Urea Cycle DisorderMRDNCT06664840
MyRareDiet A Novel Diet Tracking Tool
A study testing MDR diet assessment and 24 hour recall in people with Urea Cycle Disorder and Propionic Aciduria, run by Oregon Health and Science University.
Trial summary
The investigators propose to develop and validate MyRareDiet® (MRD) to address an unmet need in the inborn errors of metabolism (IEM) population to assist with dietary management designed to increase adherence and compliance to treatment guidelines, while facilitating the collection of dietary data from individuals… Read more about this study
- Who is it for?
- Ages 1 year to 80 years
- Conditions the study is for
- Urea Cycle Disorder, Propionic Aciduria, Maple Syrup Urine Disease
- What is being tested?
- MDR diet assessment and 24 hour recall
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 15 Nov 2024to 31 Dec 2025
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 1 year to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- diagnosed with urea cycle disorder, propionic acidemia, maple syrup urine disease or methylmalonic acidemia
- consuming a diet where ≥50% of energy is supplied by foods consumed orally
- self-known (or prescribed) dietary energy goal and protein restriction
- internet connected device to access MyRareDiet
Who cannot join
- pregnant
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: MyRareDiet™: A Diet Tracking, Monitoring and Optimization mHealth Tool for Patients With Inborn Errors of Metabolism
The investigators propose to develop and validate MyRareDiet® (MRD) to address an unmet need in the inborn errors of metabolism (IEM) population to assist with dietary management designed to increase adherence and compliance to treatment guidelines, while facilitating the collection of dietary data from individuals with IEM for research purposes.
Read the detailed description
Inborn errors of metabolism (IEM) are a group of rare genetic disorders that cause a block in a metabolic pathway leading to clinically significant consequences. Most of the disorders are caused by single gene defects that result in a deficiency of a particular enzyme that metabolizes dietary nutrients, eventually causing an accumulation of abnormal metabolites, lack of endogenous production of substances, and/or production of toxic substances that have catastrophic effects on the brain and organs. IEM can lead to developmental and intellectual disabilities, seizures, and, if untreated, coma and death. The majority of the disorders are managed by manipulation and modification of diet alone to reduce toxic metabolites, or with a combination of dietary modification and medications. The complex dietary therapies used in IEM challenge routine dietary recommendations and render diet apps designed for the general public of little utility in these conditions. Additionally, no diet app is developed specifically for the IEM community and meets the rigor required for research, particularly the need to measure specific and multiple amino acids, fats and carbohydrates in the diet to facilitate more study on impact on brain and organs. In IEM, paper diet diaries are often utilized to track food intake and inform patients and their dietitians whether their diets are consistent with the therapy recommendations. These diaries are frequently inadequate in capturing what is actually being consumed due to recall errors and other factors. Novel methods of assessing dietary intake are required to reduce the diet tracking burden, improve accuracy in dietary surveys, and improve diet adherence.
The investigators propose to develop and validate MyRareDiet® (MRD) to address an unmet need in the IEM population to assist with dietary tracking while facilitating the collection of dietary data from individuals with IEM for research purposes. MRD includes a Patient Portal, a Clinical Dietician Portal, and a Researcher Portal. The Patient Portal is to help IEM patients track and monitor their own diet so they can achieve their strict diet targets. The main features include the integration of a major food database for regular foods with a third database for medical foods, the recording of food intake, and daily dietary reports for food monitoring. The Clinical Dietician Portal helps clinical dieticians manage diet modifications for individuals with IEM. It allows a dietitian to individualize MRD for a specific patient based on findings from their nutrition assessment, and set their nutrient goals and restrictions. The Researcher Portal will facilitate the conduct of diet-based studies and the analysis of study results. It offers typical clinical trial management features such as site and staff management, protocol specification, recruitment status, and study data exports.
- Conditions
- Urea Cycle Disorder, Propionic Aciduria, Maple Syrup Urine Disease, Methylmalonic Acidemia
- Being tested
- MDR diet assessment, 24 hour recall
- Medical subject headings
- Urea Cycle Disorders, Inborn, Propionic Acidemia, Maple Syrup Urine Disease, Methylmalonic acidemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MDR diet assessmentOther
Participants will record their dietary intake on the MyRareDiet (MRD) app and nutrient content will be assessed to validate MRD compared to a traditional interviewer assessed 24 hour recall.
