant at Screening or Baseline (as documented by negative pregnancy tests).
9. All females that are are postmenopausal (amenorrheic for at least 12 consecutive months, in the appropriate age group, and without other known or suspected cause) or have been sterilized surgically (i.e., bilateral tubal ligation, total hysterectomy, or bilateral oophorectomy, all with surgery at least 1 month before dosing).
10. Females of childbearing potential must not have had unprotected sexual intercourse within 30 days before study entry and must agree to use a highly effective method of contraception (e.g., total abstinence, an intrauterine device, a double-barrier method [such as condom plus diaphragm with spermicide], a contraceptive implant, an oral contraceptive, or have a vasectomized partner with confirmed azoospermia) throughout the entire study period. If currently abstinent, the subject must agree to use a double-barrier method as described above if she becomes sexually active during the study period. Females who are using hormonal contraceptives must have been on a stable dose of the same hormonal contraceptive product for at least 4 weeks before dosing and must continue to use the same contraceptive during the study.
11. Written informed consent obtained prior to any study specific procedures.
12. Eligible for surgery and specific walking training as judged by the investigator.

E.4

Principal exclusion criteria

1. Other life-threatening injury.
2. Serious co-existing medical condition or mental disorder.
3. Results from neurophysiological examination preoperatively are inconsistent with a spinal cord injury of one thoracic segment or less.
4. Current or prior (within the past 8 weeks or within 5 half-lives of use of such a medication prior to screening) participation in any other investigational medication or device trial.
5. Known hypersensitivity to , FGF1 or heparin
6. Subjects unable to tolerate or undergo MRI scanning, including subjects with claustrophobia unless sedation can be used, cardiac pacemaker/defibrillator, ferromagnetic metal implants e.g., in skull, cardiac devices, other than those approved as safe for use in MR scanners.
7. Ongoing drug or alcohol abuse or dependence.
8. Positive serology for Hepatitis B or C, or Human Immunodeficiency Virus (HIV) at Screening.
9. Positive test for Methicillin-resistant Staphylococcus Aureus (MRSA) at screening.
10. Any disease, concomitant injury, condition or treatment that interferes with the specific walking training, the performance or interpretation of the neurological examination.
11. Has a condition or has received medical treatment that, in the judgment of the investigator, precludes successful participation in the study.
12. Previous radiation treatment (e.g. cancer treatment) in the region of the spinal cord injury.

E.5 End points

E.5.1

Primary end point(s)

Primary 1:
• adverse events (AEs/ADEs) and serious adverse events (SAEs/SADEs)
• clinical significant changes of hematology, blood chemistry, and urine values
• vital signs, i.e. clinical significant changes in
o systolic and diastolic blood pressure
o heart rate (beats per minute)
o body temperature
o respiratory rate (rate per minute)
• electrocardiograms (ECGs)
• CT of device area
• MRI
• clinical significant changes on physical examinations
Primary 2:
• Proportion of subjects with an improvement, from baseline to 18 months in the Motor Evoked Potential (MEP) scores

E.5.1.1

Timepoint(s) of evaluation of this end point

From baseline to 18 months

E.5.2

Secondary end point(s)

Secondary 1:
• FGF1 plasma concentration
Secondary 2:
• Change from baseline to Final Visit in the American Spinal Injury Association Impairment Scale (AIS) regarding neurological classification (grades)
Secondary 3:
• Change from baseline to Final Visit in the American Spinal Injury Association Impairment Scale (AIS) regarding motor function (motor score)
Secondary 4:
• Specific walking training: change from baseline to Final Visit in
o L-Force: (measures the isometric power (Nm) generated by the subject in a static position)
o L-Stiff: (measures the mechanical stiffness (Nm/degrees) in the joints of the subject (hip and knee) while the legs are led in a passive movement.)
• L-ROM: (measures the passive movement pattern (degrees) of the subject for flexion and extension in the hip and knee joints)
Secondary 5:
• Change from baseline to Final Visit in the Spinal Cord Independence Measure (SCIM)
Secondary 6:
• Change from baseline to Final Visit in the Graded Chronic Pain (GCP) Disability Scale
Secondary 7:
• Change from baseline to Final Visit in the following urodynamic variables
o Free flow
o Residual volume after micturition
o Cystometry (bladder capacity, sense of bladder filling and bladder pressure)
o Pressure flow (bladder pressure required to urinate and the flow rate a given pressure generates)
o Urethral pressure
o Ice-water test (presence of undesired bladder activity)
• Urine and leakage measurements (mean values from measurements on two separate days
Secondary 8:
• Change from baseline to Final Visit in the SCI-QoL assessment scale
• Change from baseline to Final Visit in the I-QoL assessment
• Change from baseline to Final Visit in the ICIQ-SF assessment scale
Secondary 9:
• Anti-FGF1 concentrations
Secondary 10:
• Proportion of subjects with an improvement, from baseline to Final Visit in the Motor Evoked Potential (MEP) scores

E.5.2.1

Timepoint(s) of evaluation of this end point

From baseline to Final Visit

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

Yes

E.7.1.1

First administration to humans

Yes

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Rehabilitation only, no treatment

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

F.3.3.1

Women of childbearing potential not using contraception