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Enzyme substitution therapy

Pegvaliase for Phenylketonuria (PEGASUS Trial)

Phase 3
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
If sexually active, male or female participants must not plan to become pregnant (self or partner) and must use 2 acceptable methods of contraception while participating in the study beginning at Screening and for 4 weeks after discontinuing study drug.
If on medication for ADHD, depression, or other psychiatric disorder, stable dose of medication for ≥ 8 weeks prior to enrollment and willing to maintain stable dose unless a change is medically indicated.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 215 weeks
Awards & highlights

PEGASUS Trial Summary

This trial is testing a new medication for PKU in adolescents. It is designed to see if it is safe and effective.

Who is the study for?
This trial is for adolescents aged 12-17 in the US (or 12-15 in the EU) with Phenylketonuria (PKU) who haven't been able to control their blood Phe levels through current treatments. They must be stable on any psychiatric meds, willing to follow a diet plan, and have an adult supervisor during treatment. Sexually active participants need to use two forms of contraception.Check my eligibility
What is being tested?
The study tests Pegvaliase injections against a diet-only approach in managing PKU. It's an open-label Phase 3 trial where about 54 young people are randomly assigned to either get the drug or stick with dietary management to see which is safer and works better.See study design
What are the potential side effects?
While not explicitly listed here, common side effects of pegvaliase may include allergic reactions like anaphylaxis, joint pain, headaches, injection site reactions, and nausea. Participants should also be observed for symptoms after administration due to potential severe allergic responses.

PEGASUS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am using two forms of birth control and do not plan to get pregnant or father a child while in the study.
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I've been on a stable dose of my psychiatric medication for at least 8 weeks and can keep it steady.
Select...
I am able to understand and sign the consent form.
Select...
I have someone 18 or older to watch me for at least 1 hour after I take the study drug.
Select...
I am willing to follow a diet plan supervised by a dietician for the study.
Select...
I have PKU and my blood Phe levels have been too high despite my current treatment.
Select...
I am between 12 and 17 years old if in the US, or 12 to 15 if in the EU.

PEGASUS Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 215 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 215 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in blood Phe concentration
Incidence of treatment-emergent adverse events as assessed by CTCAE v5.0
Secondary outcome measures
Change in total dietary protein intake
Other outcome measures
Characterize area under plasma concentration time curve (AUC) of pegvaliase
Characterize maximum plasma concentration (Cmax) of pegvaliase
Characterize time to reach maximum plasma concentration (Tmax) of pegvaliase
+1 more

Side effects data

From 2021 Phase 3 trial • 37 Patients • NCT03694353
35%
Nasopharyngitis
32%
Headache
19%
Oropharyngeal pain
19%
Influenza
19%
Amino acid level decreased
16%
Nausea
16%
Vomiting
16%
Arthralgia
16%
Cough
16%
Pruritus
16%
Injection site bruising
14%
Pain in extremity
14%
Fatigue
14%
Limb injury
14%
Seasonal allergy
14%
Migraine
14%
Injection site induration
11%
Diarrhoea
11%
Upper respiratory tract infection
11%
Injection site reaction
11%
Gastroenteritis
11%
Nasal congestion
11%
Toothache
11%
Contusion
11%
Alopecia
11%
Urticaria
11%
Dizziness
8%
Neck pain
8%
Sinusitis
8%
Rash
8%
Helicobacter infection
8%
Abdominal pain upper
8%
Depression
8%
Back pain
8%
Pyrexia
5%
Sinus headache
5%
Paraesthesia
5%
Hypertension
5%
Induration
5%
Cellulitis
5%
Conjunctivitis
5%
Ear infection
5%
Pneumonia
5%
Anxiety
5%
Sinus disorder
5%
Paranasal sinus discomfort
5%
Asthenia
5%
Injection site pruritus
5%
Hypoaesthesia
5%
Ligament sprain
5%
Dental caries
5%
Musculoskeletal pain
5%
Tinea pedis
5%
Tooth abscess
5%
Pharyngitis streptococcal
5%
Gastroenteritis viral
5%
Otitis media acute
5%
Gastrooesophageal reflux disease
5%
Bronchitis
5%
Injection site pain
5%
Arthropod bite
5%
Injection site erythema
5%
Pain
5%
Procedural pain
3%
Gastric ulcer haemorrhage
3%
Abortion spontaneous
3%
COVID-19
3%
Wound infection staphylococcal
3%
Ectopic pregnancy
3%
Anaphylactic reaction
3%
Systemic inflammatory response syndrome
3%
Cholecystitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
All Subjects

PEGASUS Trial Design

2Treatment groups
Experimental Treatment
Group I: PegvaliaseExperimental Treatment1 Intervention
Group II: Drug: Diet OnlyExperimental Treatment1 Intervention
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72, initiating pegvaliase treatment beginning Week 73 and, from Weeks 73 through 215.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pegvaliase
2018
Completed Phase 3
~40

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
156 Previous Clinical Trials
190,670 Total Patients Enrolled
Study DirectorStudy ChairBioMarin Pharmaceutical
1,202 Previous Clinical Trials
489,577 Total Patients Enrolled

Media Library

Pegvaliase (Enzyme substitution therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05270837 — Phase 3
Phenylketonuria Research Study Groups: Drug: Diet Only, Pegvaliase
Phenylketonuria Clinical Trial 2023: Pegvaliase Highlights & Side Effects. Trial Name: NCT05270837 — Phase 3
Pegvaliase (Enzyme substitution therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05270837 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

When was Pegvaliase cleared by the FDA?

"Pegvaliase has been shown to be effective in Phase 3 trials and has had multiple rounds of data supporting its safety, so it is estimated to be a safe medication."

Answered by AI

How many individuals are being studied in this clinical trial?

"In order to move forward, this study requires 54 willing and eligible patients. If you qualify and are interested, know that you can participate in this trial at The Community Health Clinic in Topeka, Indiana and McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas."

Answered by AI

Will elderly patients be accepted into this clinical trial?

"From the eligibility requirements, it can be inferred that the ideal participants for this trial fall between the ages of 12 to 17. Out of the 16 available clinical trials, 13 are for people over the age of 65."

Answered by AI

Is this research being conducted in numerous hospitals across Canada?

"The Community Health Clinic in Topeka, Indiana, McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas, and University of Virginia School of Medicine in Charlottesville, Virginia are a few of the 20 active sites for this clinical trial."

Answered by AI

Could you explain the inclusion criteria for this trial?

"This study is accepting 54 people with phenylketonurias aged 12 and 17. Most notable, applicants are required to meet the following criteria: Willing and able to maintain and adjust dietary and medical protein food intake according to the study protocol under the supervision of a study dietician or adequately trained designee per investigator discretion during study participation., Is 12 to 17 years old (US), inclusive, or 12 to 15 years (EU), inclusive, at the start of the Screening/Run-in Period (Day -28)., Diagnosis of PKU and failure to maintain recommended blood Phe levels on existing management ("

Answered by AI
~16 spots leftby Jan 2025