RT001 in 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

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

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: RT001; Drug: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 43
• Phase: Phase 2

Contacts and Locations

Study Locations

• Estonia
  • Tartu, Estonia
    • University of Tartu
• Latvia
  • Riga, Latvia
    • Riga Stradins Universtiy
• Netherlands
  • Utrecht, Netherlands
    • UMC Utrecht
• Sweden
  • Stockholm, Sweden
    • Karolinska

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 71 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female subject with age 20 years to 75 years at the time of signed consent
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:

1. Received treatment with other experimental therapies within the last 30 days prior to the first dose
2. Previously received treatment with RT001
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 < 70 at screening
5. Subject has a feeding tube or the need for a feeding tube is anticipated within the first 24 weeks after enrollment
6. Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period
7. Evidence of any clinically significant neurological disorder other than ALS
8. The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria
9. 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
10. Subject has had a significant illness or infection requiring medical intervention in the past 30 days
11. Female who is breastfeeding or has a positive pregnancy test
12. 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
13. 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
14. History, within the last 2 years, of alcohol abuse or physical opioid dependence

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RT001; RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.	Drug: RT001; 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; Other Names: di-deuterated linoleic acid ester
Placebo Comparator: Placebo; 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; 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.; Other Names: safflower oil

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Biojiva LLC
• Investigators: Principal Investigator: Leonard van den Berg, MD, UMC Utrecht

Study record dates

Study Major Dates

• Study Start (Actual): March 10, 2021
• Primary Completion (Actual): June 28, 2022
• Study Completion (Actual): June 28, 2022

Study Registration Dates

• First Submitted: February 17, 2021
• First Submitted That Met QC Criteria: February 17, 2021
• First Posted (Actual): February 21, 2021

Study Record Updates

• Last Update Posted (Estimated): July 1, 2025
• Last Update Submitted That Met QC Criteria: June 27, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: RT001-014

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes