Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria

NCT03333720 | Withdrawn

• 1 other identifier: PKU.LP2017
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Thirty eligible PKU patients (≥ 8 years), identified as tablet protein substitute users, will be recruited. Patients will firstly observe a 7-day baseline period, in which participants will continue with their existing diet, protein substitute and multi vitamin prescription. Patients will then receive the phaenylalanine-free protein substitute tablets daily for 28 days in addition to appropriate nutritional management. The aim of this prospective, single arm trial is to evaluate the acceptability (compliance, tolerance and palatability) of the phenylalanine-free protein substitute tablets in patients with proven PKU. The primary outcome measure is compliance, with secondary outcome measures of gastrointestinal tolerance, acceptability, blood amino acids, dietary intake, anthropometry and safety.

Conditions

Phenylketonurias

Keywords

Phenylketonuria; Inherited Metabolic Disorders

Outcome Measures

Primary Outcomes (1)

• Daily Compliance

  Compliance with currently prescribed protein substitute and prescribed multivitamin supplements will be assessed daily from days 1-7 and recorded as a percentage of tablets consumed comapred to prescription (0-100%). Compliance with the study product will subsequently be assessed daily from days 8-35. Compliance throughout the trial will be assessed using standarised questionnaires, where patients document number of med versus number of tablets prescribed. A percentage compliance level based on per patient and total patients will be computed to define overall compliance where a higher percentage represents better overall compliance.

  35 days

Secondary Outcomes (7)

• Treatment-Emergent tolerability

  12 days

• Patient Acceptability

  2 days

• Metabolic control

  2 days

• Nutritional Intake

  2 days

• Weight

  2 days

Study Arms (1)

Intervention

EXPERIMENTAL

Intervention is phenylalanine-free protein substitute. Following a 7 day baseline period, all recruits will receive the new phenylalanine-free protein substitute daily for 28 days in addition to routine nutritional management. The study product prescription will be specified on an individual basis by the metabolic Dietitian responsible for the patient's nutritional management and will be dependent on age, bodyweight and medical condition of the patient, but will wholly replace their currently prescribed tablet protein substitute and multivitamin supplements.

Dietary Supplement: Phenylalanine-free protein substitute tablets

Interventions

Phenylalanine-free protein substitute tablets DIETARY_SUPPLEMENT

Thirty eligible PKU patients (≥ 8 years), identified as current tablet users, will be recruited to this study. Patients will firstly observe a 7-day baseline period. During baseline, patients will continue with their existing diet and protein substitute prescription before receiving Phenylalanine-free protein subsitute tablets daily for 28 days in addition to appropriate nutritional management.

Intervention

Eligibility Criteria

• Age: 8 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female
• years of age and older
• Diagnosed with proven PKU
• Consume a minimum of 20g protein equivalent in tablet format while enrolled on to the trial
• If routine care and dietary management is not different from normal PKU care and dietary management, preconception patients may be considered
• Have written informed consent from patient or parent/carer carer

You may not qualify if:

• Requiring nutritional support (including enteral and parenteral nutrition)
• Major hepatic or renal dysfunction
• Maternal PKU patients (if a patient becomes pregnant during the study they will be withdrawn on immediate notice to the Metabolic Dietitian responsible for the patient's care)
• Participation in other studies within 1 month prior to entry of this study
• Investigator concern around willingness/ability of patient to comply with protocol requirements

Sponsors & Collaborators

• Nutricia UK Ltd: lead

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Rebecca Stratton, PhD

  Nutricia, Inc.

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: September 18, 2017
• First Posted: November 7, 2017
• Study Start: July 15, 2019
• Primary Completion: December 30, 2019
• Study Completion: December 31, 2019
• Last Updated: March 13, 2019

Record last verified: 2019-03

Data Sharing

• IPD Sharing: Will not share