AXER-204 in Participants With Chronic Spinal Cord Injury

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

52 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-07-16

Study Completion Date

2022-06-21

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This two-part trial will assess the safety, tolerability, pharmacokinetics, and efficacy of AXER-204 administered by lumbar puncture and slow bolus infusion. Part 1 will evaluate the safety, tolerability, and pharmacokinetics of single ascending doses of AXER-204. Part 2 will evaluate the safety, tolerability, pharmacokinetics, and efficacy of repeated doses AXER-204 in comparison to placebo.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Phase I Safety Study of NVG-291 in Healthy Adults

NCT05308953

Spinal Cord Injury

COMPLETED PHASE1

A Study to Evaluate Safety, Tolerability, and Bioavailability of Subcutaneous Lirentelimab (AK002) in Adult Healthy Volunteers

NCT04324268

Study Conducted in Healthy Volunteers

COMPLETED PHASE1

Safety And Efficacy Study Of Intravenous (IV) Administration Of Elezanumab To Assess Change In Upper Extremity Motor Score (UEMS) In Adult Participants With Acute Traumatic Cervical Spinal Cord Injury (SCI)

NCT04295538

Spinal Cord Injury (SCI)

ACTIVE_NOT_RECRUITING PHASE2

A Study of LY3079514 in Healthy Participants

NCT02242903

Healthy Volunteers

COMPLETED PHASE1

The Evaluation of Efficacy and Safety of Rituximab in Refractory CIDP Patients With IgG4 Autoantibodies

NCT03864185

Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Chronic Spinal Cord Injury

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Part 1 is open-label single-ascending dose. Part 2 is double-blind, placebo-controlled, repeat dose.

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Part 1 - None; Part 2 - Quadruple

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

AXER-204

Part 1 - Single ascending doses; Part 2 - Repeated dose

Group Type EXPERIMENTAL

AXER-204

Intervention Type DRUG

human NoGo Trap fusion protein

Placebo

Part 2 only - Repeated dose

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Phosphate buffered saline formulation

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

AXER-204

human NoGo Trap fusion protein

Intervention Type DRUG

Placebo

Phosphate buffered saline formulation

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

human NoGo Trap human Nogo Receptor decoy

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Traumatic spinal cord injury that occurred ≥ 1 year ago
2. Cervical spinal cord injury with serious neurologic deficit as evidenced by 1) bilateral ISNCSCI UEMS between 4 and 36 points inclusive, and 2) bilateral GRASSP Prehension Ability score between 4 and 17 points inclusive
3. Confirmation by MRI of the following:

1. Chronic SCI (persistent spinal cord lesion)
2. For AIS grade of A without sensory or motor zone of partial preservation extending at least two levels caudal to the level of injury, no apparent transection of the cord
3. CSF space spanning the lesion

Exclusion Criteria

1. Penetrating injury to the cord or spinal cord trauma caused by ballistic injury including gunshot that did not penetrate the spinal cord
2. History of stroke, cerebrovascular injury, or elevated intracranial pressure
3. Contraindications for lumbar puncture
4. Requiring mechanical ventilatory assistance of any type
5. Body mass index (BMI) ≥ 35 kg/m2 or body weight \<50 kg
6. History of life threatening allergic or immune-mediated reaction to vaccines, or biologic drugs, at any time or any life threatening allergic or immune-mediated reaction within the past 12 months
7. Subjects fitted with an implanted pump or port for delivery of therapeutics to the CSF
8. Uncontrolled medical condition including but not limited to cardiovascular disease, sleep apnea, obstructive lung disease, severe neuropathic or severe chronic pain, severe autonomic dysreflexia
9. Participation in any other investigational drug or device trial within 30 days or within 5 half-lives of the investigational drug or any past participation in a SCI cellular therapy trial.

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No