Clinical trial NCT04762589: RT001 in Amyotrophic Lateral Sclerosis
Official title: 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 type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Biojiva LLC
Sponsor class: INDUSTRY
First posted: 2021-02-21
Enrollment: 40
Sex: ALL
Minimum age (years): 20
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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

Detailed description: 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.

Eligibility 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

Primary outcomes:
measure: Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks
timeFrame: 24 weeks
Secondary outcomes:
measure: Composite of Death or a Specified State of Disease Progression
timeFrame: 24 weeks
measure: Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)
timeFrame: 24 weeks
measure: Change from baseline in SVC
timeFrame: 24 weeks
measure: Frequency, severity and relationship to study drug of AEs and SAEs
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Tartu
country: Estonia
facility: University of Tartu
city: Riga
country: Latvia
facility: Riga Stradins Universtiy
city: Utrecht
country: Netherlands
facility: UMC Utrecht
city: Stockholm
country: Sweden
facility: Karolinska
Study officials:
affiliation: UMC Utrecht
name: Leonard van den Berg, MD
role: PRINCIPAL_INVESTIGATOR
References: