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RT001 in Amyotrophic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT04762589
Recruitment Status : Recruiting
First Posted : February 21, 2021
Last Update Posted : June 14, 2021
Sponsor:
Information provided by (Responsible Party):
Retrotope, Inc.

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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: RT001 Drug: Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Placebo-controlled, double-blind
Primary Purpose: Treatment
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
Actual Study Start Date : March 10, 2021
Estimated Primary Completion Date : September 30, 2021
Estimated Study Completion Date : September 30, 2021


Arm Intervention/treatment
Experimental: RT001
RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.
Drug: RT001
RT001 8.64 g/d (3 capsules TID) for 1 month followed by 5.76 g/d (3 capsules BID) for an additional 5 months
Other Name: di-deuterated linoleic acid ester

Placebo Comparator: Placebo
Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.
Drug: Placebo
Placebo Comparator: Placebo Inactive comparator capsule 960 mg/capsule: 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.
Other Name: safflower oil




Primary Outcome Measures :
  1. Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks [ Time Frame: 24 weeks ]
    Change from baseline in ALSFRS-R will be compared for the RT001 treated group vs placebo


Secondary Outcome Measures :
  1. Composite of Death or a Specified State of Disease Progression [ Time Frame: 24 weeks ]
    Change from baseline in the composite endpoint will be compared for the RT001 treated group vs placebo

  2. Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) [ Time Frame: 24 weeks ]
    Change from baseline in ALSAQ40 will be compared for the RT001 treated group vs placebo

  3. Change from baseline in SVC [ Time Frame: 24 weeks ]
    Change from baseline in SVC will be compared for the RT001 treated group vs placebo

  4. Frequency, severity and relationship to study drug of AEs and SAEs [ Time Frame: 24 weeks ]
    Frequency, severity and relationship to study drug of AEs and SAEs will be compared for the RT001 treated group vs placebo



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Ages Eligible for Study:   20 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04762589


Contacts
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Contact: Mark G Midei, MD +1 (410) 371-5357 mark@retrotope.com

Locations
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Estonia
University of Tartu Recruiting
Tartu, Estonia
Principal Investigator: Karin Rallmann         
Latvia
Riga Stradins Universtiy Recruiting
Riga, Latvia
Principal Investigator: Viktorija Kenina         
Netherlands
UMC Utrecht Recruiting
Utrecht, Netherlands
Contact: Leonard van den Berg         
Principal Investigator: Leonard van den Berg         
Sweden
Karolinska Recruiting
Stockholm, Sweden
Principal Investigator: Caroline Ingre         
Sponsors and Collaborators
Retrotope, Inc.
Investigators
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Principal Investigator: Leonard van den Berg, MD UMC Utrecht
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Responsible Party: Retrotope, Inc.
ClinicalTrials.gov Identifier: NCT04762589    
Other Study ID Numbers: RT001-014
First Posted: February 21, 2021    Key Record Dates
Last Update Posted: June 14, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases