MyositisPhase 1NCT03440034
Study of Pioglitazone in Sporadic Inclusion Body Myositis
A Phase 1 study testing Pioglitazone in people with Myositis and Inclusion Body Myositis, run by Johns Hopkins University at 1 site in United States.
Trial summary
A study looking at the effect of pioglitazone in skeletal muscle of patients with sporadic inclusion body myositis (sIBM). Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Myositis, Inclusion Body Myositis, Muscular Diseases
- What is being tested?
- Pioglitazone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- 22 May 2018to 31 Dec 2020
- 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 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 50 years
- Diagnosis of sporadic inclusion body myositis (sIBM) based on the sIBM Diagnostic Criteria established by the 2010 European Neuromuscular Center.
- Must be able to ambulate at least 20 feet, with or without the use of an assistive device. Patients may not use another person, wall, or furniture for support.
- Must be able to rise from a chair without support from another person or device.
- Premenopausal women must have a negative serum pregnancy test prior to dosing with study medication.
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation.
Who cannot join
- A history of diabetes mellitus, or prior or concurrent treatment with any diabetes therapy
- Use of chronic immunosuppressive therapy including corticosteroids or intravenous immune globulin (IVIG) within the past 6 months.
- Use of Vitamin E supplements within the past 3 months
- Creatine kinase (CK) > 15x the upper limit of normal
- Any condition other than sIBM that causes significant muscle pain, muscle weakness, muscle atrophy, or joint pain. This includes but is not limited to such neurologic and neuromuscular diseases as polymyositis or dermatomyositis, myasthenia gravis, amyotrophic lateral sclerosis, stroke, multiple sclerosis, epilepsy, muscular dystrophy, fibromyalgia, rheumatoid arthritis, spinal cord injury or degenerative disease of the spine. Osteoarthritis is not exclusionary unless it limits the patient's ability to comply with study tasks. Patients with a history of a hip or vertebral fracture within the past year or surgical hip or knee replacement within the past six months will be excluded.
- Patients receiving any medications or substances that are inhibitors or inducers of CYP2C8 are ineligible. These medications include but are not limited to Gemfibrozil, Rifampin, and warfarin.
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- Pregnant women
- History of cancer less than five years prior, other than local basal or squamous cell cancer.
- Patient has any medical condition or laboratory finding during screening, which, in the investigator's opinion may interfere with participation, confound the results, or pose any additional risk to the patient.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label Pilot Study of Pioglitazone in Sporadic Inclusion Body Myositis
A study looking at the effect of pioglitazone in skeletal muscle of patients with sporadic inclusion body myositis (sIBM).
Read the detailed description
This is a 52-week, Phase 1, open-label, single center, proof of concept study of FDA-approved pioglitazone in adult patients with sporadic inclusion body myositis (sIBM). The trial consists of a 4-week screening period;16-week "lead-in" period during which all subjects are observed off-treatment. At Week 16, all subjects will be started on pioglitazone at a dose of 30 mg daily. The dose will be uptitrated to a goal dose of 45 mg daily after 2 weeks; 32-week treatment period with all subjects on 45 mg daily dose of pioglitazone.
- Conditions
- Myositis, Inclusion Body Myositis, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases
- Being tested
- Pioglitazone
- Keywords
- IBM, inclusion body myositis
- Medical subject headings
- Myositis, Myositis, Inclusion Body, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PioglitazoneDrug
Also called Actos
Pioglitazone comes as a tablet to take by mouth and can be taken with or without food.
| Study group | Role | What they receive |
|---|---|---|
| Pioglitazone | Experimental | All subjects will be provided Pioglitazone 15mg tablets, total dose of 45mg (3 tablets) for oral administration once daily. 32-week treatment period. Receives: Drug: Pioglitazone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Peroxisome proliferator-activated receptor gamma coactivator 1-alpha target gene expression
Effect of 45 milligram pioglitazone dose on the expression of Peroxisome proliferator-activated receptor gamma coactivator 1-alpha target genes
Measured: 4 weeks, 16 weeks, 32 weeks after baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2018
- Study start22 May 2018
- Primary completion31 Dec 2020
- Study completion31 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MyositisStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe 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
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label, non-randomized trial in 15 patients with sIBM
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jemima Albayda, MD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03440034
- Sponsor's protocol ID
- IRB00130996
- Status last verified
- Aug 2020
- First submitted
- 14 Nov 2017
- Last updated
- 12 Jan 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Adler BL, Bene MR, Zhang C, Say C, Khare P, Mecoli C, Tiniakou E, Paik JJ, Chung T, Darrah E, Lloyd T, Pagkatipunan R, McGowan M, Mears A, Vernon HJ, Christopher-Stine L, Le A, Albayda J. Modifying muscle metabolic dysregulation in inclusion body myositis with pioglitazone: a single-arm trial. Nat Commun. 2026 Mar 15;17(1):3995. doi: 10.1038/s41467-026-70262-0.
