Trial · NCT00924703

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2021-10-12

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003). Trials, record NCT00924703. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00924703

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
NCT number: NCT00924703
Status: Completed
Phases: Phase 2
Enrollment: 68 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Phenylketonuria
Interventions: rAvPAL-PEG
First posted: 2009-06-19
Last updated: 2021-10-12
Results first posted: 2021-10-12

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in the Blood Phenylalanine (Phe) Concentration	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)
Secondary	Pharmacokinetics-Plasma Pegvaliase Concentration	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
Secondary	Number of Subjects With Treatment Emergent Adverse Events (TEAEs)	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)
Secondary	Percentage of Participants With Positive PEG IgG Antibody	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
Secondary	Percentage of Participants With Positive PAL IgG Antibody	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

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in the Blood Phenylalanine (Phe) Concentration	At Baseline and Change from Baseline to Week 48, Week 96, Week 144, Week 216, and Week 240	Efficacy population consisted of all subjects who received at least one dose of study drug and have post-treatment blood Phe concentration measurements.	μmol/L	MEAN
SECONDARY	Pharmacokinetics-Plasma Pegvaliase Concentration	At Baseline, Week 48, Week 96, Week 144, Week 216 and Week 240	As PAL-003 is an extension of parent Phase 2 studies (PAL-002, PAL-004 \& 165-205), the parent Phase 2 treatment naïve baseline data (N=80) was used for the vital signs, immunogenicity \& PK analyses to monitor changes in vital signs mediated by pegvaliase, understand development \& maturation of immune response against pegvaliase, \& the resulting impact on PK. Refer to NCT00925054-PAL-002, NCT01212744-PAL-004 \& NCT01560286-165-205 for parent Ph2 demographic data (N=80).	ng/mL	MEAN
SECONDARY	Number of Subjects With Treatment Emergent Adverse Events (TEAEs)	Up to 109 months.	Analysis population consisted of subjects from the study of PAL-003 only	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Percentage of Participants With Positive PEG IgG Antibody	At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156	As PAL-003 is an extension of parent Phase 2 studies (PAL-002, PAL-004 \& 165-205), the parent Phase 2 treatment naïve baseline data (N=80) was used for the vital signs, immunogenicity \& PK analyses to monitor changes in vital signs mediated by pegvaliase, understand development \& maturation of immune response against pegvaliase, \& the resulting impact on PK. Refer to NCT00925054-PAL-002, NCT01212744-PAL-004 \& NCT01560286-165-205 for parent Ph2 demographic data (N=80).	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Percentage of Participants With Positive PAL IgG Antibody	At Baseline and Change from Baseline to Week 24, Week 52, Week 104 and Week 156	As PAL-003 is an extension of parent Phase 2 studies (PAL-002, PAL-004 \& 165-205), the parent Phase 2 treatment naïve baseline data (N=80) was used for the vital signs, immunogenicity \& PK analyses to monitor changes in vital signs mediated by pegvaliase, understand development \& maturation of immune response against pegvaliase, \& the resulting impact on PK. Refer to NCT00925054-PAL-002, NCT01212744-PAL-004 \& NCT01560286-165-205 for parent Ph2 demographic data (N=80).	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group27

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in the Blood Phenylalanine (Phe) Concentration	rAvPAL-PEG	Baseline	1022.4	Standard Deviation 530.39	μmol/L
Change From Baseline in the Blood Phenylalanine (Phe) Concentration	rAvPAL-PEG	Change from Baseline to Week 48	-553.5	Standard Deviation 652.95	μmol/L
Change From Baseline in the Blood Phenylalanine (Phe) Concentration	rAvPAL-PEG	Change from Baseline to Week 96	-653.3	Standard Deviation 637.81	μmol/L
Change From Baseline in the Blood Phenylalanine (Phe) Concentration	rAvPAL-PEG	Change from Baseline to Week 144	-672.3	Standard Deviation 621.06	μmol/L
Change From Baseline in the Blood Phenylalanine (Phe) Concentration	rAvPAL-PEG	Change from Baseline to Week 216	-535.9	Standard Deviation 573.08	μmol/L
Change From Baseline in the Blood Phenylalanine (Phe) Concentration	rAvPAL-PEG	Change from Baseline to Week 240	-574.9	Standard Deviation 549.90	μmol/L
Pharmacokinetics-Plasma Pegvaliase Concentration	rAvPAL-PEG	At Baseline	961.5	Standard Deviation 3013.43	ng/mL
Pharmacokinetics-Plasma Pegvaliase Concentration	rAvPAL-PEG	Week 48	4127.3	Standard Deviation 6116.72	ng/mL
Pharmacokinetics-Plasma Pegvaliase Concentration	rAvPAL-PEG	Week 96	7457.7	Standard Deviation 10685.02	ng/mL
Pharmacokinetics-Plasma Pegvaliase Concentration	rAvPAL-PEG	Week 144	7196.1	Standard Deviation 11678.89	ng/mL

1 to 10 of 27Source ClinicalTrials.gov

See all 27 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	rAvPAL-PEG	68
Overall Study	COMPLETED	rAvPAL-PEG	46
Overall Study	NOT COMPLETED	rAvPAL-PEG	22

Source ClinicalTrials.gov

Adverse events153

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Lymphadenopathy	Blood and lymphatic system disorders	rAvPAL-PEG	9	68	12
other	Injection site reaction	General disorders	rAvPAL-PEG	36	68	114
other	Injection site erythema	General disorders	rAvPAL-PEG	36	68	160
other	Injection site bruising	General disorders	rAvPAL-PEG	34	68	245
other	Pyrexia	General disorders	rAvPAL-PEG	25	68	38
other	Pain in extremity	Musculoskeletal and connective tissue disorders	rAvPAL-PEG	25	68	59
other	Injection site rash	General disorders	rAvPAL-PEG	11	68	24
other	Injection site pruritus	General disorders	rAvPAL-PEG	22	68	83
other	Injection site pain	General disorders	rAvPAL-PEG	18	68	76
other	Injection site swelling	General disorders	rAvPAL-PEG	16	68	25

1 to 10 of 153Source ClinicalTrials.gov

See all 153 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	rAvPAL-PEG	0	68
Total, other adverse events	rAvPAL-PEG	68	68
Total, serious adverse events	rAvPAL-PEG	12	68

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
29973227	DERIVED	PubMed
32593547	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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