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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 weeks
Treatment ~ Varies
Follow Up ~month 3 - month 6
This trial's timeline: 3 weeks for screening, Varies for treatment, and month 3 - month 6 for reporting.

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 • NCT01257737
22%
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
~2 spots leftby Sep 2025
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