PubMed 41832158
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Pilot Study of Pioglitazone in Sporadic Inclusion Body Myositis
- ClinicalTrials.gov ID
- NCT03440034
- Acronym / sponsor ID
- IRB00130996
- Conditions
- Myositis, Inclusion Body Myositis, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases
- Keywords
- IBM, inclusion body myositis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Study start
- 22 May 2018 (actual)
- Primary completion
- 31 Dec 2020 (actual)
- Study completion
- 31 Dec 2020 (actual)
- First posted
- 20 Feb 2018 (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.
A study looking at the effect of pioglitazone in skeletal muscle of patients with sporadic inclusion body myositis (sIBM).
Detailed description
This is a 52-week, Phase 1, open-label, single center, proof of concept study of FDA-approved pioglitazone in adult patients with sporadic inclusion body myositis (sIBM). The trial consists of a 4-week screening period;16-week "lead-in" period during which all subjects are observed off-treatment. At Week 16, all subjects will be started on pioglitazone at a dose of 30 mg daily. The dose will be uptitrated to a goal dose of 45 mg daily after 2 weeks; 32-week treatment period with all subjects on 45 mg daily dose of pioglitazone.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label, non-randomized trial in 15 patients with sIBM
| Arm | Type | Receives |
|---|---|---|
| Pioglitazone | Experimental | Drug: PioglitazoneAll subjects will be provided Pioglitazone 15mg tablets, total dose of 45mg (3 tablets) for oral administration once daily. 32-week treatment period. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age ≥ 50 years
- Diagnosis of sporadic inclusion body myositis (sIBM) based on the sIBM Diagnostic Criteria established by the 2010 European Neuromuscular Center.
- Must be able to ambulate at least 20 feet, with or without the use of an assistive device. Patients may not use another person, wall, or furniture for support.
- Must be able to rise from a chair without support from another person or device.
- Premenopausal women must have a negative serum pregnancy test prior to dosing with study medication.
Key exclusion criteria (9 listed)
- A history of diabetes mellitus, or prior or concurrent treatment with any diabetes therapy
- Use of chronic immunosuppressive therapy including corticosteroids or intravenous immune globulin (IVIG) within the past 6 months.
- Use of Vitamin E supplements within the past 3 months
- Creatine kinase (CK) > 15x the upper limit of normal
- Any condition other than sIBM that causes significant muscle pain, muscle weakness, muscle atrophy, or joint pain. This includes but is not limited to such neurologic and neuromuscular diseases as polymyositis or dermatomyositis, myasthenia gravis, amyotrophic lateral sclerosis, stroke, multiple sclerosis, epilepsy, muscular dystrophy, fibromyalgia, rheumatoid arthritis, spinal cord injury or degenerative disease of the spine. Osteoarthritis is not exclusionary unless it limits the patient's ability to comply with study tasks. Patients with a history of a hip or vertebral fracture within the past year or surgical hip or knee replacement within the past six months will be excluded.
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 |
|---|---|---|
| PioglitazoneAlso: ActosPioglitazone comes as a tablet to take by mouth and can be taken with or without food. | Drug | Pioglitazone |
Primary outcome
Change from baseline in Peroxisome proliferator-activated receptor gamma coactivator 1-alpha target gene expression
Time frame: 4 weeks, 16 weeks, 32 weeks after baseline
Effect of 45 milligram pioglitazone dose on the expression of Peroxisome proliferator-activated receptor gamma coactivator 1-alpha target genes
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 12 Jan 2021.
- Derived from this study: PubMed 41832158Adler BL, Bene MR, Zhang C, Say C, Khare P, Mecoli C, Tiniakou E, Paik JJ, Chung T, Darrah E, Lloyd T, Pagkatipunan R, McGowan M, Mears A, Vernon HJ, Christopher-Stine L, Le A, Albayda J. Modifying muscle metabolic dysregulation in inclusion body myositis with pioglitazone: a single-arm trial. Nat Commun. 2026 Mar 15;17(1):3995. doi: 10.1038/s41467-026-70262-0.
- Individual participant dataSharing planned: No
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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.
Jemima Albayda, MD principal investigator
Johns Hopkins University
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 Pioglitazone in people with Myositis and Inclusion Body Myositis, run by Johns Hopkins University 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 6 inclusion and 9 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 12 Jan 2021. 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?
Change from baseline in Peroxisome proliferator-activated receptor gamma coactivator 1-alpha target gene expression (time frame: 4 weeks, 16 weeks, 32 weeks after baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03440034, which also lists 1 linked publication.
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 12 Jan 2021.