Pyridoxine Dependant EpilepsyPDE MAXNCT04672226
Evaluation of PDE MAX
A study testing PDE MAX in people with Pyridoxine Dependant Epilepsy, run by Vitaflo International, Ltd at 2 sites in 2 countries.
Trial summary
PDE MAX is a single arm prospective, feasibility study in up to 15 participants aged one (1) year and over of PDE MAX for the dietary management of Pyridoxine Dependent Epilepsy. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Pyridoxine Dependant Epilepsy
- What is being tested?
- PDE MAX
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 1 Jun 2021to 31 Jul 2023
- Phase
- Interventional
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of Pyridoxine Dependent Epilepsy (PDE), biochemically and/or genetically confirmed.
- Males or females aged one (1) year and above. Any participant aged 16 years and over at screening must have the capacity to consent for themselves.
- Currently following a lysine-restricted diet for a minimum of four (4) weeks prior to screening.
- Willing to take the study product and follow advice given by the dietitian.
- Willingly given, written, informed consent from patient or parent/guardian.
- Willingly given, written assent (if appropriate).
Who cannot join
- Inability to comply with the study protocol, in the opinion of the investigator.
- Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator, such as but not limited to arginine and pyridoxine. In which case, supplementation must have started four (4) weeks prior to screening with no anticipated changes to intakes during the study duration.
- Participants who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period. Participants of child-bearing potential will be required to undergo pregnancy test prior to enrolment.
- N.B.: Participants who become pregnant unexpectedly during this study may, in consultation with their doctor, continue on the study's dietary product if they wish but will not have any investigations that would not normally be carried out during pregnancy.
- Allergy to any ingredient present in the study product.
- Other concurrent medical or psychiatric conditions, which, in the opinion of the Investigator, would place the subject at increased risk, preclude obtaining voluntary consent/assent or compliance with required study procedures, or would confound the objectives of the study.
- Is participating in any other interventional study and has received any other investigational drug, product or device within 30 days prior to screening or are taking part in a non-medication study which, in the opinion of the investigator, would interfere with study compliance or outcome assessments.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Feasibility Study to Evaluate PDE MAX, a Food for Special Medical Purposes (FSMP) for Use in the Dietary Management of Pyridoxine Dependent Epilepsy (PDE) With Regards to Acceptability, Tolerability, Adherence and Effect on Metabolic Control
PDE MAX is a single arm prospective, feasibility study in up to 15 participants aged one (1) year and over of PDE MAX for the dietary management of Pyridoxine Dependent Epilepsy.
Read the detailed description
PDE Max is a newly-developed product designed specifically to meet the nutritional requirements of patients following a lysine-restricted diet for PDE.
This is a feasibility study to evaluate PDE MAX, a food for special medical purposes (FSMP) for use in the dietary management of Pyridoxine Dependent Epilepsy (PDE) with regards to acceptability, tolerability, adherence and effect on metabolic control.
Participants will be given an eight-week supply of PDE MAX and they will be asked to complete a daily diary and short questionnaire to record information on: adherence, gastrointestinal tolerance, palatability and how the product is used.
Blood and urine samples will be taken at the beginning and end of the study to measure several biochemical parameters.
Physical and neurological assessments will be carried out by the local Metabolic Consultant at the beginning and end of the study.
Routine monitoring of lysine levels will continue.
- Conditions
- Pyridoxine Dependant Epilepsy
- Being tested
- PDE MAX
- Keywords
- PDE
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PDE MAXDietary supplement
PDE MAX will be prescribed by the study dietitian based on the patient's individual requirement.
| Study group | Role | What they receive |
|---|---|---|
| PDE MAX | Experimental | PDE MAX will be prescribed by the study dietitian based on the patient's individual requirement. Receives: Dietary Supplement: PDE MAX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Product acceptability rated on a Likert scale by the patient after eight week intake
Assessment of participant's acceptability following an eight week intake of the study product
Measured: 8 weeks
Questionnaire of self-reported changes in gastrointestinal tolerance during eight week intake
Assessment of participant's gastrointestinal tolerance during the eight week intake of the study product
Measured: 8 weeks
Questionnaire of self-reported adherence to the prescribed amount of study product
Assessment of participant's adherence to prescribed amount during the eight week intake of the study product
Measured: 8 weeks
Secondary outcomes (16)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of pipecolic acid in plasma.
