A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis - A study of RT001 treatment in amyotrophic lateral sclerosis.

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON51123

Enrollment

Registered

2021-01-18

Start date

2021-02-23

Completion date

Unknown

Last updated

2024-04-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

ALS

Interventions

RT001 or placebo soft capsules, oral use

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1. Male or female subject with age 20 years to 75 years at the time of signed consent for double-blind part of the study. 2. Patients who are defined as "definite ALS," "probable ALS" or "probable-laboratory-supported ALS," met diagnostic criteria revised EL Escorial for Airlie House. 3. ALSFRS-R > 20 4. Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life 5. Patients of less than 3 years after the onset of ALS 6. Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend) 7. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation

Exclusion criteria

Exclusion criteria: 1. Received treatment with other experimental therapies within the last 30 days prior to the first dose (for double-blind part of the study only) 2. Previously received treatment with RT001 (for double-blind part of the study only) 3. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed) 4. SVC

Design outcomes

Primary

Measure	Time frame
Primary objective: To evaluate the effect of RT001 on the ALSFRS-R in subjects with ALS. Primary endpoint: • Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks of the double-blind treatment period	—

Secondary

Measure	Time frame
Secondary objectives: • To evaluate the effect of RT001 on ALS clinical outcomes • To evaluate the effect of RT001 on various functional assessments, pulmonary functional assessments and motor assessments • To assess the long-term safety and tolerability of RT001 Secondary endpoints: • Time to Death or a Specified State of Disease Progression (composite of death, loss of independent ambulation, decline of ALSFRS-R from baseline of more than 6 points, loss of upper arm function, need for tracheostomy, use of respirator, or use of tube feeding) • Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) • Change from baseline in SVC Exploratory endpoints: - double bline part of the study: • Change from baseline in the Clinical Global Impression of Change (CGI-C) scale • Change from baseline in the Global Impression of Severity (CGI-S) • Change from baseline in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD) - Open-label part of the study: • Change from baseline and Day 1 in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks of the extension treatment period Safety endpoints: • Safety will be evaluated by AEs, physical examination results, vital signs, ECG findings, and clinical laboratory test results. All descriptive statistics will be presented by treatment group.	—

Measure

Time frame

Secondary objectives: • To evaluate the effect of RT001 on ALS clinical outcomes • To evaluate the effect of RT001 on various functional assessments, pulmonary functional assessments and motor assessments • To assess the long-term safety and tolerability of RT001 Secondary endpoints: • Time to Death or a Specified State of Disease Progression (composite of death, loss of independent ambulation, decline of ALSFRS-R from baseline of more than 6 points, loss of upper arm function, need for tracheostomy, use of respirator, or use of tube feeding) • Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) • Change from baseline in SVC Exploratory endpoints: - double bline part of the study: • Change from baseline in the Clinical Global Impression of Change (CGI-C) scale • Change from baseline in the Global Impression of Severity (CGI-S) • Change from baseline in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD) - Open-label part of the study: • Change from baseline and Day 1 in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks of the extension treatment period Safety endpoints: • Safety will be evaluated by AEs, physical examination results, vital signs, ECG findings, and clinical laboratory test results. All descriptive statistics will be presented by treatment group.

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)