Amyotrophic Lateral Sclerosis (ALS)
Conditions
Keywords
Amyotrophic Lateral Sclerosis, regenerative, synapse
Brief summary
This study will evaluate the long-term safety and efficacy of participants enrolled in SPG302-ALS-101 with Amyotrophic Lateral Sclerosis (ALS)
Detailed description
This is an open-label extension study of SPG302-ALS-001 to investigate the long-term safety, tolerability, and efficacy of SPG302 administered orally in participants with Amyotrophic Lateral Sclerosis (ALS). This study will allow participants in the parent study to continue dosing. Enrolled participants will continue at the dose they received at the end of the first trial, and will self-administer SPG302 orally every day for up to 52 weeks. Participants will have an in-person clinic visit every 3 months (± 3 days) and a monthly phone visit. An end of treatment visit will occur within 7 days of the last dose of SPG302. A final visit to collect safety data will be conducted up to 1 month (± 3 days) post last dose.
Interventions
DRUGSPG302
Open label SPG302 to be self-administered daily by eligible participants for 52 weeks.
Sponsors
Spinogenix
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
None (Open Label)
Intervention model description
Open Label extension
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
• Must have participated in all study activities of SPG302-ALS-001, the parent study
Exclusion criteria
* Unable to reliably and regularly swallow whole oral medications on a daily basis. * Medical conditions that investigator or sponsor determine would interfere with participation in clinical trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment emergent adverse events and serious adverse events | Up to 52 weeks | Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) |
| C-SSRS (Columbia Suicide Severity Rating Scale) | Up to 52 weeks | Prospective suicidality assessment is performed using the Columbia-Suicide Severity Rating Scale (C-SSRS), a questionnaire to evaluate suicidal ideation and behavior. Answer yes on item 4 or 5 of the Suicidal Ideation section or yes on any item of the Suicidal Behavior section is considered positive. The suicidal behavior lethality sub-scale evaluates the level of actual or potential medical damage |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in the Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) scores | up to 52 weeks | Questionnaire administered by a clinician that includes a series of questions about participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. This outcome would evaluate these scores based on patient demographics including clinical presentation at outset of study, presence of ventilation assistance, and patient demographics. Results will be compared to matched historical controls |
| Change in Edinburgh Cognitive and Behavioural ALS Screen (ECAS) | up to 52 weeks | The Edinburgh Cognitive and Behavioural ALS Screen (ECAS) assesses cognitive and behavioral changes in people with (ALS) through a 136-point test covering language, verbal fluency, executive function, memory, and visuospatial cognitive domains. A lower score indicates worsening of symptoms. |
| Changes from baseline in neurofilament light biomarker (NfL) | up to 52 weeks. | To assess the effect of SGP302 on NfL, a biomarker of neurodegeneration. A higher level of this biomarker indicates a progression of this disease. |
Countries
Australia
Outcome results
None listed