To observe any change from baseline, after an 8-week intake of PDE MAX, in pipecolic acid in plasma.
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in bloodspots
To observe the changes from baseline, after an 8-week intake of PDE MAX, in 6-oxo-pipecolic acid in bloodspots
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in plasma
To observe any changes from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in urine
To observe any changes from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in urine
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of P6C in bloodspots
To observe any changes from baseline, after an 8-week intake of PDE MAX, of P6C in bloodspots
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of P6C in plasma
To observe any changes from baseline, after an 8-week intake of PDE MAX, of P6C in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of αAASA in plasma
To observe any changes from baseline, after an 8-week intake of PDE MAX, of αAASA in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of αAASA in urine
To observe any changes from baseline, after an 8-week intake of PDE MAX, of αAASA in urine
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of the amino acid profile in plasma
To observe any changes from baseline, after an 8-week intake of PDE MAX, of the amino acid profile in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of whole blood serotonin
To observe any changes from baseline, after an 8-week intake of PDE MAX, of whole blood serotonin
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of pyridoxal phosphate in plasma
To observe any changes from baseline, after an 8-week intake of PDE MAX, of pyridoxal phosphate in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of vitamers in plasma
To observe any changes from baseline, after an 8-week intake of PDE MAX, of vitamers in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of organic acids in urine
To observe any changes from baseline, after an 8-week intake of PDE MAX, of organic acids in urine
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 2OPP in bloodspots
To observe the changes from baseline, after an 8-week intake of PDE MAX, of 2OPP in bloodspots
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 2OPP in plasma
To observe the changes from baseline, after an 8-week intake of PDE MAX, of 2OPP in plasma
Measured: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 2OPP in urine
To observe the changes from baseline, after an 8-week intake of PDE MAX, of 2OPP in urine
Measured: Day 0 (visit 1) to day 56 (visit 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2020
- Study start1 Jun 2021
- Primary completion20 Jul 2023
- Study completion31 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pyridoxine Dependant EpilepsyStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Radboud University Medical Center, Great Ormond Street Hospital for Children NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Clara van Karnebeek, Principal investigator, Amsterdam UMC, location VUmc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04672226
- Sponsor's protocol ID
- MCT-W-PDEPS-2017-10-11
- Other IDs
- 254178, 20/NW/0370
- Acronym
- PDE MAX
- Status last verified
- Feb 2024
- First submitted
- 3 Dec 2020
- Last updated
- 16 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Feasibility Study to Evaluate PDE MAX, a Food for Special Medical Purposes (FSMP) for Use in the Dietary Management of Pyridoxine Dependent Epilepsy (PDE) With Regards to Acceptability, Tolerability, Adherence and Effect on Metabolic Control
- ClinicalTrials.gov ID
- NCT04672226
- Acronym / sponsor ID
- PDE MAX / MCT-W-PDEPS-2017-10-11
- Conditions
- Pyridoxine Dependant Epilepsy
- Keywords
- PDE
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Collaborators
- Radboud University Medical Center, Great Ormond Street Hospital for Children NHS Foundation Trust
- Study start
- 1 Jun 2021 (actual)
- Primary completion
- 20 Jul 2023 (actual)
- Study completion
- 31 Jul 2023 (actual)
- First posted
- 17 Dec 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.
PDE MAX is a single arm prospective, feasibility study in up to 15 participants aged one (1) year and over of PDE MAX for the dietary management of Pyridoxine Dependent Epilepsy.
