Trial Outcomes & Findings for A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks (NCT NCT01560286)
NCT ID: NCT01560286
Last Updated: 2019-08-06
Results Overview
All patients will have their plasma Phenylalanine (Phe) assessed. Please note that "Pharmacokinetics Sub-study" was not performed, and thus no results are available.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
Baseline, Week 24
Results posted on
2019-08-06
Participant Flow
Participant milestones
| Measure |
BMN 165 (rAvPAL-PEG)
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
24
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
Baseline characteristics by cohort
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Age, Continuous
|
29.3 years
STANDARD_DEVIATION 11.43 • n=5 Participants
|
|
Age, Customized
< 18 years
|
1 Participants
n=5 Participants
|
|
Age, Customized
> or = 18 years
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
24 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
24 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
24 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 24Population: The efficacy population will consist of all subjects who received any amount of study drug and have post-treatment blood Phe concentration measurements.
All patients will have their plasma Phenylalanine (Phe) assessed. Please note that "Pharmacokinetics Sub-study" was not performed, and thus no results are available.
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Blood Phenylalanine Concentration
Baseline
|
1168.8 umol/L
Standard Deviation 290.98
|
|
Blood Phenylalanine Concentration
Week 24
|
617.6 umol/L
Standard Deviation 529.28
|
SECONDARY outcome
Timeframe: Minimum Weekly Assessment of Injection Sites, Vital Signs and Adverse Events. Other Safety Assessments will be performed at other intervals (below):Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Number of Participants With Study Drug Related Adverse Events
|
23 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody Positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive Anti-PAL Immunoglobulin G [IgG]
Baseline
|
4.3 percentage
|
|
Percentage of Participants With Positive Anti-PAL Immunoglobulin G [IgG]
Week 24
|
95.5 percentage
|
SECONDARY outcome
Timeframe: Week 1, Week 24Population: The PK population will consist of all subjects who received any amount of study drug and have post-treatment plasma BMN 165 concentration measurements.
PK assessment from pre-dose blood draw.
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Trough Concentration of BMN 165
Week 1 Day 1
|
0.2 ng/mL
Standard Deviation 1.09
|
|
Trough Concentration of BMN 165
Week 24
|
2341.1 ng/mL
Standard Deviation 4768.28
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody Positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive Anti-PEG IgG
Baseline
|
26.1 percentage
|
|
Percentage of Participants With Positive Anti-PEG IgG
Week 24
|
22.7 percentage
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody Positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive PAL-IgM
Baseline
|
34.8 percentage
|
|
Percentage of Participants With Positive PAL-IgM
Week 24
|
90.9 percentage
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive Anti-PEG-IgM
Baseline
|
4.3 percentage
|
|
Percentage of Participants With Positive Anti-PEG-IgM
Week 24
|
9.1 percentage
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive Neutralizing Antibodies [Nab]
Baseline
|
0 percentage
|
|
Percentage of Participants With Positive Neutralizing Antibodies [Nab]
Week 24
|
27.3 percentage
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive Anti-PAL-IgE Antibodies
Baseline
|
0 percentage
|
|
Percentage of Participants With Positive Anti-PAL-IgE Antibodies
Week 24
|
0 percentage
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety population will consist of all subjects who receive any amount of study drug throughout the study duration.
