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Histone Deacetylase Inhibitor
Phenylbutyrate Oral Tablet for Inclusion Body Myositis
Phase 1
Waitlist Available
Led By Duaa Jabari, MD
Research Sponsored by University of Kansas Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 3 - month 6
Awards & highlights
Summary
This trial is testing phenylbutyrate to see if it is safe for people with a muscle disease called sporadic inclusion body myositis. Ten patients will take the medication for a few months, and researchers will check specific health indicators to see how the drug affects them. Phenylbutyrate has been previously tested in other muscle diseases, such as spinal muscular atrophy.
Eligible Conditions
- Inclusion Body Myositis
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ month 3 - month 6
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 3 - month 6
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
Secondary outcome measures
Inclusion Body Myositis Functional Rating Scale (IBMFRS)
Side effects data
From 2017 Phase 4 trial • 88 Patients • NCT0125773722%
Hyperammonaemia
16%
Upper respiratory tract infection
13%
Vomiting
13%
Headache
9%
Ammonia increased
7%
Diarrhoea
7%
Ear infection
7%
Amino acid level decreased
7%
Cough
7%
Amino acid level increased
7%
Pyrexia
4%
Dehydration
4%
Nausea
4%
Oropharyngeal pain
2%
Gastroenteritis
2%
Influenza
2%
Hypokalaemia
2%
Bronchitis
2%
Sinusitis
2%
Pain
2%
Dizziness
100%
80%
60%
40%
20%
0%
Study treatment Arm
HPN-100- Pediatric
HPN-100 - Adult
Trial Design
1Treatment groups
Experimental Treatment
Group I: PhenylbutyrateExperimental Treatment1 Intervention
Open-label phenylbutyrate
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Phenylbutyrate Oral Tablet
2020
Completed Phase 1
~10
Find a Location
Who is running the clinical trial?
University of Kansas Medical CenterLead Sponsor
472 Previous Clinical Trials
171,429 Total Patients Enrolled
Duaa Jabari, MDPrincipal InvestigatorUniversity of Kansas Medical Center
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