Detailed description
PDE Max is a newly-developed product designed specifically to meet the nutritional requirements of patients following a lysine-restricted diet for PDE.
This is a feasibility study to evaluate PDE MAX, a food for special medical purposes (FSMP) for use in the dietary management of Pyridoxine Dependent Epilepsy (PDE) with regards to acceptability, tolerability, adherence and effect on metabolic control.
Participants will be given an eight-week supply of PDE MAX and they will be asked to complete a daily diary and short questionnaire to record information on: adherence, gastrointestinal tolerance, palatability and how the product is used.
Blood and urine samples will be taken at the beginning and end of the study to measure several biochemical parameters.
Physical and neurological assessments will be carried out by the local Metabolic Consultant at the beginning and end of the study.
Routine monitoring of lysine levels will continue.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PDE MAX | Experimental | Dietary Supplement: PDE MAXPDE MAX will be prescribed by the study dietitian based on the patient's individual requirement. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of Pyridoxine Dependent Epilepsy (PDE), biochemically and/or genetically confirmed.
- Males or females aged one (1) year and above. Any participant aged 16 years and over at screening must have the capacity to consent for themselves.
- Currently following a lysine-restricted diet for a minimum of four (4) weeks prior to screening.
- Willing to take the study product and follow advice given by the dietitian.
- Willingly given, written, informed consent from patient or parent/guardian.
Key exclusion criteria (7 listed)
- Inability to comply with the study protocol, in the opinion of the investigator.
- Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator, such as but not limited to arginine and pyridoxine. In which case, supplementation must have started four (4) weeks prior to screening with no anticipated changes to intakes during the study duration.
- Participants who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period. Participants of child-bearing potential will be required to undergo pregnancy test prior to enrolment.
- N.B.: Participants who become pregnant unexpectedly during this study may, in consultation with their doctor, continue on the study's dietary product if they wish but will not have any investigations that would not normally be carried out during pregnancy.
- Allergy to any ingredient present in the study product.
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 |
|---|---|---|
| PDE MAXPDE MAX will be prescribed by the study dietitian based on the patient's individual requirement. | Dietary supplement | PDE MAX |
Primary outcome
Product acceptability rated on a Likert scale by the patient after eight week intake
Time frame: 8 weeks
Assessment of participant's acceptability following an eight week intake of the study product
Questionnaire of self-reported changes in gastrointestinal tolerance during eight week intake
Time frame: 8 weeks
Assessment of participant's gastrointestinal tolerance during the eight week intake of the study product
Questionnaire of self-reported adherence to the prescribed amount of study product
Time frame: 8 weeks
Assessment of participant's adherence to prescribed amount during the eight week intake of the study product
16 secondary outcomes
Change in concentration from baseline, after an 8-week intake of PDE MAX, of pipecolic acid in plasma.
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in bloodspots
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 6-oxo-pipecolic acid in urine
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of P6C in bloodspots
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of P6C in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of αAASA in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of αAASA in urine
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of the amino acid profile in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of whole blood serotonin
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of pyridoxal phosphate in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of vitamers in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of organic acids in urine
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 2OPP in bloodspots
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 2OPP in plasma
Time frame: Day 0 (visit 1) to day 56 (visit 2)
Change in concentration from baseline, after an 8-week intake of PDE MAX, of 2OPP in urine
Time frame: Day 0 (visit 1) to day 56 (visit 2)
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 16 Feb 2024.
- Individual participant dataSharing planned: No
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.
Clara van Karnebeek principal investigator
Amsterdam UMC, location VUmc
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PDE MAX in people with Pyridoxine Dependant Epilepsy, run by Vitaflo International, 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 6 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 16 Feb 2024. 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 Netherlands, 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.
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?
Product acceptability rated on a Likert scale by the patient after eight week intake (time frame: 8 weeks). The study lists 3 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 16 Feb 2024.