Sporadic Inclusion Body MyositisPhase 2NCT03710941
Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
A Phase 2 study testing REGN2477+REGN1033 in people with Sporadic Inclusion Body Myositis, run by Regeneron Pharmaceuticals.
Trial summary
The primary objective of the study is to evaluate the efficacy of REGN2477+REGN1033 in combination on total lean mass, as measured by Dual-energy X-ray absorptiometry (DXA) in patients with sporadic inclusion body myositis (sIBM). Read more about this study
- Who is it for?
- Ages 45 to 75 years
- Conditions the study is for
- Sporadic Inclusion Body Myositis
- What is being tested?
- REGN2477+REGN1033
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 19 Feb 2019to 5 Nov 2020
- Phase
- Phase 2
- 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 45 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and postmenopausal* women
- Diagnosed with sIBM based on the European Neuromuscular Centre (ENMC) IBM Research Diagnostic Criteria
- Ability to walk ≥150 meters, with or without a walking aid such as cane or walker, in 6 minutes.
- Ability to climb 4 steps of stairs unassisted (may use handrails)
- Willing and able to comply with clinic visits and study-related procedures
Who cannot join
- Other neurological conditions (eg, hemiplegia post stroke, Parkinson's) or musculo-skeletal conditions (eg, severe osteoarthritis) causing mobility impairment
- Mini-Mental State Examination (MMSE) score <24
- Ongoing, chronic, high-dose (>20 mg prednisone equivalent per day), systemic corticosteroid therapy within 6 weeks prior to screening.
- Any condition that precludes adequate intake of energy and protein; malnutrition; presence of an eating disorder.
- Unintentional weight loss of ≥10% in the past 6 months (patient-reported)
- Hospitalization for heart failure in last year or New York Heart Association Class 4
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- History of hypertrophic cardiomyopathy
- Any drugs known to influence muscle mass and performance such as anabolic steroids or growth hormone within 6 weeks prior to screening
- Unable to fit on the site's DXA scanner table, within borders for scanning of total lean mass
- Note: Other protocol Inclusion/Exclusion criteria apply
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Intravenously Administered REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
The primary objective of the study is to evaluate the efficacy of REGN2477+REGN1033 in combination on total lean mass, as measured by Dual-energy X-ray absorptiometry (DXA) in patients with sporadic inclusion body myositis (sIBM).
The secondary objectives of the study are:
- To evaluate the efficacy of REGN2477+REGN1033 on the IBM-Functional Rating Scale (IBM-FRS)
- To evaluate the efficacy of REGN2477+REGN1033 on the sIBM Physical Functioning Assessment (sIFA)
- To evaluate the safety and tolerability of REGN2477+REGN1033
- To evaluate the effects of REGN2477+REGN1033 on body composition by DXA, including appendicular lean mass and total fat mass
- To evaluate the efficacy of REGN2477+REGN1033 on measures of muscle performance and physical function
- To evaluate the efficacy of REGN2477+REGN1033 on patient reported outcome measures including the fear of falling, falls and near falls, and health-related quality of life
- To evaluate the pharmacokinetic(s) (PK) profile of REGN2477+REGN1033, including functional REGN2477 and functional REGN1033 concentrations in serum over time
- To evaluate the immunogenicity of REGN2477+REGN1033
- Conditions
- Sporadic Inclusion Body Myositis
- Being tested
- REGN2477+REGN1033, Matching placebo
- 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.
REGN2477+REGN1033Drug
Single, sequential, repeat-dose Intravenous (IV) or matching placebo
Matching placeboDrug
Single, sequential, repeat-dose Intravenous (IV)
| Study group | Role | What they receive |
|---|---|---|
| REGN2477+REGN1033 | Experimental | Single, sequential, repeat-dose IV or matching placebo Receives: Drug: REGN2477+REGN1033 |
| Placebo | Experimental | Single, sequential, repeat-dose IV Receives: Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)
Measured: Up to Week 20
Secondary outcomes (22)
Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)
Measured: Up to Week 26
Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)
Measured: Up to Week 26
Incidence and severity of treatment-emergent adverse events (TEAEs)
Measured: Up to Week 30
Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA
Measured: Up to Week 30
Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA
Measured: Up to Week 30
Change in the 1-repetition maximum (1-RM) chest press strength
Measured: Up to Week 26
Change in hand-grip strength as measured by dynamometry
Measured: Up to Week 26
Change in distance walked in the 6-minute walk test (6MWT)
Measured: Up to Week 26
Change in time to complete the 10-meter walk test (10MWT)
Measured: Up to Week 26
Change in instrumented stair climb power
Measured: Up to Week 26
Change in instrumented, sensor-based sit-to-stand test time
Measured: Up to Week 26
Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I)
Measured: Up to Week 30
Change in Patient Global Impression of Severity assessments (PGIS/PGIC)
Measured: Up to Week 30
Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)
Measured: Up to Week 30
Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)
Measured: Up to Week 30
Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)
Measured: Up to Week 30
Change in 36-item Short Form Health Survey (SF-36), including the Physical Function Items (PF-10) and the Vitality Scale of the SF-36
Measured: Up to Week 30
Change in Thigh muscle volume as measured by MRI (sub-study)
Measured: Up to Week 30
Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale
Measured: Up to Week 30
Pharmacokinetics (PK) profile of REGN2477
Assessed via serum concentration of REGN2477 over time
Measured: Up to Week 30
Pharmacokinetics (PK) profile of REGN1033
Assessed via serum concentration of REGN1033 over time
Measured: Up to Week 30
Immunogenicity of REGN2477+REGN1033
As determined by the presence or absence of anti-drug antibodies (ADA)
Measured: Up to Week 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2018
- Study start19 Feb 2019 (estimated)
- Primary completion27 Aug 2020 (estimated)
- Study completion5 Nov 2020 (estimated)
Why it stopped: The study was withdrawn due to an internal decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sporadic Inclusion Body MyositisStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03710941
- Sponsor's protocol ID
- R2477-1033-sIBM-1828
- Other IDs
- 2018-002853-31, 2018
- Status last verified
- Apr 2019
- First submitted
- 15 Oct 2018
- Last updated
- 29 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Intravenously Administered REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
- ClinicalTrials.gov ID
- NCT03710941
- Acronym / sponsor ID
- R2477-1033-sIBM-1828
- Conditions
- Sporadic Inclusion Body Myositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study was withdrawn due to an internal decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 19 Feb 2019 (estimated)
- Primary completion
- 27 Aug 2020 (estimated)
- Study completion
- 5 Nov 2020 (estimated)
- First posted
- 18 Oct 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.
