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Clinical Trials/NCT04672226
NCT04672226CompletedNot Applicable

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

Vitaflo International, Ltd3 sites in 2 countries11 target enrollmentStarted: June 1, 2021Last updated:
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Conditions
Interventions

Trial Snapshot

Phase
Not Applicable
Status
Completed
Enrollment
11
Locations
3
Primary Endpoint
Product acceptability rated on a Likert scale by the patient after eight week intake
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief 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.

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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
1 Year to — (Child, Adult, Older Adult)
Sex
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

  • •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.

Arms & Interventions

PDE MAX

Experimental

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

Intervention: PDE MAX (Dietary Supplement)

Outcomes

Primary Outcomes

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

Secondary Outcomes

  • Change in concentration from baseline, after an 8-week intake of PDE MAX, of αAASA in urine(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(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(Day 0 (visit 1) to day 56 (visit 2))
  • Change in concentration from baseline, after an 8-week intake of PDE MAX, of pipecolic acid in plasma.(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(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(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(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(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(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(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(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(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(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(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(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(Day 0 (visit 1) to day 56 (visit 2))

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (3)

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