Amyotrophic Lateral Sclerosis MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Retrotope, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary objective is to evaluate the effect of RT001 on the ALSFRS-R in subjects with ALS. ;Secondary Objective: Secondary Objectives: • To evaluate the effect of RT001 on ALS clinical outcomes • To evaluate the effect of RT001 on various functional assessments, pulmonary functional assessments and motor assessments • To assess the long-term safety and tolerability of RT001 ;Primary end point(s): Primary endpoints: Change from baseline in the ALSFRS-R total score at 24 weeks of the double-blind treatment period ;Timepoint(s) of evaluation of this end point: 24 weeks (Day 168) of the double-blind treatment period | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints are: - Time to Death or a Specified State of Disease Progression (composite of death, loss of independent ambulation, decline of ALSFRS-R from baseline of more than 6 points, loss of upper arm function, need for tracheostomy, use of respirator, or use of tube feeding) - Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) - Change from baseline in SVC Exploratory endpoints Double-blind part of the study: - Change from Baseline in the Clinical Global Impression of Change (CGI-C) scale - Change from Baseline in the Clinical Global Impression of Severity (CGI-S) - Change from Baseline in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD) Open-label part of the study: Change from baseline in ALSFRS-R at 24 weeks of the extension treatmen period Safety endpoints - Safety will be evaluated by AEs, physical examination results, vital signs, ECG findings, and clinical laboratory test results. All descriptive statistics will be presented by treatment group. ;Timepoint(s) of evaluation of this end point: 24 weeks (Day 168) 24 weeks (Day 168) of the double-blind treatment period and at 24 weeks of the extension treatment period | — |
Countries
Estonia, Netherlands, Sweden
Contacts
Public ContactChief Medical Officer
Retrotope Inc.
Outcome results
None listed