Evaluation of PDE MAX

NCT04672226 | Completed

• 3 other identifiers: MCT-W-PDEPS-2017-10-1125417820/NW/0370
• Study Type: interventional
• Target: 11
• Locations: 2 countries
• Sites: 2

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.

Conditions

Pyridoxine Dependant Epilepsy

Keywords

PDE

Outcome Measures

Primary Outcomes (3)

• 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

  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

  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

  8 weeks

Secondary Outcomes (16)

• 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 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 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 P6C in bloodspots

  Day 0 (visit 1) to day 56 (visit 2)

Study Arms (1)

PDE MAX

EXPERIMENTAL

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

Dietary Supplement: PDE MAX

Interventions

PDE MAX DIETARY_SUPPLEMENT

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

PDE MAX

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vitaflo International, Ltd: lead
• Radboud University Medical Center: collaborator
• Great Ormond Street Hospital for Children NHS Foundation Trust: collaborator

Study Sites (2)

Radboud UMC

Nijmegen, 6500, Netherlands

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Study Officials

• Clara van Karnebeek

  Amsterdam University Medical Centers

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2020
• First Posted: December 17, 2020
• Study Start: June 1, 2021
• Primary Completion: July 20, 2023
• Study Completion: July 31, 2023
• Last Updated: February 16, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); NL (1)