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RT001 in Amyotrophic Lateral Sclerosis

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Unknown status

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

RT001

Placebo

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

20 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female subject with age 20 years to 75 years at the time of signed consent
Patients who are defined as "definite ALS," "probable ALS" or "probable-laboratory-supported ALS," met diagnostic criteria revised EL Escorial for Airlie House.
ALSFRS-R > 20
Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life
Patients of less than 3 years after the onset of ALS
Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend)
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:

Received treatment with other experimental therapies within the last 30 days prior to the first dose
Previously received treatment with RT001
Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
SVC < 70 at screening
Subject has a feeding tube or the need for a feeding tube is anticipated within the first 24 weeks after enrollment
Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period
Evidence of any clinically significant neurological disorder other than ALS
The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria
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
Subject has had a significant illness or infection requiring medical intervention in the past 30 days
Female who is breastfeeding or has a positive pregnancy test
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
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
History, within the last 2 years, of alcohol abuse or physical opioid dependence

Sites / Locations

University of TartuRecruiting
Riga Stradins UniverstiyRecruiting
UMC UtrechtRecruiting
KarolinskaRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

RT001

Placebo

Arm Description

RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.

Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.

Outcomes

Primary Outcome Measures

Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks

Change from baseline in ALSFRS-R will be compared for the RT001 treated group vs placebo

Secondary Outcome Measures

Composite of Death or a Specified State of Disease Progression

Change from baseline in the composite endpoint will be compared for the RT001 treated group vs placebo

Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)

Change from baseline in ALSAQ40 will be compared for the RT001 treated group vs placebo

Change from baseline in SVC

Change from baseline in SVC will be compared for the RT001 treated group vs placebo

Frequency, severity and relationship to study drug of AEs and SAEs

Frequency, severity and relationship to study drug of AEs and SAEs will be compared for the RT001 treated group vs placebo

Full Information

NCT ID

NCT04762589

First Posted

February 17, 2021

Last Updated

April 7, 2022

Sponsor

Retrotope, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04762589

Brief Title

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

2. Study Status

Record Verification Date

June 2021

Overall Recruitment Status

Unknown status

Study Start Date

March 10, 2021 (Actual)

Primary Completion Date

August 30, 2022 (Anticipated)

Study Completion Date

September 30, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Retrotope, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Yes

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Masking Description

Placebo-controlled, double-blind

Allocation

Randomized

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

RT001

Arm Type

Experimental

Arm Description

RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks.

Intervention Type

Drug

Intervention Name(s)

RT001

Other Intervention Name(s)

di-deuterated linoleic acid ester

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

safflower oil

Intervention Description

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.

Primary Outcome Measure Information:

Title

Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks

Description

Change from baseline in ALSFRS-R will be compared for the RT001 treated group vs placebo

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

Composite of Death or a Specified State of Disease Progression

Description

Change from baseline in the composite endpoint will be compared for the RT001 treated group vs placebo

Time Frame

24 weeks

Title

Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)

Description

Change from baseline in ALSAQ40 will be compared for the RT001 treated group vs placebo

Time Frame

24 weeks

Title

Change from baseline in SVC

Description

Change from baseline in SVC will be compared for the RT001 treated group vs placebo

Time Frame

24 weeks

Title

Frequency, severity and relationship to study drug of AEs and SAEs

Description

Frequency, severity and relationship to study drug of AEs and SAEs will be compared for the RT001 treated group vs placebo

Time Frame

24 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

20 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male or female subject with age 20 years to 75 years at the time of signed consent Patients who are defined as "definite ALS," "probable ALS" or "probable-laboratory-supported ALS," met diagnostic criteria revised EL Escorial for Airlie House. ALSFRS-R > 20 Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life Patients of less than 3 years after the onset of ALS Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend) 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: Received treatment with other experimental therapies within the last 30 days prior to the first dose Previously received treatment with RT001 Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed) SVC < 70 at screening Subject has a feeding tube or the need for a feeding tube is anticipated within the first 24 weeks after enrollment Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period Evidence of any clinically significant neurological disorder other than ALS The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria 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 Subject has had a significant illness or infection requiring medical intervention in the past 30 days Female who is breastfeeding or has a positive pregnancy test 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 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 History, within the last 2 years, of alcohol abuse or physical opioid dependence

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Mark G Midei, MD

Phone

+1 (410) 371-5357

mark@retrotope.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Leonard van den Berg, MD

Organizational Affiliation

UMC Utrecht

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Tartu

City

Tartu

Country

Estonia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Karin Rallmann

Facility Name

Riga Stradins Universtiy

City

Riga

Country

Latvia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Viktorija Kenina

Facility Name

UMC Utrecht

City

Utrecht

Country

Netherlands

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Leonard van den Berg

First Name & Middle Initial & Last Name & Degree

Leonard van den Berg

Facility Name

Karolinska

City

Stockholm

Country

Sweden

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Caroline Ingre

12. IPD Sharing Statement

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RT001 in Amyotrophic Lateral Sclerosis

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