RT001 in Amyotrophic Lateral Sclerosis

NCT ID: NCT04762589

Last Updated: 2025-07-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 43 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-03-10
• Study Completion Date: 2022-06-28

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

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

RT001

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

Group Type: EXPERIMENTAL

RT001

Intervention Type: 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

Placebo

Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: 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.

Interventions

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

Intervention Type: 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.

Intervention Type: DRUG

Other Intervention Names

di-deuterated linoleic acid ester; safflower oil

Eligibility Criteria

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

• Minimum Eligible Age: 20 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Biojiva LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Leonard van den Berg, MD

Role: PRINCIPAL_INVESTIGATOR

UMC Utrecht

Locations

University of Tartu

Tartu, , Estonia

Riga Stradins Universtiy

Riga, , Latvia

UMC Utrecht

Utrecht, , Netherlands

Karolinska

Stockholm, , Sweden

Countries

Estonia; Latvia; Netherlands; Sweden

Other Identifiers

RT001-014

Identifier Type: -

Identifier Source: org_study_id