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Phenylacetate Prodrug

RAVICTI for Urea Cycle Deficiencies

Phase 4
Waitlist Available
Research Sponsored by Horizon Therapeutics, LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, initial treatment period week 1, week 2, week 3, week 4 (0, 4, 8 hours post dose)
Awards & highlights

Study Summary

This trial will compare the safety and tolerability of two drugs for urea cycle disorders. It will last 25 weeks and include periods where subjects take one drug, then the other, and then both drugs.

Eligible Conditions
  • Urea Cycle Deficiencies

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, initial treatment period week 1, week 2, week 3, week 4 (0, 4, 8 hours post dose)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, initial treatment period week 1, week 2, week 3, week 4 (0, 4, 8 hours post dose) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period
Secondary outcome measures
Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period
Peak Plasma Concentration (Cmax) of Ammonia at the End of the Initial Treatment Period
Plasma Ammonia Area Under the Curve (AUC) 0 to 8h at the End of the Initial Treatment Period
+1 more

Side effects data

From 2017 Phase 4 trial • 27 Patients • NCT02246218
40%
Vomiting
40%
Upper respiratory tract infection
30%
Hyperammonaemia
20%
Constipation
20%
Pyrexia
20%
Otitis media
20%
Nasopharyngitis
20%
Viral infection
20%
Cough
20%
Metabolic acidosis
20%
Hypophagia
20%
Rash
10%
Urinary tract infection
10%
Viral upper respiratory tract infection
10%
Rhinovirus infection
10%
Influenza
10%
Pneumatosis intestinalis
10%
Sinusitis
10%
Venous thrombosis limb
10%
Diarrhoea
10%
Croup infectious
10%
Amino acid level decreased
10%
Status asthmaticus
10%
Conjunctivitis
10%
Nail ridging
10%
Stoma site reaction
10%
Vesicoureteric reflux
10%
Gross motor delay
10%
Excessive cerumen production
10%
Tibia fracture
10%
Apnoeic attack
10%
Teething
10%
Cyanosis
10%
Dacryostenosis congenital
10%
Anaemia
10%
Asthma
10%
Gastroenteritis
10%
Peritonitis
10%
Rhinorrhoea
10%
Apnoea
10%
Nasal congestion
10%
Respiratory disorder
10%
Eczema
10%
Pharyngitis
10%
Pneumonia
10%
Dermatitis diaper
10%
Seasonal allergy
10%
Ammonia increased
10%
Carbon dioxide decreased
100%
80%
60%
40%
20%
0%
Study treatment Arm
RAVICTI: Age 2 Months to < 2 Years
RAVICTI: Age 0 to <2 Months

Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: RAVICTI -> RAVICTIExperimental Treatment1 Intervention
Initial Treatment, Maintenance, Safety Extension Periods: RAVICTI, Oral Liquid Product 17.5 mL maximum total daily dose. Dosing will be based on participants disease and treatment status at entry to the study.
Group II: NaPBA -> RAVICTIActive Control1 Intervention
Initial Treatment Period: NaPBA dosing based on participants disease and treatment status at entry to the study. Transition, Maintenance, Safety Extension Periods: RAVICTI, Oral Liquid Product 17.5 mL maximum total daily dose. Dosing will be based on participants disease and treatment status at entry to the study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
RAVICTI
2018
Completed Phase 4
~50

Find a Location

Who is running the clinical trial?

Horizon Therapeutics, LLCLead Sponsor
3 Previous Clinical Trials
318 Total Patients Enrolled
Colleen Canavan, BSStudy DirectorHorizon Therapeutics, LLC
5 Previous Clinical Trials
301 Total Patients Enrolled
Denise CoughlanStudy DirectorHorizon Therapeutics, LLC
1 Previous Clinical Trials
11 Total Patients Enrolled
~2 spots leftby Jun 2025
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