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CompletedNCT04762589Updated Jul 1, 2025

RT001 in Amyotrophic Lateral Sclerosis

A Phase 2 interventional study of RT001 and Placebo in Amyotrophic Lateral Sclerosis, sponsored by Biojiva LLC. Completed at 4 sites in 4 countries. Open to participants aged 20 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-07-01.

Sponsored by Biojiva LLC · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Jun 2022, 4 years 4 months ago, and no results have been posted to the registry.
Phase
Phase 2
Study type
Interventional
Enrollment
43
Allocation
Randomized
Ages
20 Years to 75 Years
Sex
All
01

Study 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 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.

02

Conditions studied

  • Amyotrophic Lateral Sclerosis
03

In context

Amyotrophic Lateral Sclerosis

978 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 277 are open to participants now.

This study's enrollment of 43 is above the median of 38 across 662 interventional studies indexed under Amyotrophic Lateral Sclerosis.

Browse Amyotrophic Lateral Sclerosis studies →

Lead sponsor

Biojiva LLC is the lead sponsor of 2 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
20 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 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

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 neurological disorder other than ALS
  8. The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to 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 , who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier 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
Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

DSM diagnostic criteria

The American Psychiatric Association’s manual of mental health diagnoses. DSM-5 (2013, revised as DSM-5-TR in 2022) replaced DSM-IV, and some studies still name the older edition. “Meets DSM criteria for …” means diagnosed by that edition’s diagnostic criteria, usually by a clinician in an interview.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
43 participants (actual)

Study arms

  • Experimental
    RT001

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

    Drug: RT001

  • 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

Interventions

  • DrugRT001

    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

    Also known as: di-deuterated linoleic acid ester

  • DrugPlacebo

    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.

    Also known as: safflower oil

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 24 weeks

Secondary outcomes

  1. 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

    Time frame: 24 weeks

  2. 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

    Time frame: 24 weeks

  3. Change from baseline in SVC

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

    Time frame: 24 weeks

  4. 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

    Time frame: 24 weeks

07

Study locations

4 sites
  • University of Tartu
    Tartu, Estonia
  • Riga Stradins Universtiy
    Riga, Latvia
  • UMC Utrecht
    Utrecht, Netherlands
  • Karolinska
    Stockholm, Sweden
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Mar 2021 to primary completion in Jun 2022, 15 months.

Of 36 industry-sponsored phase 2 interventional studies under Amyotrophic Lateral Sclerosis, half reached primary completion within 21 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 35. This study's 15 months is below that.

Counting every sponsor, half of 73 studies reached it within 24 months; a quarter within 16 and three quarters within 35.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 17, 2021
First posted
Feb 21, 2021
StartActual
Mar 10, 2021
Primary completionActual
Jun 28, 2022
CompletionActual
Jun 28, 2022
Last update
Jul 1, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jul 1, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04762589
Lead sponsor
Biojiva LLC
Responsible party
Sponsor
First posted
Feb 21, 2021
Start date
Mar 10, 2021
Primary completion
Jun 28, 2022
Completion
Jun 28, 2022
Last update
Jul 1, 2025

Study contacts

Leonard van den Berg, MD
principal investigator · UMC Utrecht

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Studies of the drug tested here: RT001

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