Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: An open-label, multicentre, phase 1 dose-escalation trial

The Lancet

Volumn 384, Issue 9937, 2014, Pages 37-44

  Longo, Nicola ^a   Harding, Cary O ^b   Burton, Barbara K ^c   Grange, Dorothy K ^d   Vockley, Jerry ^e   Wasserstein, Melissa ^f   Rice, Gregory M ^g   Dorenbaum, Alejandro ^h   Neuenburg, Jutta K ^h   Musson, Donald G ^h   Gu, Zhonghua ^h   Sile, Saba ^h  

^a UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^c NORTHWESTERN UNIVERSITY   (United States)

^d WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^f MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^g UNIVERSITY OF WISCONSIN MADISON   (United States)

^h BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DRUG ANTIBODY; MACROGOL; MEDROXYPROGESTERONE ACETATE; PHENYLALANINE; PHENYLALANINE AMMONIA LYASE; PHENYLALANINE 4 MONOOXYGENASE; RECOMBINANT ENZYME; RECOMBINANT PHENYLALANINE AMMONIA LYASE; UNCLASSIFIED DRUG;

ADULT; ANABAENA VARIABILIS; AREA UNDER THE CURVE; ARM PAIN; ARTHRALGIA; ARTICLE; AUTOIMMUNE DISEASE; CLINICAL ARTICLE; CONNECTIVE TISSUE DISEASE; DIZZINESS; DRUG BLOOD LEVEL; DRUG CONJUGATION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG FATALITY; DRUG HYPERSENSITIVITY; DRUG INTOXICATION; DRUG SAFETY; DRUG TOLERABILITY; FEMALE; GASTROINTESTINAL DISEASE; HEADACHE; HUMAN; INFECTION; INFESTATION; INJECTION SITE CONTUSION; INJECTION SITE REACTION; INJURY; LEG PAIN; MALE; MULTICENTER STUDY; MUSCLE SPASM; MUSCULOSKELETAL DISEASE; MUSCULOSKELETAL PAIN; MYALGIA; PHASE 1 CLINICAL TRIAL; PHENYLKETONURIA; PRIORITY JOURNAL; RASH; RHINOPHARYNGITIS; SIDE EFFECT; SINGLE DRUG DOSE; SKIN DISEASE; TIME TO MAXIMUM PLASMA CONCENTRATION; URTICARIA; ANAPHYLAXIS; DRUG EFFECT; ELIMINATION RATE CONSTANT; ERYTHEMA; MAXIMUM PLASMA CONCENTRATION; OPEN STUDY; PAIN; PLASMA CONCENTRATION-TIME CURVE; SWELLING;

ADULT; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; INJECTIONS, SUBCUTANEOUS; MALE; PHENYLALANINE; PHENYLALANINE AMMONIA-LYASE; PHENYLKETONURIAS; POLYETHYLENE GLYCOLS; TIME FACTORS; TREATMENT OUTCOME; UNITED STATES;

EID: 84903759443     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(13)61841-3     Document Type: Article

References (17)

1. Scriver CR, Kaufman S. Hyperphenylalaninemia: phenylalanine hydroxylase deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The metabolic and molecular bases of inherited disease, 7th edn. New York: McGraw-Hill, 2001: 1667-724.
2. Mitchell JJ, Trakadis YJ, Scriver CR Phenylalanine hydroxylase deficiency. Genet Med 2011; 13: 697-707.
3. Ozalp I, Coskun T, Tokol S., Demircin G, Mönch E. Inherited metabolic disorders in Turkey. J Inherit Metab Dis 1990; 13: 732-38.
4. DiLella AG, Kwok SC, Ledley F.D., Marvit J., Woo SL Molecular structure and polymorphic map of the human phenylalanine hydroxylase gene. Biochemistry 1986; 25: 743-49.
5. Aoki K. Long term follow-up of patients with inborn errors of metabolism detected by the newborn screening program in Japan. Southeast Asian J Trop Med Public Health 2003; 34 (suppl 3): 19-23.
6. National PKU Alliance. PKU facts. http://www.npkua.org/index.php/pku- facts (accessed Jan 28, 2014).
7. National Institutes of Health Consensus Development Panel. National Institutes of Health Consensus Development Conference Statement: phenylketonuria: screening and management, October 16-18, 2000. Pediatrics 2001; 108: 972-82.
8. Fisch RO. Comments on diet and compliance in phenylketonuria. Eur J Pediatr 2000; 159 (suppl 2): S142-44.
9. Walter JH, White FJ, Hall S.K., et al How practical are recommendations for dietary control in phenylketonuria? Lancet 2002; 360: 55-57.
10. Azen C, Koch R, Friedman E., Wenz E, Fishler K. Summary of findings from the United States Collaborative Study of children treated for phenylketonuria. Eur J Pediatr 1996; 155 (suppl 1): S29-32.
11. White DA, Nortz MJ, Mandernach T, Huntington K., Steiner RD Deficits in memory strategy use related to prefrontal dysfunction during early development: evidence from children with phenylketonuria. Neuropsychology 2001; 15: 221-29.
12. White DA, Nortz MJ, Mandernach T, Huntington K., Steiner RD Age-related working memory impairments in children with prefrontal dysfunction associated with phenylketonuria. J Int Neuropsychol Soc 2002; 8: 1-11.
13. Leuzzi V, Pansini M, Sechi E., et al Executive function impairment in early-treated PKU subjects with normal mental development. J Inherit Metab Dis 2004; 27: 115-25.
14. Feldmann R, Denecke J, Grenzebach M., Weglage J. Frontal lobe-dependent functions in treated phenylketonuria: blood phenylalanine concentrations and long-term deficits in adolescents and young adults. J Inherit Metab Dis 2005; 28: 445-55.
15. Gassió R, Fusté E, López-Sala A, Artuch R, Vilaseca MA, Campistol J. School performance in early and continuously treated phenylketonuria. Pediatr Neurol 2005; 33: 267-71.
16. Sarkissian CN, Gámez A, Wang L., et al Preclinical evaluation of multiple species of PEGylated recombinant phenylalanine ammonia lyase for the treatment of phenylketonuria. Proc Natl Acad Sci USA 2008; 105: 20894-99.
17. Gregory CO, Yu C, Singh RH Blood phenylalanine monitoring for dietary compliance among patients with phenylketonuria: comparison of methods. Genet Med 2007; 9: 761-65.