RT001 in Amyotrophic Lateral Sclerosis

NCT04762589 | Completed Phase 2 FDA Drug

• 1 other identifier: RT001-014
• Study Type: interventional
• Target: 43
• Locations: 4 countries
• Sites: 4

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Composite of Death or a Specified State of Disease Progression

  24 weeks

• Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)

  24 weeks

• Change from baseline in SVC

  24 weeks

• Frequency, severity and relationship to study drug of AEs and SAEs

  24 weeks

Study Arms (2)

RT001

EXPERIMENTAL

RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.

Drug: RT001

Placebo

PLACEBO COMPARATOR

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

Interventions

RT001 DRUG

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

Also known as: di-deuterated linoleic acid ester

RT001

Placebo DRUG

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.

Also known as: safflower oil

Placebo

Eligibility Criteria

• Age: 20 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Biojiva LLC: lead

Study Sites (4)

University of Tartu

Tartu, Estonia

Location

Riga Stradins Universtiy

Riga, Latvia

Location

UMC Utrecht

Utrecht, Netherlands

Location

Karolinska

Stockholm, Sweden

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

RT001; Safflower Oil

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Dietary Fats, Unsaturated; Dietary Fats; Fats; Lipids; Fats, Unsaturated; Plant Oils; Oils; Plant Preparations; Biological Products; Complex Mixtures; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Study Officials

• Leonard van den Berg, MD

  UMC Utrecht

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Placebo-controlled, double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2021
• First Posted: February 21, 2021
• Study Start: March 10, 2021
• Primary Completion: June 28, 2022
• Study Completion: June 28, 2022
• Last Updated: July 1, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1); LA (1); SE (1); NL (1)