Phase 1
N=24
A Pilot Study of RNS60 in Amyotrophic Lateral Sclerosis (ALS)
ALS
Bottom Line
View on ClinicalTrials.gov: NCT02525471 ↗Enrolled (actual)
24
Serious AEs
6.3%
Results posted
Mar 2021
Primary outcome: Primary: Safety as Measured by the Number of Participants Experiencing Adverse Events — 16 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- RNS60 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sabrina Paganoni, M.D.
- Primary completion
- Jun 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety as Measured by the Number of Participants Experiencing Adverse Events |
16 | — |
| PRIMARY Tolerability to Complete the Entire 24 Week Study on Study Drug |
13 | — |
| PRIMARY Blood Biomarkers of Inflammation. |
-25.4; -0.02 | — |
| PRIMARY Change From Baseline in Standardized Uptake Value (SUV) Normalized to Whole Brain Mean (SUVR) at Approximately 60-90 Minutes Post Injection |
0.02 | — |
| PRIMARY Clinical Outcome: Change in Pulmonary Function From Baseline to Week 23, Measured by Slow Vital Capacity (SVC) |
-3.3 | — |
| PRIMARY Clinical Outcome: Change in Strength From Baseline to Week 23, Measured by Accurate Test of Limb Isometric Strength (ATLIS) |
-9.0; -9.1 | — |
| PRIMARY Clinical Outcome: Change in Functional Status Between Baseline and Week 23, Measured by Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) |
-5.6 | — |
Summary
The purpose of this study is to determine the safety and tolerability of RNS60 in patients with Amyotrophic lateral sclerosis (ALS). Investigators will also measure the impact of RNS60 on several markers of neuro-inflammation, measured by blood biomarkers and positron emission tomography (PET) imaging.
Eligibility Criteria
Inclusion Criteria
- Amyotrophic Lateral Sclerosis (ALS) volunteers must be diagnosed as having possible, probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria.
- Age 18-80, able to provide informed consent, and comply with study procedures.
- Participants must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-naïve participants are permitted in the study).
- Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control) for the duration of the study and 3 months after study completion.
- Males should practice contraception for the duration of the study and 3 months after completion.
- Ability to safely lie flat for 90 min for Positron Emission Tomography (PET) procedures in the opinion of the study physician.
- High or mixed affinity to bind translocator (TSPO) protein (Ala/Ala or Ala/Thr)
Exclusion Criteria
- Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine transaminase (ALT) > 3 times the upper limit of the normal.
- Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal.
- The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to PI judgment.
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the study.
- History of HIV, clinically significant chronic hepatitis, or other active infection.
- Females must not be lactating or pregnant.
- Active participation in another ALS clinical trial within 30 days of the Screening Visit
- Exposure to immunomodulatory medications within 30 days of the Screening Visit.
- Any contraindication to undergo MRI studies such as
- History of a cardiac pacemaker or pacemaker wires
- Metallic particles in the body
- Vascular clips in the head
- Prosthetic heart valves
- Claustrophobia
- Radiation exposure that exceeds the site's current guidelines
- Current use of tobacco products including cigarettes, cigars, snuff and chewing tobacco, or nicotine replacement products such as gum or patch
Data sourced from ClinicalTrials.gov (NCT02525471). 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.