RT001 in Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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RT001-014 is 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

Detailed Description
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Forty subjects at 4 EU sites will undergo baseline examination, and then be randomized 1:1 to receive either RT001 or placebo. Repeat visits will occur every 2 months. The final visit will be at 6 months.

Official Title
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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

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: RT001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female subject with age 20 years to 75 years at the time of signed consent Patients who are defined as definite ALS, probable ALS or probable-laboratory-supported ALS, met diagnostic criteria revised EL Escorial for Airlie House. ALSFRS-R > 20 Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life Patients of less than 3 years after the onset of ALS Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend) 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: Received treatment with other experimental therapies within the last 30 days prior to the first dose Previously received treatment with RT001 Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed) SVC < 70 at screening Subject has a feeding tube or the need for a feeding tube is anticipated within the first 24 weeks after enrollment Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period Evidence of any clinically significant neurological disorder other than ALS The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria The subject has a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function Subject has had a significant illness or infection requiring medical intervention in the past 30 days Female who is breastfeeding or has a positive pregnancy test Male participant or female participant of childbearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier birth control method throughout the study Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled History, within the last 2 years, of alcohol abuse or physical opioid dependence

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RT001<br>RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. | Drug: RT001<br>* RT001 8.64 g/d (3 capsules TID) for 1 month followed by 5.76 g/d (3 capsules BID) for an additional 5 months<br>* Other names: di-deuterated linoleic acid ester;|
| Placebo Comparator: Placebo<br>Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. | Drug: Placebo<br>* Placebo Comparator: Placebo Inactive comparator capsule 960 mg/capsule: 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.<br>* Other names: safflower oil;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks | Change from baseline in ALSFRS-R will be compared for the RT001 treated group vs placebo | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Composite of Death or a Specified State of Disease Progression | Change from baseline in the composite endpoint will be compared for the RT001 treated group vs placebo | 24 weeks | 
| Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) | Change from baseline in ALSAQ40 will be compared for the RT001 treated group vs placebo | 24 weeks | 
| Change from baseline in SVC | Change from baseline in SVC will be compared for the RT001 treated group vs placebo | 24 weeks | 
| Frequency, severity and relationship to study drug of AEs and SAEs | Frequency, severity and relationship to study drug of AEs and SAEs will be compared for the RT001 treated group vs placebo | 24 weeks |