Fatty Acid Oxidation DisordersPhase 1NCT03833128
A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders
A Phase 1 study testing Low Dose REN001 and High Dose REN001 in people with Fatty Acid Oxidation Disorders, run by Reneo Pharma Ltd at 7 sites in 3 countries.
Trial summary
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fatty Acid Oxidation Disorders
- What is being tested?
- Low Dose REN001 and High Dose REN001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 3 countries
- Study dates
- 4 Apr 2019to 21 Mar 2022
- 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 FAOD
- A diagnostic acylcarnitine profile, in blood or cultured fibroblasts
- A stable treatment regimen for at least 30 days prior to enrollment
Who cannot join
- Unstable or poorly controlled disease
- Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer
- Have been hospitalized within 3 months prior to screening for any major medical event
- Pregnant or nursing females
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
United States5 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
UT Southwestern Medical Center
Dallas, Texas, 75390-8591
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
Division of Medical Genetics, University Utah
Salt Lake City, Utah, 84108
France1 site
Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center
Garches, 92380
Spain1 site
Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12
Madrid, CP 28041
About this study
Official title: An Open Label Study to Determine the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Subjects With Fatty Acid Oxidation Disorders (FAOD)
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
Read the detailed description
This is a Phase 1b, open-label, multiple-dose study of the safety and tolerability of 2 dose levels of REN001 in subjects with fatty acid oxidation disorders (FAODs) with confirmed mutations in the Carnitine palmitoyltransferase II deficiency (CPT2), Very long-chain Acyl-CoA dehydrogenase deficiency (VLCAD), Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHAD) or Trifunctional Protein Deficiency (TFP). All subjects will provide written consent prior to commencing any study related activities or assessments. Potential subjects will be screened for study participation up to 8 weeks prior to the start of dosing.The study is divided into two parts, Part A and Part B. Part A has finished enrollment and further eligible patients will participate in Part B only.
- Conditions
- Fatty Acid Oxidation Disorders
- Being tested
- Low Dose REN001, High Dose REN001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low Dose REN001Drug
Oral
High Dose REN001Drug
Oral
| Study group | Role | What they receive |
|---|---|---|
| Group 1 - Part A | Experimental | REN001 Low Dose oral once daily x 12 weeks Receives: Drug: Low Dose REN001 |
| Group 2 - Part A | Experimental | REN001 High Dose oral once daily x 12 weeks Receives: Drug: High Dose REN001 |
| Group 3 - Part B | Experimental | REN001 High Dose oral once daily x 12 weeks Receives: Drug: High Dose 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: Continous 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 posted6 Feb 2019
- Study start4 Apr 2019
- Primary completion24 Jan 2022
- Study completion21 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fatty Acid Oxidation DisordersStudies 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Reneo Pharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jerry Vockley, M.D., Ph.D., Principal investigator, University of Pittsburgh Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03833128
- Sponsor's protocol ID
- REN001-102
- Status last verified
- Dec 2022
- First submitted
- 4 Feb 2019
- Last updated
- 9 Dec 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study to Determine the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Subjects With Fatty Acid Oxidation Disorders (FAOD)
- ClinicalTrials.gov ID
- NCT03833128
- Acronym / sponsor ID
- REN001-102
- Conditions
- Fatty Acid Oxidation Disorders
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Reneo Pharma LtdIndustry (pharma, biotech, devices)
- Study start
- 4 Apr 2019 (actual)
- Primary completion
- 24 Jan 2022 (actual)
- Study completion
- 21 Mar 2022 (actual)
- First posted
- 6 Feb 2019 (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 Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
Detailed description
This is a Phase 1b, open-label, multiple-dose study of the safety and tolerability of 2 dose levels of REN001 in subjects with fatty acid oxidation disorders (FAODs) with confirmed mutations in the Carnitine palmitoyltransferase II deficiency (CPT2), Very long-chain Acyl-CoA dehydrogenase deficiency (VLCAD), Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHAD) or Trifunctional Protein Deficiency (TFP). All subjects will provide written consent prior to commencing any study related activities or assessments. Potential subjects will be screened for study participation up to 8 weeks prior to the start of dosing.The study is divided into two parts, Part A and Part B. Part A has finished enrollment and further eligible patients will participate in Part B only.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 - Part A | Experimental | Drug: Low Dose REN001REN001 Low Dose oral once daily x 12 weeks |
| Group 2 - Part A | Experimental | Drug: High Dose REN001REN001 High Dose oral once daily x 12 weeks |
| Group 3 - Part B | Experimental | Drug: High Dose REN001REN001 High Dose oral once daily x 12 weeks |
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 FAOD
- A diagnostic acylcarnitine profile, in blood or cultured fibroblasts
- A stable treatment regimen for at least 30 days prior to enrollment
Key exclusion criteria (4 listed)
- Unstable or poorly controlled disease
- Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer
- Have been hospitalized within 3 months prior to screening for any major medical event
- Pregnant or nursing females
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 |
|---|---|---|
| Low Dose REN001Oral | Drug | Group 1 - Part A |
| High Dose REN001Oral | Drug | Group 2 - Part A, Group 3 - Part B |
Primary outcome
Adverse Events
Time frame: Continous 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 9 Dec 2022.
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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.
Jerry Vockley, M.D., Ph.D. principal investigator
University of Pittsburgh Medical Center
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Low Dose REN001 and High Dose REN001 in people with Fatty Acid Oxidation Disorders, run by Reneo Pharma Ltd at 7 sites in 3 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 4 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 9 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, France, Spain. 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: Continous 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 9 Dec 2022.