Phase 2
N=52
AXER-204 in Participants With Chronic Spinal Cord Injury
Chronic Spinal Cord Injury
Bottom Line
View on ClinicalTrials.gov: NCT03989440 ↗Enrolled (actual)
52
Serious AEs
11.8%
Results posted
Aug 2023
Primary outcome: Primary: Incidence of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 5; 4; 5; 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AXER-204 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- ReNetX Bio, Inc.
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
5; 4; 5; 6; 14; 10 | — |
| PRIMARY Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of AXER-204 in Serum |
NA; NA; 3670; 20900; NA | — |
| PRIMARY Cmax in Serum |
NA; NA; 277; 641; NA | — |
| PRIMARY Tmax in Serum |
NA; NA; 16.8; 12.8; NA | — |
| PRIMARY t1/2 in Serum |
NA; NA; NA; 53.9; NA | — |
| PRIMARY Clearance From Serum |
NA; NA; NA; 4.84; NA | — |
| PRIMARY Volume of Distribution |
NA; NA; NA; 344; NA | — |
| PRIMARY Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of AXER-204 in CSF |
57200; 5810000; 12500000; 10000000; NA | — |
| PRIMARY Cmax of AXER-204 in CSF |
3340; 87900; 280000; 412000; NA | — |
| PRIMARY Tmax of AXER-204 in CSF |
24.2; 23.9; 23.2; 22.9; NA | — |
| PRIMARY t1/2 of AXER-204 in CSF |
NA; 23.1; 13.5; 12.5; NA | — |
| SECONDARY Change in International Standards for Neurological Classification of SCI (ISNCSCI) Bilateral Upper Extremity Motor Score (UEMS) |
2.05; 1.52 | 0.59 |
| SECONDARY Change in Graded Redefined Assessment of Strength, Sensation and Prehension (GRASSP) Bilateral Prehension Performance Score |
0.42; 1.97 | 0.13 |
| SECONDARY Change in Version III of the Spinal Cord Independence Measure (SCIM III) Self-care |
-0.25; 0.91 | 0.16 |
| SECONDARY Patient Global Impression of Change (PGIC) Responder Rate |
NA; NA; NA; NA; 3; 4 | 0.68 |
Summary
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.
Eligibility Criteria
Key Inclusion Criteria
- Traumatic spinal cord injury that occurred ≥ 1 year ago
- 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
- Confirmation by MRI of the following:
- Chronic SCI (persistent spinal cord lesion)
- 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
- CSF space spanning the lesion
Key Exclusion Criteria
- Penetrating injury to the cord or spinal cord trauma caused by ballistic injury including gunshot that did not penetrate the spinal cord
- History of stroke, cerebrovascular injury, or elevated intracranial pressure
- Contraindications for lumbar puncture
- Requiring mechanical ventilatory assistance of any type
- Body mass index (BMI) ≥ 35 kg/m2 or body weight <50 kg
- 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
- Subjects fitted with an implanted pump or port for delivery of therapeutics to the CSF
- Uncontrolled medical condition including but not limited to cardiovascular disease, sleep apnea, obstructive lung disease, severe neuropathic or severe chronic pain, severe autonomic dysreflexia
- 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.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03989440). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.