Clinical trial NCT05299658: An Open-Label Extension for the Phase 2 Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS
Official title: An Open-Label Extension for the Phase 2, Randomised, Double-Blind, Placebo-Controlled Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Open-label extension
Lead sponsor: Clene Nanomedicine
Sponsor class: INDUSTRY
First posted: 2022-03-29
Enrollment: 40
Sex: ALL
Minimum age (years): 30
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label extension
Primary purpose: TREATMENT

Brief summary: This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.

Detailed description: A forty-eight (48) week optional open-label extension period (Open- Label Period), which may be extended by 12-week increments until discontinued by the Sponsor for participants that have completed protocol CNMAu8.205.

Eligibility criteria: Inclusion Criteria:

1. Participants must have completed the randomized placebo-controlled Treatment Period without compliance issues.
2. Able to understand and give written informed consent to participate in the open-label extension.
3. If referred from a third party (neurologist or a State based ALS organisation), participant agrees to maintain transfer of care to a neurologist participating in the study.

Exclusion Criteria:

1. Lack of treatment compliance during the randomized placebo controlled Treatment Period.
2. Positive pregnancy test at the Week 36 visit, or, females who plan to get pregnant during the course of this extension or within 6 months of the end of this extension.
3. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.
4. Patient with clinically significant abnormalities in haematology, blood chemistry, ECG, or physical examination identified during the W36 visit which according to Investigator may interfere with continued participation.
5. Patients with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (\< 150 x 10\^9 per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter) at the Week 36 visit.
6. Patient is considered a suicide risk in the opinion of the Investigator, has previously made a suicide attempt, or is currently demonstrating active suicidal ideation. Subjects with intermittent passive suicidal ideation are not necessarily excluded based on the assessment of the Investigator.

Primary outcomes:
measure: Safety measures
timeFrame: Up to an estimated 96 weeks.
measure: Electromyography measures
timeFrame: Up to an estimated 96 weeks.
Secondary outcomes:
Other outcomes:
measure: MScanFit MUNE
timeFrame: Up to an estimated 96 weeks.
measure: MUSIX
timeFrame: Up to an estimated 96 weeks.
measure: Spilt Hand Index
timeFrame: Up to an estimated 96 weeks.
measure: Neurophysiological index
timeFrame: Up to an estimated 96 weeks.
Locations:
city: Concord
country: Australia
facility: Concord Hospital
state: New South Wales
city: Randwick
country: Australia
facility: Neuroscience Research Australia
state: New South Wales
Study officials:
References: