Inclusion Body MyositisPhase 1NCT04421677
Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
A Phase 1 study testing Phenylbutyrate Oral Tablet in people with Inclusion Body Myositis and Sporadic Inclusion Body Myositis, run by University of Kansas Medical Center at 1 site in United States.
Trial summary
This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Inclusion Body Myositis, Sporadic Inclusion Body Myositis
- What is being tested?
- Phenylbutyrate Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Aug 2020to 20 Jan 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Fulfill ENMC 2011 diagnostic criteria for IBM
- Age > 18 years
- Women must be post-menopausal (no menses in >12 months) or status post hysterectomy
- Able to give informed consent
Who cannot join
- Presence of any one of the following medical conditions: chronic infection; chronic renal insufficiency; cancer other than skin cancer less than five years prior; multiple sclerosis or prior episode of central nervous system demyelination; or other chronic serious medical illnesses
- Presence of any of the following on routine blood screening: WBC<3000; Platelets < 100,000; hematocrit < 30%; BUN > 30 mg %; creatine > 1.5 mg%; liver disease with serum albumin < 3 G/DL
- Women who are pregnant or lactating
- History of non-compliance with other therapies
- Coexistence of other muscular disease
- Drug or alcohol abuse within past three months
Show all 11Show fewer
- Known bleeding disorder
- Known liver disease
- Known congestive heart failure
- Known hypernatremia
- Inability to give informed consent
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kansas Medical Center
Kansas City, Kansas, 66160
About this study
This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during which certain biomarkers will be measured at baseline and at the end of the run-in period in addition to final measurement at the end of the treatment period.
- Being tested
- Phenylbutyrate Oral Tablet
- Keywords
- Inclusion Body Myositis, IBM, PBA, Phenylbutyrate
- Medical subject headings
- Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Phenylbutyrate Oral TabletDrug
Also called PBA
Phenylbutyrate, an orally active chemical chaperone approved by the US Food and Drug Administration for treatment of urea cycle disorders, mimics the function of intracellular molecular chaperones in preventing protein aggregation and oligomerization.
| Study group | Role | What they receive |
|---|---|---|
| Phenylbutyrate | Experimental | Open-label phenylbutyrate Receives: Drug: Phenylbutyrate Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
Since this is a Phase I pilot trial, the primary outcome will be testing the safety and tolerability of phenylbutyrate in patients with Inclusion Body Myositis. This will be measured by the incidence of adverse events reported in subjects throughout the treatment period of the trial.
Measured: Month 3 - Month 6
Secondary outcomes (1)
Inclusion Body Myositis Functional Rating Scale (IBMFRS)
The IBMFRS is a quickly administered (10-minute) ordinal rating scale (ratings 0-40) used to determine patients' assessment of their capability and independence in 10 functional activities. The higher a subject scores on the scale, the better functional ability the subject has.
Measured: Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2020
- Study start20 Aug 2020
- Primary completion20 Jan 2022
- Study completion20 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inclusion Body MyositisStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Duaa Jabari, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04421677
- Sponsor's protocol ID
- PBA in IBM
- Status last verified
- May 2022
- First submitted
- 18 May 2020
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
- ClinicalTrials.gov ID
- NCT04421677
- Acronym / sponsor ID
- PBA in IBM
- Keywords
- Inclusion Body Myositis, IBM, PBA, Phenylbutyrate
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Study start
- 20 Aug 2020 (actual)
- Primary completion
- 20 Jan 2022 (actual)
- Study completion
- 20 Jan 2022 (actual)
- First posted
- 9 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during which certain biomarkers will be measured at baseline and at the end of the run-in period in addition to final measurement at the end of the treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Phenylbutyrate | Experimental | Drug: Phenylbutyrate Oral TabletOpen-label phenylbutyrate |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Fulfill ENMC 2011 diagnostic criteria for IBM
- Age > 18 years
- Women must be post-menopausal (no menses in >12 months) or status post hysterectomy
- Able to give informed consent
Key exclusion criteria (11 listed)
- Presence of any one of the following medical conditions: chronic infection; chronic renal insufficiency; cancer other than skin cancer less than five years prior; multiple sclerosis or prior episode of central nervous system demyelination; or other chronic serious medical illnesses
- Presence of any of the following on routine blood screening: WBC<3000; Platelets < 100,000; hematocrit < 30%; BUN > 30 mg %; creatine > 1.5 mg%; liver disease with serum albumin < 3 G/DL
- Women who are pregnant or lactating
- History of non-compliance with other therapies
- Coexistence of other muscular disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Phenylbutyrate Oral TabletAlso: PBAPhenylbutyrate, an orally active chemical chaperone approved by the US Food and Drug Administration for treatment of urea cycle disorders, mimics the function of intracellular molecular chaperones in preventing protein aggregation and oligomerization. | Drug | Phenylbutyrate |
Primary outcome
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
Time frame: Month 3 - Month 6
Since this is a Phase I pilot trial, the primary outcome will be testing the safety and tolerability of phenylbutyrate in patients with Inclusion Body Myositis. This will be measured by the incidence of adverse events reported in subjects throughout the treatment period of the trial.
1 secondary outcome
Inclusion Body Myositis Functional Rating Scale (IBMFRS)
Time frame: Month 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: Undecided
Related trials for Inclusion Body Myositis
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Inclusion Body Myositis (IBM), Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Duaa Jabari, MD principal investigator
University of Kansas Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Phenylbutyrate Oral Tablet in people with Inclusion Body Myositis and Sporadic Inclusion Body Myositis, run by University of Kansas Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events (time frame: Month 3 - Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.