24 hour recallOther
A trained nutritionist will conduct a telephone multi-pass 24 hour diet recall on 3 randomized nonconsecutive days. The dietary intake will be analyzed using NDSR and metabolic pro and nutrient intake will be compared to the nutrient intake assessed with the MRD app.
| Study group | Role | What they receive |
|---|---|---|
| MyRareDiet | Experimental | MyRareDiet (MRD) entries: Participants will enter their dietary intake into the MyRareDiet app on a series of three random nonconsecutive days. Participants will receive reminders from both OHSU bionutritionist (via email, phone, or text) and from the MRD system to record their dietary intake on the MRD app for particular randomized days. Receives: Other: MDR diet assessment |
| 24 hour recall | Active comparator | 24-hour diet recalls: A series of three randomized nonconsecutive days of 24-hour food recall interviews by telephone will be completed by trained OHSU Bionutrition personnel to determine nutrient intake. Dietary intake will be evaluated with on Nutrition Data System for Research (NDSR) and Metabolic Pro nutrient databases. Receives: Other: 24 hour recall |
What is being measured
The primary outcome is the main question the study is designed to answer.
Energy
Total reported energy intake
Measured: 24 hour
Protein
Total percent of calories from protein
Measured: 24 hour
Secondary outcomes (2)
Carbohydrate
total percent of calories from carbohydrate
Measured: 24 hour
Fat
Total percent of calories from Fat
Measured: 24 hour
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2024
- Study start15 Nov 2024 (estimated)
- Primary completion30 Sep 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- This study is to conduct a validity study to test the agreement between MyRareDiet and the validated interview-based 24-hour diet recall method used in the National Health and Nutrition Examination Survey (NHANES).
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Collaborators
- BrightOutcome, National Urea Cycle Disorder Foundation, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Principal investigator, Melanie B Gillingham, Oregon Health and Science University
- Investigators
- Dershung Yang, PhD, Principal investigator, BrightOutcomes, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06664840
- Sponsor's protocol ID
- 1R44HD107766
- Other IDs
- 1R44HD107766
- Acronym
- MRD
- Status last verified
- Oct 2024
- First submitted
- 26 Oct 2024
- Last updated
- 30 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The confidentiality of subjects' data will be maintained by use of de-identification procedures. Informed consent forms that include the subject's name will be stored separately in locked file cabinets. Electronic records will be handled according to HIPAA rules where individually identifiable data, such as names, any identification numbers and certain demographic information, are stored separately and password-protected so that they can be accessed only by the project Investigators. A unique subject identifier (ID) will be generated for each participant and used by the subject to access the developed prototype system and for researchers to retrieve information related to a specific subject. At the end of this study, all patient identifiable data in all formats will be completely destroyed.
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MyRareDiet™: A Diet Tracking, Monitoring and Optimization mHealth Tool for Patients With Inborn Errors of Metabolism
- ClinicalTrials.gov ID
- NCT06664840
- Acronym / sponsor ID
- MRD / 1R44HD107766
- Conditions
- Urea Cycle Disorder, Propionic Aciduria, Maple Syrup Urine Disease, Methylmalonic Acidemia
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Collaborators
- BrightOutcome, National Urea Cycle Disorder Foundation, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- 15 Nov 2024 (estimated)
- Primary completion
- 30 Sep 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 30 Oct 2024 (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 investigators propose to develop and validate MyRareDiet® (MRD) to address an unmet need in the inborn errors of metabolism (IEM) population to assist with dietary management designed to increase adherence and compliance to treatment guidelines, while facilitating the collection of dietary data from individuals with IEM for research purposes.
Detailed description
Inborn errors of metabolism (IEM) are a group of rare genetic disorders that cause a block in a metabolic pathway leading to clinically significant consequences. Most of the disorders are caused by single gene defects that result in a deficiency of a particular enzyme that metabolizes dietary nutrients, eventually causing an accumulation of abnormal metabolites, lack of endogenous production of substances, and/or production of toxic substances that have catastrophic effects on the brain and organs. IEM can lead to developmental and intellectual disabilities, seizures, and, if untreated, coma and death. The majority of the disorders are managed by manipulation and modification of diet alone to reduce toxic metabolites, or with a combination of dietary modification and medications. The complex dietary therapies used in IEM challenge routine dietary recommendations and render diet apps designed for the general public of little utility in these conditions. Additionally, no diet app is developed specifically for the IEM community and meets the rigor required for research, particularly the need to measure specific and multiple amino acids, fats and carbohydrates in the diet to facilitate more study on impact on brain and organs. In IEM, paper diet diaries are often utilized to track food intake and inform patients and their dietitians whether their diets are consistent with the therapy recommendations. These diaries are frequently inadequate in capturing what is actually being consumed due to recall errors and other factors. Novel methods of assessing dietary intake are required to reduce the diet tracking burden, improve accuracy in dietary surveys, and improve diet adherence.
