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 Purpose:

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 Status:



Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

RT001, Placebo




Phase 2

Study Chair(s)/Principal Investigator(s):

Leonard van den Berg, MD, UMC Utrecht

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Mark G Midei, MD / email hidden; JavaScript is required / +1 (410) 371-5357

Full Study Summary:

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 Sponsor:

Retrotope, Inc.

Estimated Enrollment:


Estimated Study Start Date:

03 / 10 / 2021

Estimated Study Completion Date:

09 / 30 / 2022

Posting Last Modified Date:

04 / 08 / 2022

Date Study Added to neals.org:

02 / 21 / 2021

Minimum Age:

20 Years

Maximum Age:

75 Years

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

University of Tartu | Recruiting

Principal Investigator : Karin Rallmann


Riga Stradins Universtiy | Recruiting

Principal Investigator : Viktorija Kenina


UMC Utrecht | Recruiting

Leonard van den Berg

Principal Investigator : Leonard van den Berg


Karolinska | Recruiting

Principal Investigator : Caroline Ingre