McArdle DiseasePhase 1NCT04226274
A Study of the Safety of REN001 in Patients With McArdle Disease
A Phase 1 study testing REN001 in people with McArdle Disease, run by Reneo Pharma Ltd at 2 sites in 2 countries.
Trial summary
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- McArdle Disease
- What is being tested?
- REN001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 10 Dec 2019to 11 Oct 2021
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must give written, signed and dated informed consent
- Confirmed diagnosis of McArdle Disease
- Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes to agents that affect metabolism such as medication for diabetes
- Follow a stable dietary regimen as documented by a 3-day dietary record obtained during the screening period.
Who cannot join
- Documented history of ongoing rhabdomyolysis
- Evidence of acute crisis from their underlying disease
- Currently following or planning to start a ketogenic diet
- Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer
- Have been hospitalized within the 3 months prior to screening for any major medical condition
- Severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the Investigator in discussion with the Medical Monitor, would make the subject inappropriate for entry into this study
- Pregnant or nursing females
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United Kingdom1 site
National Hospital for Neurology and Neurosurgery, Queens Square
London, WC1 3BG
About this study
Official title: A Phase 1b, Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With McArdle Disease (Glycogen Storage Disorder 5)
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease
- Conditions
- McArdle Disease
- Being tested
- REN001
- Medical subject headings
- Glycogen Storage Disease Type V
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
REN001Drug
Once daily for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| REN001 | Experimental | Oral Receives: Drug: REN001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
Measured: up to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start10 Dec 2019
- Primary completion11 Oct 2021
- Study completion11 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for McArdle DiseaseStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Reneo Pharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rosaline Quinlivan, MD, Principal investigator, MRC Centre for Neuromuscular Diseases, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04226274
- Sponsor's protocol ID
- REN001-103
- Status last verified
- Jan 2022
- First submitted
- 9 Jan 2020
- Last updated
- 12 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With McArdle Disease (Glycogen Storage Disorder 5)
- ClinicalTrials.gov ID
- NCT04226274
- Acronym / sponsor ID
- REN001-103
- Conditions
- McArdle Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Reneo Pharma LtdIndustry (pharma, biotech, devices)
- Study start
- 10 Dec 2019 (actual)
- Primary completion
- 11 Oct 2021 (actual)
- Study completion
- 11 Oct 2021 (actual)
- First posted
- 13 Jan 2020 (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.
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| REN001 | Experimental | Drug: REN001Oral |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Subjects must give written, signed and dated informed consent
- Confirmed diagnosis of McArdle Disease
- Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes to agents that affect metabolism such as medication for diabetes
- Follow a stable dietary regimen as documented by a 3-day dietary record obtained during the screening period.
Key exclusion criteria (7 listed)
- Documented history of ongoing rhabdomyolysis
- Evidence of acute crisis from their underlying disease
- Currently following or planning to start a ketogenic diet
- Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer
- Have been hospitalized within the 3 months prior to screening for any major medical condition
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 |
|---|---|---|
| REN001Once daily for 12 weeks | Drug | REN001 |
Primary outcome
Adverse Events
Time frame: up to Week 12
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
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 12 Jan 2022.
Related trials for McArdle Disease
Active, not recruitingObservational study
Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.
Pompe Disease, McArdle Disease
Active, not recruitingObservational study
Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases
Glycogen Storage Disease, McArdle Disease
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.
Rosaline Quinlivan, MD principal investigator
MRC Centre for Neuromuscular Diseases, London
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing REN001 in people with McArdle Disease, run by Reneo Pharma Ltd at 2 sites in 2 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 4 inclusion and 7 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 12 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Spain, United Kingdom. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Adverse Events (time frame: up to Week 12). 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 12 Jan 2022.