Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial.
Ania C MuntauAlberto BurlinaFrançois EyskensPeter FreisingerVincenzo LeuzziHatice Serap SivriGwendolyn GramerRenata PazdírkováMaureen ClearyAmelia S Lotz-HavlaPaul LaneAnia Carolina MuntauFrank RutschPublished in: Orphanet journal of rare diseases (2021)
Long-term treatment with sapropterin plus a Phe-restricted diet in patients who initiated sapropterin at < 4 years of age with BH4-responsive PKU or mild HPA maintained improvements in dietary Phe tolerance over 3.5 years. These results continue to support the favourable risk/benefit profile for sapropterin in paediatric patients (< 4 years of age) with BH4-responsive PKU. Frequent monitoring of blood Phe levels and careful titration of dietary Phe intake to ensure adequate levels of protein intake is necessary to optimise the benefits of sapropterin treatment. Trial registration ClinicalTrials.gov, NCT01376908. Registered 17 June 2011, https://clinicaltrials.gov/ct2/show/NCT01376908 .
Keyphrases
- clinical trial
- open label
- study protocol
- phase iii
- phase ii
- emergency department
- end stage renal disease
- computed tomography
- cancer therapy
- physical activity
- double blind
- weight loss
- prognostic factors
- body mass index
- cross sectional
- binding protein
- positron emission tomography
- combination therapy
- magnetic resonance
- replacement therapy
- smoking cessation