The investigators propose to develop and validate MyRareDiet® (MRD) to address an unmet need in the IEM population to assist with dietary tracking while facilitating the collection of dietary data from individuals with IEM for research purposes. MRD includes a Patient Portal, a Clinical Dietician Portal, and a Researcher Portal. The Patient Portal is to help IEM patients track and monitor their own diet so they can achieve their strict diet targets. The main features include the integration of a major food database for regular foods with a third database for medical foods, the recording of food intake, and daily dietary reports for food monitoring. The Clinical Dietician Portal helps clinical dieticians manage diet modifications for individuals with IEM. It allows a dietitian to individualize MRD for a specific patient based on findings from their nutrition assessment, and set their nutrient goals and restrictions. The Researcher Portal will facilitate the conduct of diet-based studies and the analysis of study results. It offers typical clinical trial management features such as site and staff management, protocol specification, recruitment status, and study data exports.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This study is to conduct a validity study to test the agreement between MyRareDiet and the validated interview-based 24-hour diet recall method used in the National Health and Nutrition Examination Survey (NHANES).
| Arm | Type | Receives |
|---|---|---|
| MyRareDiet | Experimental | Other: MDR diet assessmentMyRareDiet (MRD) entries: Participants will enter their dietary intake into the MyRareDiet app on a series of three random nonconsecutive days. Participants will receive reminders from both OHSU bionutritionist (via email, phone, or text) and from the MRD system to record their dietary intake on the MRD app for particular randomized days. |
| 24 hour recall | Active comparator | Other: 24 hour recall24-hour diet recalls: A series of three randomized nonconsecutive days of 24-hour food recall interviews by telephone will be completed by trained OHSU Bionutrition personnel to determine nutrient intake. Dietary intake will be evaluated with on Nutrition Data System for Research (NDSR) and Metabolic Pro nutrient databases. |
Who may qualify
- Ages
- 1 year to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- diagnosed with urea cycle disorder, propionic acidemia, maple syrup urine disease or methylmalonic acidemia
- consuming a diet where ≥50% of energy is supplied by foods consumed orally
- self-known (or prescribed) dietary energy goal and protein restriction
- internet connected device to access MyRareDiet
Key exclusion criteria (1 listed)
- pregnant
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 |
|---|---|---|
| MDR diet assessmentParticipants will record their dietary intake on the MyRareDiet (MRD) app and nutrient content will be assessed to validate MRD compared to a traditional interviewer assessed 24 hour recall. | Other | MyRareDiet |
| 24 hour recallA trained nutritionist will conduct a telephone multi-pass 24 hour diet recall on 3 randomized nonconsecutive days. The dietary intake will be analyzed using NDSR and metabolic pro and nutrient intake will be compared to the nutrient intake assessed with the MRD app. | Other | 24 hour recall |
Primary outcome
Energy
Time frame: 24 hour
Total reported energy intake
Protein
Time frame: 24 hour
Total percent of calories from protein
2 secondary outcomes
Carbohydrate
Time frame: 24 hour
Fat
Time frame: 24 hour
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2024.
- Individual participant dataSharing planned: No. The confidentiality of subjects' data will be maintained by use of de-identification procedures. Informed consent forms that include the subject's name will be stored separately in locked file cabinets. Electronic records will be handled according to HIPAA rules where individually identifiable data, such as names, any identification numbers and certain demographic information, are stored separately and password-protected so that they can be accessed only by the project Investigators. A unique subject identifier (ID) will be generated for each participant and used by the subject to access the developed prototype system and for researchers to retrieve information related to a specific subject. At the end of this study, all patient identifiable data in all formats will be completely destroyed.
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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.
Dershung Yang, PhD principal investigator
BrightOutcomes, Inc.
Questions about this trial
What is this clinical trial studying?
A study testing MDR diet assessment and 24 hour recall in people with Urea Cycle Disorder and Propionic Aciduria, run by Oregon Health and Science University.
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 1 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: opening soon, as of the sponsor's last update on 30 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Melanie B Gillingham, PhD (503-494-1682, gillingm@ohsu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Energy (time frame: 24 hour). The study lists 2 primary outcomes in all. 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 30 Oct 2024; AzTrials last refreshed its copy on 26 Oct 2026.