Amyotrophic Lateral SclerosisPhase 2NCT04762589
RT001 in Amyotrophic Lateral Sclerosis
A Phase 2 study testing RT001 in people with Amyotrophic Lateral Sclerosis, run by Biojiva LLC at 4 sites in 4 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 20 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- RT001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 10 Mar 2021to 28 Jun 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
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
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
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- RT001, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RT001Drug
Also called di-deuterated linoleic acid ester
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
PlaceboDrug
Also called safflower oil
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.
| Study group | Role | What they receive |
|---|---|---|
| RT001 | Experimental | RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. Receives: Drug: RT001 |
| Placebo | Placebo comparator | Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 24 weeks
Secondary outcomes (4)
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
Measured: 24 weeks
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
Measured: 24 weeks
Change from baseline in SVC
Change from baseline in SVC will be compared for the RT001 treated group vs placebo
Measured: 24 weeks
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
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2021
- Study start10 Mar 2021
- Primary completion28 Jun 2022
- Study completion28 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biojiva LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Leonard van den Berg, MD, Principal investigator, UMC Utrecht
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04762589
- Sponsor's protocol ID
- RT001-014
- Status last verified
- Jun 2025
- First submitted
- 17 Feb 2021
- Last updated
- 1 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04762589
- Acronym / sponsor ID
- RT001-014
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Biojiva LLCIndustry (pharma, biotech, devices)
- Study start
- 10 Mar 2021 (actual)
- Primary completion
- 28 Jun 2022 (actual)
- Study completion
- 28 Jun 2022 (actual)
- First posted
- 21 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RT001 | Experimental | Drug: RT001RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboInactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. |
Who may qualify
- Ages
- 20 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RT001Also: di-deuterated linoleic acid esterRT001 8.64 g/d (3 capsules TID) for 1 month followed by 5.76 g/d (3 capsules BID) for an additional 5 months | Drug | RT001 |
| PlaceboAlso: safflower oilPlacebo Comparator: Placebo Inactive comparator capsule 960 mg/capsule: 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 20 weeks. | Drug | Placebo |
Primary outcome
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
4 secondary outcomes
Composite of Death or a Specified State of Disease Progression
Time frame: 24 weeks
Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)
Time frame: 24 weeks
Change from baseline in SVC
Time frame: 24 weeks
Frequency, severity and relationship to study drug of AEs and SAEs
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Leonard van den Berg, MD principal investigator
UMC Utrecht
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RT001 in people with Amyotrophic Lateral Sclerosis, run by Biojiva LLC at 4 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Estonia, Latvia, Netherlands, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in the revised ALS Functional Rating Score (ALSFRS-R) at 24 weeks (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2025.