Fatty Acid Oxidation DisorderFORWARDNCT04482049
A Prospective Study of the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders
An observational study of Fatty Acid Oxidation Disorder, run by Reneo Pharma Ltd at 15 sites in 11 countries.
Trial summary
The purpose of the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fatty Acid Oxidation Disorder
- How many people
- 61 participants
- Where
- 15 sitesin 11 countries
- Study dates
- 15 Jan 2021to 4 May 2022
- Phase
- Observational
- 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
Study population: Patients will be recruited from centres experienced in the identification and subsequent management of patients with long chain FAOD.
Who may be able to join
- A genetically confirmed diagnosis of one of the following:
- Carnitine palmitoyltransferase 2 deficiency
- Very long-chain Acyl-CoA dehydrogenase deficiency
- Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
- Trifunctional protein deficiency
- A stable treatment regimen for at least 30 days
- Ambulatory and able to perform the study exercise test, using walking aids if necessary
- Willing and able to personally sign and date an informed consent document indicating that the subject has been informed of all pertinent aspects of the study
Who cannot join
- Unstable or poorly controlled disease as determined by one or more of the following:
- Presence of symptoms of acute rhabdomyolysis with clinically significant elevations in serum CK
- Evidence of acute crisis from their underlying disease
- Currently taking a PPAR agonist
- Have motor abnormalities other than those related to the fatty acid oxidation disorder that could interfere with the study procedures, as determine by the investigator
- Evidence of significant concomitant medical or psychiatric disease that in the opinion of the Investigator may interfere with the conduct or safety of this study
- Pregnant or nursing females
Trial locations and contacts
15 sites in 11 countries.
15 sites in 11 countries
United States3 sites
Vanderbuilt University Medical Center
Nashville, Tennessee, 37232
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
Oregon Health & Science University (OHSU)
Portland, Oregon, 97239
Italy2 sites
Universita di Brescia
Brescia, 25123
Azienda Ospedaliera Universitaria Policlinico "G. Martino" di Messina
Messina, 98124
Spain2 sites
Hospital 12 de Octubre
Madrid, 28041
Unidad de Diagnóstico y Tratamiento de Enfermedades Metabólicas Complejo Hospitalario Universitario de Santiago Travesía de Choupana s/n
Santiago de Compostela, 15706
Czechia1 site
Vseobecna fakultni nemocnice v Praze, Klinika pediatrie a dedicnych poruch metabolizmu
Prague, 12808
Denmark1 site
Rigshospitalet, Klinik for nerve- og muskelsygdomme
Copenhagen, 2100
About this study
Official title: A Prospective, Multicenter, Non-interventional Study to Investigate the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders (FAOD)
The purpose of the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD).
Read the detailed description
In this study there will be no drug intervention. The study will include a Baseline visit and a follow up visit scheduled at Month 4. At these visits medical history, safety assessments, concomitant medications, exercise tests and quality of life questionnaire data will be collected.
- Conditions
- Fatty Acid Oxidation Disorder
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate change in function as measured by 12 minute walk test in adult subjects with long chain FAOD
Change from baseline in 12MWT
Measured: Week 16
Secondary outcomes (1)
To evaluate change in symptoms related to FAOD using a newly developed muscle symptom questionnaire specifically designed for adult patients with long chain FAOD
Change from baseline in FAOD-Muscle Symptom Inventory
Measured: Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2020
- Study start15 Jan 2021
- Primary completion4 May 2022
- Study completion4 May 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 DisorderStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Reneo Pharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Melanie Gillingham, PhD, RD, LD, Principal investigator, Dept of Molecular and Medical Genetics, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04482049
- Sponsor's protocol ID
- REN001-903
- Acronym
- FORWARD
- Status last verified
- Nov 2023
- First submitted
- 14 Jul 2020
- Last updated
- 18 Nov 2023
- 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 18 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Non-interventional Study to Investigate the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders (FAOD)
- ClinicalTrials.gov ID
- NCT04482049
- Acronym / sponsor ID
- FORWARD / REN001-903
- Conditions
- Fatty Acid Oxidation Disorder
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Reneo Pharma LtdIndustry (pharma, biotech, devices)
- Study start
- 15 Jan 2021 (actual)
- Primary completion
- 4 May 2022 (actual)
- Study completion
- 4 May 2022 (actual)
- First posted
- 22 Jul 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 the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD).
Detailed description
In this study there will be no drug intervention. The study will include a Baseline visit and a follow up visit scheduled at Month 4. At these visits medical history, safety assessments, concomitant medications, exercise tests and quality of life questionnaire data will be collected.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients will be recruited from centres experienced in the identification and subsequent management of patients with long chain FAOD.
Key inclusion criteria (8 listed)
- A genetically confirmed diagnosis of one of the following:
- A stable treatment regimen for at least 30 days
- Ambulatory and able to perform the study exercise test, using walking aids if necessary
- Willing and able to personally sign and date an informed consent document indicating that the subject has been informed of all pertinent aspects of the study
Key exclusion criteria (7 listed)
- Unstable or poorly controlled disease as determined by one or more of the following:
- Currently taking a PPAR agonist
- Have motor abnormalities other than those related to the fatty acid oxidation disorder that could interfere with the study procedures, as determine by the investigator
- Evidence of significant concomitant medical or psychiatric disease that in the opinion of the Investigator may interfere with the conduct or safety of this study
- 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.
Primary outcome
To evaluate change in function as measured by 12 minute walk test in adult subjects with long chain FAOD
Time frame: Week 16
Change from baseline in 12MWT
1 secondary outcome
To evaluate change in symptoms related to FAOD using a newly developed muscle symptom questionnaire specifically designed for adult patients with long chain FAOD
Time frame: Week 16
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 18 Nov 2023.
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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.
Melanie Gillingham, PhD, RD, LD principal investigator
Dept of Molecular and Medical Genetics, Oregon Health and Science University
15 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fatty Acid Oxidation Disorder, run by Reneo Pharma Ltd at 15 sites in 11 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 8 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 18 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Italy, Spain, Austria, Belgium and 6 more countries. 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.
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?
To evaluate change in function as measured by 12 minute walk test in adult subjects with long chain FAOD (time frame: Week 16). 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 18 Nov 2023.