Progressive Supranuclear PalsyPhase 2NCT04937530
RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY
A Phase 2 study testing RT001 in people with Progressive Supranuclear Palsy, run by Biojiva LLC at 1 site in Germany.
Trial summary
This is a randomized, placebo-controlled trial of RT001 in patients with PSP. Read more about this study
- Who is it for?
- Ages 40 to 80 years
- Conditions the study is for
- Progressive Supranuclear Palsy
- 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
- 40 participants
- Where
- 1 siteGermany
- Study dates
- 23 Jun 2021to 30 Aug 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 40 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Sign the informed consent form prior to entry into the study
- Male or female subject with age 40 years to 80 years at the time of signed consent
- Meets the MDS-PSP study group criteria for possible or probable progressive supranuclear palsy with Richardson syndrome (postural instability and falls with vertical ocular motor dysfunction)
- Presence of PSP symptoms for less than 4 years
- Score of <40 on the PSPRS-28
- Ambulatory patients (with or without assistive device - no handheld help) and capable of performing study assessments/evaluations
- Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend) who can assist in assuring that the subject is able to travel to the required visits.
- Willing to provide the necessary blood samples
Who cannot join
- Received treatment with other experimental therapies within the last 30 days prior to the first dose. The last dose of the prior experimental agent must have occurred more than 5 half-lives prior to enrollment in the current trial.
- Previously received treatment with RT001
- Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening/Baseline till last study procedure completed)
- Mini mental state examination (MMSE) score less than 20 at screening
- 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 PSP in particular CBS
Show all 16Show fewer
- Evidence of a clear and robust benefit from levodopa at the time of screening. Participants are permitted to take levodopa and other Parkinson's medications if the dose had been stable for 60 days prior to screening
- The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria
- Subject has had a significant illness or infection requiring medical intervention in the past 30 days
- Subject has evidence of any disease or condition (based on either history, physical or laboratory values) that might interfere with the conduct of the study
- Is currently receiving active deep brain stimulation (DBS) that cannot be turned off
- Any condition with a life expectancy of less than 2 years
- 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 highly effective 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
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
Agaharied Teaching Hospital, University of Munich
Munich, MD, 21111
Mark G. Midei, MD, mark@retrotope.com
About this study
Official title: A Randomized, Double-blind, Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Progressive Supranuclear Palsy
This is a randomized, placebo-controlled trial of RT001 in patients with PSP.
Read the detailed description
This is a randomized, placebo-controlled trial of RT001 in patients with PSP to assess the efficacy, safety and tolerability of RT001. Subjects will be randomized to RT001 or placebo and will receive study drug for 48 weeks.
- Conditions
- Progressive Supranuclear Palsy
- Being tested
- RT001, Placebo
- Medical subject headings
- Supranuclear Palsy, Progressive
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 11 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 44 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 44 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 44 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 PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group
The PSPRS-28 is a clinician-rated instrument to assess disability and severity of PSP. The PSPRS-28 evaluates 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline.
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2021
- Study start23 Jun 2021
- Primary completion30 Jul 2022 (estimated)
- Study completion30 Aug 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Progressive Supranuclear PalsyStudies 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
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biojiva LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stefan Lorenzl, MD, PhD, Principal investigator, Ludwig-Maximilians - University of Munich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04937530
- Sponsor's protocol ID
- RT001-013
- Status last verified
- Jun 2021
- First submitted
- 16 Jun 2021
- Last updated
- 25 Jun 2021
- 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 25 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Progressive Supranuclear Palsy
- ClinicalTrials.gov ID
- NCT04937530
- Acronym / sponsor ID
- RT001-013
- Conditions
- Progressive Supranuclear Palsy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Biojiva LLCIndustry (pharma, biotech, devices)
- Study start
- 23 Jun 2021 (actual)
- Primary completion
- 30 Jul 2022 (estimated)
- Study completion
- 30 Aug 2022 (estimated)
- First posted
- 24 Jun 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.
This is a randomized, placebo-controlled trial of RT001 in patients with PSP.
Detailed description
This is a randomized, placebo-controlled trial of RT001 in patients with PSP to assess the efficacy, safety and tolerability of RT001. Subjects will be randomized to RT001 or placebo and will receive study drug for 48 weeks.
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 44 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 44 weeks. |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Sign the informed consent form prior to entry into the study
- Male or female subject with age 40 years to 80 years at the time of signed consent
- Meets the MDS-PSP study group criteria for possible or probable progressive supranuclear palsy with Richardson syndrome (postural instability and falls with vertical ocular motor dysfunction)
- Presence of PSP symptoms for less than 4 years
- Score of <40 on the PSPRS-28
Key exclusion criteria (16 listed)
- Received treatment with other experimental therapies within the last 30 days prior to the first dose. The last dose of the prior experimental agent must have occurred more than 5 half-lives prior to enrollment in the current trial.
- Previously received treatment with RT001
- Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening/Baseline till last study procedure completed)
- Mini mental state examination (MMSE) score less than 20 at screening
- Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period
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 11 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 44 weeks. | Drug | Placebo |
Primary outcome
Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group
Time frame: 48 weeks
The PSPRS-28 is a clinician-rated instrument to assess disability and severity of PSP. The PSPRS-28 evaluates 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2021.
- 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.
Stefan Lorenzl, MD, PhD principal investigator
Ludwig-Maximilians - University of Munich
1 site in 1 country: 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 Progressive Supranuclear Palsy, run by Biojiva LLC at 1 site in Germany.
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 8 inclusion and 16 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: status not confirmed, as of the sponsor's last update on 25 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mark G. Midei, MD ((650) 437-0700, mark@retrotope.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group (time frame: 48 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 25 Jun 2021.