AXER-204 in Participants With Chronic Spinal Cord Injury (RESET)
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|ClinicalTrials.gov Identifier: NCT03989440|
Recruitment Status : Recruiting
First Posted : June 18, 2019
Last Update Posted : January 20, 2021
|Condition or disease||Intervention/treatment||Phase|
|Chronic Spinal Cord Injury||Drug: AXER-204 Drug: Placebo||Phase 1 Phase 2|
AXER-204 is a human fusion protein that acts as a soluble decoy/trap for the myelin-associated inhibitors of axonal growth known as Nogo-A, MAG, and OMgp. AXER-204 and a surrogate protein used in early preclinical studies have been found to promote axon growth and recovery of function in animal models of spinal cord injury.
Part 1 of the trial is a multicenter, open-label, single ascending dose study in participants with chronic cervical spinal cord injury. Four cohorts of 6 participants each are planned, with participants within each cohort expected to receive the same dose of AXER-204.
Part 2 is a multicenter, randomized, double-blind, placebo-controlled, repeat dose study in chronic cervical spinal cord injury participants. Approximately 32 participants will be randomized (ratio 1:1) to receive repeated doses of AXER-204 or placebo (a phosphate buffered saline formulation). The dose level and dose frequency will be dependent upon outcomes from Part 1.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||56 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Part 1 is open-label single-ascending dose. Part 2 is double-blind, placebo-controlled, repeat dose.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||Part 1 - None; Part 2 - Quadruple|
|Official Title:||A Multicenter, Two Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of AXER-204 in Subjects With Chronic Spinal Cord Injury|
|Actual Study Start Date :||July 16, 2019|
|Estimated Primary Completion Date :||June 2022|
|Estimated Study Completion Date :||June 2022|
Part 1 - Single ascending doses; Part 2 - Repeated dose
human NoGo Trap fusion protein
Placebo Comparator: Placebo
Part 2 only - Repeated dose
Phosphate buffered saline formulation
- Incidence of treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 Post-dose to 28-days after last dose ]An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
- Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of AXER-204 [ Time Frame: Day 1 pre-dose up to Day 29 ]AXER-204 will be measured in cerebrospinal fluid (CSF) and in serum
- Change in International Standards for Neurological Classification of SCI (ISNCSCI) Upper Extremity Motor Score (UEMS) [ Time Frame: Baseline to Day 169 and Day 253 ]The ISNCSCI scale includes a Motor and Sensory examination for each side of the body (left/right). The Sensory examination involves 'light touch' and 'pinprick' for each dermatome (28) on both sides of the body (total 56). A score of 0, 1 or 2 can be given to each dermatome resulting in a total max. of 112 points. The Motor level is determined by examining the muscle function within each of the 10 myotomes on each side of the body (20 myotomes in total). A score ranging from 1 to 5 can be given to each myotome tested resulting in a maximum score of 100 (50 for upper extremity and 50 for lower extremity). Higher values indicate better function with the maximum scores corresponding to normal function.
- Change in Graded Redefined Assessment of Strength, Sensation and Prehension (GRASSP) scores [ Time Frame: Baseline to Day 169 and Day 253 ]The GRASSP is a clinical impairment measure for the upper limb for use after tetraplegia. The measure includes domains which are important in describing hand function: Strength with scores from 0-50 for each side, Sensory testing with scores ranging from 0-12 for each hand, Prehension ability with scores 0-12 for each hand, and Prehension performance with scores from 0-30 for each hand. Higher subscores indicate better function.
- Change in version III of the Spinal Cord Independence Measure (SCIM III) self-care and mobility domain scores [ Time Frame: Baseline to Day 169 and Day 253 ]The SCIM III is a questionnaire that assesses activities of daily living (ADL) regarding self-care, mobility, respiration, and sphincter management. The self-care and mobility domains have scores ranging from 0-20 and 0-40 respectively. Higher scores correspond to better ability to carry out ADL.
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): NCT03989440
|Contact: Gilbert Block, Chief Medical Officer, MD, PhDfirstname.lastname@example.org|
|United States, California|
|Keck Medicine of USC||Recruiting|
|Los Angeles, California, United States, 90033|
|Contact: Sandra Oviedo email@example.com|
|Principal Investigator: Charles Liu, MD, PhD|
|United States, Georgia|
|Atlanta, Georgia, United States, 30309|
|Contact: Casey Kandilakis, PT,DPT,NCS firstname.lastname@example.org|
|Principal Investigator: Donald Leslie, MD|
|United States, Illinois|
|Shirley Ryan AbilityLab||Recruiting|
|Chicago, Illinois, United States, 60611|
|Contact: Connie Casale 312-238-1522 email@example.com|
|Principal Investigator: David Chen, MD|
|United States, Massachusetts|
|Spaulding Rehabilitation||Not yet recruiting|
|Boston, Massachusetts, United States, 02129|
|Contact: Jenny Min 617-952-6173 firstname.lastname@example.org|
|Principal Investigator: Ross Zafonte, DO|
|Sub-Investigator: Chloe Slocum, MD, MPH|
|United States, Ohio|
|The Ohio State University Wexner Medical Center||Recruiting|
|Columbus, Ohio, United States, 43210|
|Contact: Amy Bartlett, BA, CCRC 614-366-9050 email@example.com|
|Principal Investigator: Jan Schwab, MD, PhD|
|United States, Pennsylvania|
|Thomas Jefferson University||Recruiting|
|Philadelphia, Pennsylvania, United States, 19107|
|Contact: Marilyn Owens, RN, BSN|
|Principal Investigator: Ralph Marino, MD|
|Study Director:||George Maynard, PhD||ReNetX Bio|