The primary objective of the study is to evaluate the efficacy of REGN2477+REGN1033 in combination on total lean mass, as measured by Dual-energy X-ray absorptiometry (DXA) in patients with sporadic inclusion body myositis (sIBM).
The secondary objectives of the study are:
- To evaluate the efficacy of REGN2477+REGN1033 on the IBM-Functional Rating Scale (IBM-FRS)
- To evaluate the efficacy of REGN2477+REGN1033 on the sIBM Physical Functioning Assessment (sIFA)
- To evaluate the safety and tolerability of REGN2477+REGN1033
- To evaluate the effects of REGN2477+REGN1033 on body composition by DXA, including appendicular lean mass and total fat mass
- To evaluate the efficacy of REGN2477+REGN1033 on measures of muscle performance and physical function
- To evaluate the efficacy of REGN2477+REGN1033 on patient reported outcome measures including the fear of falling, falls and near falls, and health-related quality of life
- To evaluate the pharmacokinetic(s) (PK) profile of REGN2477+REGN1033, including functional REGN2477 and functional REGN1033 concentrations in serum over time
- To evaluate the immunogenicity of REGN2477+REGN1033
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| REGN2477+REGN1033 | Experimental | Drug: REGN2477+REGN1033Single, sequential, repeat-dose IV or matching placebo |
| Placebo | Experimental | Drug: Matching placeboSingle, sequential, repeat-dose IV |
Who may qualify
- Ages
- 45 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Men and postmenopausal* women
- Diagnosed with sIBM based on the European Neuromuscular Centre (ENMC) IBM Research Diagnostic Criteria
- Ability to walk ≥150 meters, with or without a walking aid such as cane or walker, in 6 minutes.
- Ability to climb 4 steps of stairs unassisted (may use handrails)
- Willing and able to comply with clinic visits and study-related procedures
Key exclusion criteria (10 listed)
- Other neurological conditions (eg, hemiplegia post stroke, Parkinson's) or musculo-skeletal conditions (eg, severe osteoarthritis) causing mobility impairment
- Mini-Mental State Examination (MMSE) score <24
- Ongoing, chronic, high-dose (>20 mg prednisone equivalent per day), systemic corticosteroid therapy within 6 weeks prior to screening.
- Any condition that precludes adequate intake of energy and protein; malnutrition; presence of an eating disorder.
- Unintentional weight loss of ≥10% in the past 6 months (patient-reported)
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 |
|---|---|---|
| REGN2477+REGN1033Single, sequential, repeat-dose Intravenous (IV) or matching placebo | Drug | REGN2477+REGN1033 |
| Matching placeboSingle, sequential, repeat-dose Intravenous (IV) | Drug | Placebo |
Primary outcome
Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA)
Time frame: Up to Week 20
22 secondary outcomes
Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS)
Time frame: Up to Week 26
Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA)
Time frame: Up to Week 26
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time frame: Up to Week 30
Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA
Time frame: Up to Week 30
Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA
Time frame: Up to Week 30
Change in the 1-repetition maximum (1-RM) chest press strength
Time frame: Up to Week 26
Change in hand-grip strength as measured by dynamometry
Time frame: Up to Week 26
Change in distance walked in the 6-minute walk test (6MWT)
Time frame: Up to Week 26
Change in time to complete the 10-meter walk test (10MWT)
Time frame: Up to Week 26
Change in instrumented stair climb power
Time frame: Up to Week 26
Change in instrumented, sensor-based sit-to-stand test time
Time frame: Up to Week 26
Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I)
Time frame: Up to Week 30
Change in Patient Global Impression of Severity assessments (PGIS/PGIC)
Time frame: Up to Week 30
Change in Patient Global Impression of Change Items assessments (PGIS/PGIC)
Time frame: Up to Week 30
Change in Clinician Global Impression of Severity assessments (CGIS/CGIC)
Time frame: Up to Week 30
Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC)
Time frame: Up to Week 30
Change in 36-item Short Form Health Survey (SF-36), including the Physical Function Items (PF-10) and the Vitality Scale of the SF-36
Time frame: Up to Week 30
Change in Thigh muscle volume as measured by MRI (sub-study)
Time frame: Up to Week 30
Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale
Time frame: Up to Week 30
Pharmacokinetics (PK) profile of REGN2477
Time frame: Up to Week 30
Pharmacokinetics (PK) profile of REGN1033
Time frame: Up to Week 30
Immunogenicity of REGN2477+REGN1033
Time frame: Up to Week 30
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2019.
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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.
Clinical Trial Management study director
Regeneron Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing REGN2477+REGN1033 in people with Sporadic Inclusion Body Myositis, run by Regeneron Pharmaceuticals.
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 5 inclusion and 10 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: withdrawn, as of the sponsor's last update on 29 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was withdrawn before enrolling participants, so there are no results.
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?
Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA) (time frame: Up to Week 20). 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 29 Apr 2019.