CGMP-based protein substitutes in semi-adherent PKU Patients Version 1
Research type
Research Study
Full title
Introducing a Casein Glycomacropeptide (CGMP)-based Food for Special Medical Purposes (FSMP) in the dietary management of semi-adherent Phenylketonuria (PKU) patients. A randomized controlled trial investigating the tolerance/acceptability of CGMP-AA Protein substitute and its effects on adherence, anthropometric measures, nutritional status, quality of life and cognitive function.
IRAS ID
194679
Contact name
Denise Hofman
Contact email
Sponsor organisation
Unviersity of Leeds
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Phenylketonuria (PKU) is a rare metabolic disorder (prevalence in Europe on average 1:10.000). Patients suffering from PKU are unable to sufficiently break down the amino acid phenylalanine (Phe). To prevent severe neurological impairments and mental retardation caused by the accumulation of Phe in their blood, patients need to follow a lifelong low-protein diet supplemented with Phe-free protein substitutes. Due to the strict nature of this diet, as well as the bitter taste and off odour of conventional protein substitutes, adherence to dietary recommendations tends to decrease when patients grow older. However, these patients are often either wary of, or unused to, naturally protein-rich foods, especially those of animal origin. As a result they tend to follow a self-selected, unmeasured, un-supplemented low-protein diet which may be vegan or vegetarian and limited in food variety (semi-adherent). Such practice may put such patients at risk for increased Phe levels and micronutrient deficiencies, which could contribute to neuropsychological deficits often observed in PKU patients.
Casein glycomacropeptide (CGMP) is a protein that is naturally low in Phe. It can provide a safe alternative source of protein for patients with PKU. Short-term studies have shown that CGMP products are rated as being more acceptable in terms of taste, appearance and odour than conventional protein substitutes, which may increase adherence to dietary management. Increased adherence, in turn, could lead to improved Phe control and – ultimately – to improved nutritional status, Quality of Life (QoL) and cognitive functioning of PKU patients.
The current study will follow a targeted 12(±1) week intervention investigating the tolerance/acceptability of a CGMP-based protein substitute and its effects on adherence, anthropometric measures (weight, height, body composition), nutritional status, QoL and cognitive function in semi-adherent PKU patients. Adherent PKU patients will be tested as controls at baseline.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
16/YH/0273
Date of REC Opinion
15 Aug 2016
REC opinion
Further Information Favourable Opinion