RAVICTI for Urea Cycle Disorders, Inborn

Phase-Based Progress Estimates
3
Effectiveness
3
Safety
Urea Cycle Disorders, Inborn+1 More
RAVICTI - Drug
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

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 Disorders, Inborn
  • Enzymes

Treatment Effectiveness

Effectiveness Progress

3 of 3
This is further along than 93% of similar trials

Other trials for Urea Cycle Disorders, Inborn

Study Objectives

1 Primary · 18 Secondary · Reporting Duration: Day 1, Week 1, 2, 3, 4, 5, 9, 13, 17, 21, 25

Baseline through Week 25
Rate of Drug Discontinuations Due to Adverse Events
Week 25
Rate of Adverse Events
Baseline through Week 4
Rate of Drug Discontinuations Due to Any Reasons
Week 13
Clinical Global Impression (CGI) Scales Improvement (Investigator Only)
Clinical Global Impression (CGI) Scales Severity (Investigator Only)
Neuropsychological Assessment Adult Self Report (ASR)
Neuropsychological Checklist Adult Behavior (ABCL)
Neuropsychological Checklists Child Behavior (CBCL)
Week 4
EQ-5D-5L Health Status Quality of Life
Week 1
Amino Acid Assessment
Plasma Ammonia Level
Week 25
Annualized Rate of Hyperammonemic Crisis (HAC)
Week 4
Plasma Concentration of Phenylacetic Acid (PAA)
Plasma Concentration of Phenylacetylglutamine (PAGN)
Plasma Concentration of Phenylbutyric Acid (PBA)
Rate of Treatment Success
Urinary Excretion of Phenylacetylglutamine (PAGN)
Week 5
Palatability of Study Drug (Hedonic Scale)
Week 5
Drug preference (NaPBA arm only)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Urea Cycle Disorders, Inborn

Side Effects for

RAVICTI: Age 2 Months to < 2 Years
40%Upper respiratory tract infection
40%Vomiting
30%Hyperammonaemia
20%Viral infection
20%Constipation
20%Pyrexia
20%Hypophagia
20%Cough
20%Rash
20%Metabolic acidosis
20%Nasopharyngitis
20%Otitis media
10%Urinary tract infection
10%Cyanosis
10%Influenza
10%Eczema
10%Pneumatosis intestinalis
10%Vesicoureteric reflux
10%Apnoea
10%Pharyngitis
10%Rhinovirus infection
10%Teething
10%Gastroenteritis
10%Peritonitis
10%Viral upper respiratory tract infection
10%Croup infectious
10%Asthma
10%Status asthmaticus
10%Apnoeic attack
10%Ammonia increased
10%Venous thrombosis limb
10%Seasonal allergy
10%Carbon dioxide decreased
10%Stoma site reaction
10%Anaemia
10%Tibia fracture
10%Dacryostenosis congenital
10%Excessive cerumen production
10%Amino acid level decreased
10%Nail ridging
10%Rhinorrhoea
10%Diarrhoea
10%Gross motor delay
10%Nasal congestion
10%Dermatitis diaper
10%Respiratory disorder
10%Pneumonia
10%Sinusitis
10%Conjunctivitis
This histogram enumerates side effects from a completed 2017 Phase 4 trial (NCT02246218) in the RAVICTI: Age 2 Months to < 2 Years ARM group. Side effects include: Upper respiratory tract infection with 40%, Vomiting with 40%, Hyperammonaemia with 30%, Viral infection with 20%, Constipation with 20%.

Trial Design

2 Treatment Groups

Arm 2, NaPBA (sodium phenylbutyrate)
1 of 2
Arm 1, RAVICTI
1 of 2
Active Control
Experimental Treatment

18 Total Participants · 2 Treatment Groups

Primary Treatment: RAVICTI · No Placebo Group · Phase 4

Arm 1, RAVICTI
Drug
Experimental Group · 1 Intervention: RAVICTI · Intervention Types: Drug
Arm 2, NaPBA (sodium phenylbutyrate)
Drug
ActiveComparator Group · 1 Intervention: NaPBA · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Glycerol phenylbutyrate
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day 1, week 1, 2, 3, 4, 5, 9, 13, 17, 21, 25

Who is running the clinical trial?

Horizon Therapeutics, LLCLead Sponsor
3 Previous Clinical Trials
318 Total Patients Enrolled
3 Trials studying Urea Cycle Disorders, Inborn
318 Patients Enrolled for Urea Cycle Disorders, Inborn
Colleen Canavan, BSStudy DirectorHorizon Therapeutics, LLC
5 Previous Clinical Trials
301 Total Patients Enrolled
2 Trials studying Urea Cycle Disorders, Inborn
115 Patients Enrolled for Urea Cycle Disorders, Inborn

Eligibility Criteria

Age Any Age · All Participants · 6 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have experienced a HAC or a documented high ammonia of ≥100 µmol/L.
You have a confirmed diagnosis of a specific disease.
You must have signed an informed consent form before participating in this study.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 20th, 2021

Last Reviewed: October 26th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.