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CompletedNCT04672226PDE MAXUpdated Feb 16, 2024

Evaluation of PDE MAX

An interventional study of PDE MAX in Pyridoxine Dependant Epilepsy, sponsored by Vitaflo International, Ltd. Completed at 2 sites in 2 countries. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2024-02-16.

Sponsored by Vitaflo International, Ltd · Not applicable, Interventional, and Treatment

Phase
Not applicable
Study type
Interventional
Enrollment
11
Allocation
Not applicable
Ages
1 Year and older
Sex
All
01

Study 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 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.

02

Conditions studied

  • Pyridoxine Dependant Epilepsy

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Keywords

  • PDE
03

In context

Epilepsy

1,806 studies on the registry are indexed under Epilepsy; 418 are open to participants now.

This study's enrollment of 11 is below the median of 50 across 1,207 interventional studies indexed under Epilepsy.

Browse Epilepsy studies →

Lead sponsor

Vitaflo International, Ltd is the lead sponsor of 23 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
1 Year and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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).

Exclusion criteria

Exclusion Criteria:

  • 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.
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
11 participants (actual)

Study arms

  • Experimental
    PDE MAX

    PDE MAX will be prescribed by the study dietitian based on the patient's individual requirement.

    Dietary Supplement: PDE MAX

Interventions

  • Dietary supplementPDE MAX

    PDE MAX will be prescribed by the study dietitian based on the patient's individual requirement.

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 8 weeks

  2. 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

    Time frame: 8 weeks

  3. 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

    Time frame: 8 weeks

Secondary outcomes

  1. 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.

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  3. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  4. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  5. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  6. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  7. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  8. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  9. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  10. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  11. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  12. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  13. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  14. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  15. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

  16. 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

    Time frame: Day 0 (visit 1) to day 56 (visit 2)

07

Study locations

2 sites
  • Radboud UMC
    Nijmegen, 6500, Netherlands
  • Great Ormond Street Hospital for Children
    London, United Kingdom
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 16, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04672226
Lead sponsor
Vitaflo International, Ltd
Collaborators
Radboud University Medical Center, Great Ormond Street Hospital for Children NHS Foundation Trust
Responsible party
Sponsor
First posted
Dec 17, 2020
Start date
Jun 1, 2021
Primary completion
Jul 20, 2023
Completion
Jul 31, 2023
Last update
Feb 16, 2024

Study contacts

Clara van Karnebeek
principal investigator · Amsterdam University Medical Centers

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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