Overview

RT001 in Amyotrophic Lateral Sclerosis

Status:
Recruiting

Trial end date:
2021-09-30

Target enrollment:
0

Participant gender:
All

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

Phase:

Phase 2

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Retrotope, Inc.

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