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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
Diagnosis of PKU and failure to maintain recommended blood Phe levels on existing management (sapropterin dihydrochloride and Phe-restricted diet) demonstrated by 2 blood Phe concentration measurements > 600 μmol/L during the Screening/Run-in Period (7 to 10 days in between blood Phe assessments) and average blood Phe concentration > 600 μmol/L over the past 12 months (per available data).
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).
Must not have
A history of organ transplantation or on chronic immunosuppressive therapy.
Previous treatment with pegvaliase.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial is testing pegvaliase injections in adolescents with PKU. The goal is to see if these injections are safe and effective. Pegvaliase helps the body break down a harmful substance that people with PKU cannot process on their own. Pegvaliase is a newer treatment for PKU, following earlier treatments like dietary restriction and sapropterin.

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.
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.
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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have had an organ transplant or am on long-term immune-weakening medication.
Select...
I have been treated with pegvaliase before.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2021 Phase 3 trial • 37 Patients • NCT03694353
35%
Nasopharyngitis
32%
Headache
19%
Amino acid level decreased
19%
Oropharyngeal pain
19%
Influenza
16%
Pruritus
16%
Nausea
16%
Vomiting
16%
Arthralgia
16%
Cough
16%
Injection site bruising
14%
Limb injury
14%
Migraine
14%
Pain in extremity
14%
Seasonal allergy
14%
Fatigue
14%
Injection site induration
11%
Contusion
11%
Nasal congestion
11%
Diarrhoea
11%
Toothache
11%
Upper respiratory tract infection
11%
Injection site reaction
11%
Gastroenteritis
11%
Alopecia
11%
Urticaria
11%
Dizziness
8%
Neck pain
8%
Depression
8%
Sinusitis
8%
Rash
8%
Helicobacter infection
8%
Abdominal pain upper
8%
Back pain
8%
Pyrexia
5%
Bronchitis
5%
Gastrooesophageal reflux disease
5%
Injection site pain
5%
Paraesthesia
5%
Sinus headache
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%
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

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Phenylketonuria (PKU) is managed mainly through dietary restriction of phenylalanine to prevent cognitive impairment and other complications. Pegvaliase, an enzyme substitution therapy, breaks down phenylalanine in the bloodstream, reducing its levels. This treatment is crucial for PKU patients as it provides an alternative to strict dietary management, potentially enhancing quality of life and adherence to treatment.
Progress in experimental phenylketonuria: a critical review.

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
158 Previous Clinical Trials
114,947 Total Patients Enrolled
Study DirectorStudy ChairBioMarin Pharmaceutical
1,268 Previous Clinical Trials
503,791 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
~4 spots leftby Jan 2025