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
- Conditions
- Amyotrophic Lateral SclerosisMedDRA version: 21.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2020-003962-38-EE
- Lead Sponsor
- Retrotope, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 44
1. Male or female subject with age 20 years to 75 years at the time of signed consent for double-blind part of the study
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. ALS-FRS > 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 14
1. Received treatment with other experimental therapies within the last 30 days prior to the first dose (for double-blind part of the study only)
2. Previously received treatment with RT001 (for double-blind part of the study only)
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 for the duration of the trial.
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
10. Subject has had a significant illness or infection requiring medical intervention in the past 30 days (for double-blind part of the study only)
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 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 & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Primary objective is to evaluate the effect of RT001 on the ALSFRS-R in subjects with ALS.<br>;Secondary Objective: Secondary Objectives:<br>• To evaluate the effect of RT001 on ALS clinical outcomes<br>• To evaluate the effect of RT001 on various functional assessments, pulmonary functional assessments and motor assessments<br>• To assess the long-term safety and tolerability of RT001<br>;Primary end point(s): Primary endpoints:<br>Change from baseline in the ALSFRS-R total score at 24 weeks of the double-blind treatment period<br><br>;Timepoint(s) of evaluation of this end point: 24 weeks (Day 168) of the double-blind treatment period <br>
- Secondary Outcome Measures
Name Time Method