Back to results

Evaluation of PDE MAX (PDE MAX)

Primary Purpose

Pyridoxine Dependant Epilepsy

Status

Completed

Phase

Not Applicable

Locations

International

Study Type

Interventional

Intervention

PDE MAX

About this trial

This is an interventional treatment trial for Pyridoxine Dependant Epilepsy focused on measuring PDE

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Radboud UMC
Great Ormond Street Hospital for Children

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PDE MAX

Arm Description

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

Outcomes

Primary Outcome Measures

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

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

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

Secondary Outcome Measures

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.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Full Information

NCT ID

NCT04672226

First Posted

December 3, 2020

Last Updated

August 18, 2023

Sponsor

Vitaflo International, Ltd

Collaborators

Radboud University Medical Center, Great Ormond Street Hospital for Children NHS Foundation Trust

1. Study Identification

Unique Protocol Identification Number

NCT04672226

Brief Title

Evaluation of PDE MAX

Acronym

PDE MAX

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Completed

Study Start Date

June 1, 2021 (Actual)

Primary Completion Date

July 20, 2023 (Actual)

Study Completion Date

July 31, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vitaflo International, Ltd

Collaborators

Radboud University Medical Center, Great Ormond Street Hospital for Children NHS Foundation Trust

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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. Routine monitoring of lysine levels will continue.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pyridoxine Dependant Epilepsy

Keywords

PDE

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

11 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PDE MAX

Arm Type

Experimental

Arm Description

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

Intervention Type

Dietary Supplement

Intervention Name(s)

PDE MAX

Intervention Description

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

Primary Outcome Measure Information:

Title

Product acceptability rated on a Likert scale by the patient after eight week intake

Description

Assessment of participant's acceptability following an eight week intake of the study product

Time Frame

8 weeks

Title

Questionnaire of self-reported changes in gastrointestinal tolerance during eight week intake

Description

Assessment of participant's gastrointestinal tolerance during the eight week intake of the study product

Time Frame

8 weeks

Title

Questionnaire of self-reported adherence to the prescribed amount of study product

Description

Assessment of participant's adherence to prescribed amount during the eight week intake of the study product

Time Frame

8 weeks

Secondary Outcome Measure Information:

Title

Change in concentration from baseline, after an 8-week intake of PDE MAX, of pipecolic acid in plasma.

Description

To observe any change from baseline, after an 8-week intake of PDE MAX, in pipecolic acid in plasma.

Time Frame