Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies

NCT06298292 | Not Yet Recruiting

• 1 other identifier: ZerominisCT01
• Study Type: observational
• Target: 15
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA Type I, II, III or ALKAPTONURIA, HOMOCYSTINURIA, or MAPLE SYRUP URINE DISEASE (MSUD) over the age of 7 years.

Conditions

Tyrosinemia, Type I; Tyrosinemia, Type II; Tyrosinemia, Type III; Alkaptonuria; Homocystine; Metabolic Disorder; MSUD (Maple Syrup Urine Disease)

Outcome Measures

Primary Outcomes (1)

• Daily compliance

  Compliance, with currently prescribed protein substitute will be assessed at the beginning of the 7-days trial. Usage and compliance with the study product will subsequently assessed daily from days 1-7 using standardised questionnaires, where patients document the the amount of consumed study product vs the prescribed doses.

  7 days

Secondary Outcomes (4)

• Treatment-Emergent tolerability

  7 days

• Patient Acceptability

  7 days

• Metabolic control

  7 days

• Incidence of study product emergent events.

  7 days

Study Arms (3)

ZeroTP minis

Intervention: Tyrosine-free and Phenylalanine-free protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemia I, II, III or alkaptonuria will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.

Dietary Supplement: Zero minis (range of protein substitutes in tablet form)

ZeroMet minis

Intervention: Methionine-free and Cystine-enriched protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of homocystinuria will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.

Dietary Supplement: Zero minis (range of protein substitutes in tablet form)

ZeroVIL minis

Intervention: Valine-, Isoleucine- and Leucine-free protein substitute in tablet form. Subjects who currently take a protein substitute for the dietary management of MSUD will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or their entire usual protein substitute with the new product. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute.

Dietary Supplement: Zero minis (range of protein substitutes in tablet form)

Interventions

Zero minis (range of protein substitutes in tablet form) DIETARY_SUPPLEMENT

Intervention: range of protein substitute tablets. Subjects who currently take a protein substitute for the dietary management of either tyrosinaemias or alkaptonuria, homocystinuria, or MSUD will be recruited. Subjects will take the study product for 7 days. Daily questionnaires will be completed (ease of preparation,administration; any problems or gastrointestinal effects). Subjects will replace some or their entire usual protein substitute with the new product suitable for their diagnosed rare metabolic disease. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Additional questions at the beginning and end of the study will record information on organoleptic properties, presentation and packaging of the product. Routine weekly blood samples will be collected and analysed for amino acids typically for the metabolic control of the individual specified metabolic disorders as is usual clinical practice.

Also known as: ZeroTP minis, ZeroMet minis, ZeroVIL minis

ZeroMet minis; ZeroTP minis; ZeroVIL minis

Eligibility Criteria

• Age: 7 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

15 children out of a patient pool with proven tyrosinaemia type I, II, III or alkaptonuria, homocystinuria, or MSUD will be recruited from two specialist metabolic centres.

You may qualify if:

• Diagnosis of Tyrosinaemia type I, II III or Alkaptonuria requiring a tyrosine- and phenylalanine-free protein substitute.
• Diagnosis of Homocystinuria requiring a methionine-free, cystine-enriched protein substitute.
• Diagnosis of MSUD requiring a valine-, leucine- and isoleucine-free protein substitute.
• Subjects who are already taking a protein substitute for one of the specified rare metabolic disorders and are willing to try the study product for 7 days.
• Children aged 7 years and over.
• Written informed consent obtained from parental caregiver.

You may not qualify if:

• Presence of serious concurrent illness
• Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
• Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
• Any children having taken antibiotics over the previous 2 weeks leading up to the study.
• Children less than 7 years of age.

Sponsors & Collaborators

• metaX Institut fuer Diatetik GmbH: lead
• Birmingham Women's and Children's NHS Foundation Trust: collaborator
• Great Ormond Street Hospital for Children NHS Foundation Trust: collaborator

MeSH Terms

Conditions

Tyrosinemias; Alkaptonuria; Metabolic Diseases; Ketosis

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; Nutritional and Metabolic Diseases; Acidosis; Acid-Base Imbalance

Study Officials

• Anita MacDonald, Professor

  Birmingham Children´s Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Bernhard Hoffmann, PhD

CONTACT

+49 6031 166 72 71; behoffmann@metax.org

Yvonne Mücke, PhD

CONTACT

+49 6031 166 72 79; yvonne.muecke@metax.org

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2024
• First Posted: March 7, 2024
• Study Start: April 1, 2024
• Primary Completion: March 31, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: March 7, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share