Antibody positivity
Outcome measures
| Measure |
rAvPAL-PEG
n=24 Participants
All 24 Enrolled Subjects
|
|---|---|
|
Percentage of Participants With Positive Anti-PAL-PEG IgE Antibodies
Baseline
|
0 percentage
|
|
Percentage of Participants With Positive Anti-PAL-PEG IgE Antibodies
Week 24
|
0 percentage
|
Adverse Events
rAvPAL-PEG
Serious events: 1 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
rAvPAL-PEG
n=24 participants at risk
All 24 Enrolled Subjects
|
|---|---|
|
Investigations
Blood creatine phosphokinase increased
|
4.2%
1/24 • Number of events 1 • Week 0- Week 24
|
Other adverse events
| Measure |
rAvPAL-PEG
n=24 participants at risk
All 24 Enrolled Subjects
|
|---|---|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
3/24 • Number of events 3 • Week 0- Week 24
|
|
Gastrointestinal disorders
Dyspepsia
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Gastrointestinal disorders
Nausea
|
33.3%
8/24 • Number of events 9 • Week 0- Week 24
|
|
Gastrointestinal disorders
Toothache
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
3/24 • Number of events 5 • Week 0- Week 24
|
|
General disorders
Injection site bruising
|
41.7%
10/24 • Number of events 13 • Week 0- Week 24
|
|
General disorders
Injection site erythema
|
54.2%
13/24 • Number of events 40 • Week 0- Week 24
|
|
General disorders
Injection site induration
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
General disorders
Injection site pruritus
|
20.8%
5/24 • Number of events 11 • Week 0- Week 24
|
|
General disorders
Injection site rash
|
33.3%
8/24 • Number of events 16 • Week 0- Week 24
|
|
General disorders
Injection site reaction
|
75.0%
18/24 • Number of events 123 • Week 0- Week 24
|
|
General disorders
Injection site swelling
|
12.5%
3/24 • Number of events 5 • Week 0- Week 24
|
|
General disorders
Injection site urticaria
|
8.3%
2/24 • Number of events 3 • Week 0- Week 24
|
|
General disorders
Local swelling
|
8.3%
2/24 • Number of events 4 • Week 0- Week 24
|
|
General disorders
Mass
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
General disorders
Pain
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
General disorders
Pyrexia
|
25.0%
6/24 • Number of events 8 • Week 0- Week 24
|
|
Immune system disorders
Seasonal allergy
|
8.3%
2/24 • Number of events 5 • Week 0- Week 24
|
|
Infections and infestations
Nasopharyngitis
|
16.7%
4/24 • Number of events 4 • Week 0- Week 24
|
|
Infections and infestations
Oral herpes
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Infections and infestations
Rhinovirus infection
|
8.3%
2/24 • Number of events 3 • Week 0- Week 24
|
|
Infections and infestations
Sinusitis
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Infections and infestations
Upper respiratory tract infection
|
37.5%
9/24 • Number of events 10 • Week 0- Week 24
|
|
Investigations
C-reactive protein increased
|
12.5%
3/24 • Number of events 3 • Week 0- Week 24
|
|
Investigations
Complement factor decreased
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Investigations
Red blood cell sedimentation rate increased
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Investigations
Urine albumin/creatinine ratio increased
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
75.0%
18/24 • Number of events 80 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.5%
3/24 • Number of events 8 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
8.3%
2/24 • Number of events 3 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
12.5%
3/24 • Number of events 3 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.5%
3/24 • Number of events 4 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.5%
3/24 • Number of events 3 • Week 0- Week 24
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Nervous system disorders
Dizziness
|
16.7%
4/24 • Number of events 5 • Week 0- Week 24
|
|
Nervous system disorders
Headache
|
29.2%
7/24 • Number of events 12 • Week 0- Week 24
|
|
Nervous system disorders
Sinus headache
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Psychiatric disorders
Anxiety
|
12.5%
3/24 • Number of events 4 • Week 0- Week 24
|
|
Psychiatric disorders
Depression
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.8%
5/24 • Number of events 5 • Week 0- Week 24
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
12.5%
3/24 • Number of events 8 • Week 0- Week 24
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Erythema
|
8.3%
2/24 • Number of events 3 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.8%
5/24 • Number of events 6 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
8/24 • Number of events 13 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
20.8%
5/24 • Number of events 10 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
8.3%
2/24 • Number of events 2 • Week 0- Week 24
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
12.5%
3/24 • Number of events 6 • Week 0- Week 24
|
|
Vascular disorders
Flushing
|
16.7%
4/24 • Number of events 5 • Week 0- Week 24
|
Additional Information
Sr. Medical Director, Clinical Science
BioMarin Pharmaceutical Inc
Phone: 415-475-5854
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60