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 SclerosisStudy Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
RT001, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Leonard van den Berg, MD, UMC Utrecht
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Mark G Midei, MD / email hidden; JavaScript is required / +1 (410) 371-5357
Full Study Summary:
Study Sponsor:
Retrotope, Inc.
Estimated Enrollment:
40
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
Tartu
Estonia
Riga Stradins Universtiy | Recruiting
Principal Investigator : Viktorija Kenina
Riga
Latvia
UMC Utrecht | Recruiting
Leonard van den Berg
Principal Investigator : Leonard van den Berg
Utrecht
Netherlands
Karolinska | Recruiting
Principal Investigator : Caroline Ingre
Stockholm
Sweden