Adults 16 to 55, any sex, with Phenylketonuria. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change From Baseline in the Blood Phenylalanine (Phe) ConcentrationPrimary· At Baseline and Change from Baseline to Week 48, Week 96, Week 144, Week 216, and Week 240
Blood Phe Concentration (Change from Baseline) and Daily Dose in PAL-003 Subjects by Disposition (PAL-003 Population)
Baseline
Group
Value
95% CI
rAvPAL-PEG
1022.4
± 530.39
Change from Baseline to Week 48
Group
Value
95% CI
rAvPAL-PEG
-553.5
± 652.95
Change from Baseline to Week 96
Group
Value
95% CI
rAvPAL-PEG
-653.3
± 637.81
Change from Baseline to Week 144
Group
Value
95% CI
rAvPAL-PEG
-672.3
± 621.06
Change from Baseline to Week 216
Group
Value
95% CI
rAvPAL-PEG
-535.9
± 573.08
Change from Baseline to Week 240
Group
Value
95% CI
rAvPAL-PEG
-574.9
± 549.90
Pharmacokinetics-Plasma Pegvaliase ConcentrationSecondary· At Baseline, Week 48, Week 96, Week 144, Week 216 and Week 240
Steady-state Pharmacokinetics (PK) of pegvaliase was measured in subjects who have achieved and maintained target blood Phe
At Baseline
Group
Value
95% CI
rAvPAL-PEG
961.5
± 3013.43
Week 48
Group
Value
95% CI
rAvPAL-PEG
4127.3
± 6116.72
Week 96
Group
Value
95% CI
rAvPAL-PEG
7457.7
± 10685.02
Week 144
Group
Value
95% CI
rAvPAL-PEG
7196.1
± 11678.89
Week 216
Group
Value
95% CI
rAvPAL-PEG
7764.4
± 9292.34
Week 240
Group
Value
95% CI
rAvPAL-PEG
6557.8
± 9220.95
Number of Subjects With Treatment Emergent Adverse Events (TEAEs)Secondary· Up to 109 months.
A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase (PAL-003)
Subjects with any adverse event
Group
Value
95% CI
rAvPAL-PEG
68
Subjects with any serious adverse events
Group
Value
95% CI
rAvPAL-PEG
12
Subjects with any treatment-related adverse event
Group
Value
95% CI
rAvPAL-PEG
65
Any treatment-related serious adverse event
Group
Value
95% CI
rAvPAL-PEG
6
Death
Group
Value
95% CI
rAvPAL-PEG
0
Percentage of Participants With Positive PEG IgG AntibodySecondary· At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156
The presence of IgG Antibodies against PEG (polyethylene glycol) is measured overtime
Baseline
Group
Value
95% CI
rAvPAL-PEG
29
Change from Baseline to Week 24
Group
Value
95% CI
rAvPAL-PEG
24
Change from Baseline to Week 52
Group
Value
95% CI
rAvPAL-PEG
14
Change from Baseline to Week 104
Group
Value
95% CI
rAvPAL-PEG
2
Change from Baseline to Week 156
Group
Value
95% CI
rAvPAL-PEG
7
Percentage of Participants With Positive PAL IgG AntibodySecondary· At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156
The presence of IgG Antibodies against PAL (phenylalanine ammonia lyase) is measured overtime
Baseline
Group
Value
95% CI
rAvPAL-PEG
10
Change from Baseline to Week 24
Group
Value
95% CI
rAvPAL-PEG
63
Change from Baseline to Week 52
Group
Value
95% CI
rAvPAL-PEG
60
Change from Baseline to Week 104
Group
Value
95% CI
rAvPAL-PEG
48
Change from Baseline to Week 156
Group
Value
95% CI
rAvPAL-PEG
45
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 109 months..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
rAvPAL-PEG
Serious: 12/68 (18%)
Deaths: 0/68
Serious adverse events (21 terms)
Reaction
System
rAvPAL-PEG
Asthma
Respiratory, thoracic and mediastinal disorders
—
Hypersensitivity
Immune system disorders
—
Abortion induced
Surgical and medical procedures
—
Anaphylactic reaction
Immune system disorders
—
Appendicitis
Infections and infestations
—
Arthralgia
Musculoskeletal and connective tissue disorders
—
Bacteraemia
Infections and infestations
—
Bacterial infection
Infections and infestations
—
Bone marrow failure
Blood and lymphatic system disorders
—
Diarrhoea infectious
Infections and infestations
—
Hyponatraemia
Metabolism and nutrition disorders
—
Hypotension
Vascular disorders
—
Injection related reaction
Injury, poisoning and procedural complications
—
Lymphadenopathy
Blood and lymphatic system disorders
—
Neuropathy peripheral
Nervous system disorders
—
Neutropenia
Blood and lymphatic system disorders
—
Panniculitis
Skin and subcutaneous tissue disorders
—
Postoperative wound complication
Injury, poisoning and procedural complications
—
Staphylococcal infection
Infections and infestations
—
Suicide attempt
Psychiatric disorders
—
Urticaria
Skin and subcutaneous tissue disorders
—
Other adverse events (132 terms — click to expand)
This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain reduced blood Phe concentrations in PKU subjects.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06971731 — A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)
· Phase 3
· recruiting
NCT07526909 — Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Cla
· NA
· recruiting
NCT06941532 — GMP Powdered Substitutes in PKU and TYR
· NA
· recruiting
NCT06901323 — Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Child
· Phase 4
· active not recruiting
NCT06302348 — A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
· Phase 3
· recruiting
Other BioMarin Pharmaceutical trials
Trials by the same sponsor.
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· recruiting
NCT06309979 — A Study to Assess Growth in Children With Idiopathic Short Stature
· recruiting
NCT06455059 — Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
· Phase 3
· active not recruiting
NCT06305234 — A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by BioMarin Pharmaceutical
Last refreshed: 12 October 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00924703.