Sporadic Inclusion Body MyositisPhase 2/3RESILIENTNCT01925209

Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients

A Phase 2/3 study testing BYM338/bimagrumab in people with Sporadic Inclusion Body Myositis, run by Novartis Pharmaceuticals at 38 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic inclusion body myositis (sIBM). Read more about this study

Who is it for?: Ages 36 to 85 years
Conditions the study is for: Sporadic Inclusion Body Myositis
What is being tested?: BYM338/bimagrumab
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 251 participants
Where: 38 sitesin 10 countries
Study dates: 26 Sep 2013to 6 Jan 2016
Phase: Phase 2/3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 36 to 85 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Diagnosed with sporadic inclusion body myositis;
Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);

Who cannot join

Must not have other conditions that significantly limit ability to move around;
Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months;
Must meet cardiovascular requirements;
Must not be pregnant or nursing;
Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc.);

Trial locations and contacts

38 sites in 10 countries.

Country

City, postcode or hospital

38 sites in 10 countries

United States12 sites

Novartis Investigative Site
Baltimore, Maryland, 21287
Novartis Investigative Site
Boston, Massachusetts, 02114
Novartis Investigative Site
Boston, Massachusetts, 02115
Novartis Investigative Site
Columbus, Ohio, 43221
Novartis Investigative Site
Dallas, Texas, 75235
Novartis Investigative Site
Houston, Texas, 77030
Novartis Investigative Site
Kansas City, Kansas, 66160
Novartis Investigative Site
Miami, Florida, 33101
Novartis Investigative Site
Orange, California, 92868
Novartis Investigative Site
Phoenix, Arizona, 85028
Novartis Investigative Site
Portland, Oregon, 97239
Novartis Investigative Site
Sacramento, California, 95817

Other trials in United States

Japan7 sites

Novartis Investigative Site
Kodaira, Tokyo, 187-8551
Novartis Investigative Site
Kumamoto, Kumamoto, 860-8556
Novartis Investigative Site
Nagoya, Aichi-ken, 466-8560
Novartis Investigative Site
Osaka, Osaka, 534-0021
Novartis Investigative Site
Sendai, Miyagi, 980-8574
Novartis Investigative Site
Tokushima, Tokushima, 770-8503
Novartis Investigative Site
Wakayama, Wakayama, 641-8510

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Italy5 sites

Novartis Investigative Site
Brescia, BS, 25123
Novartis Investigative Site
Messina, ME, 98125
Novartis Investigative Site
Milan, MI, 20133
Novartis Investigative Site
Padova, PD, 35128
Novartis Investigative Site
Rome, Lazio, 00168

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Australia3 sites

Novartis Investigative Site
Cauldfield, Victoria, 3162
Novartis Investigative Site
Nedlands, Western Australia, 6009
Novartis Investigative Site
St Leonards, New South Wales, 2065

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Belgium3 sites

Novartis Investigative Site
Brussels, 1200
Novartis Investigative Site
Edegem, 2650
Novartis Investigative Site
Ghent, 9000

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United Kingdom3 sites

Novartis Investigative Site
London, NW1 2BU
Novartis Investigative Site
Newcastle upon Tyne, NE4 5PL
Novartis Investigative Site
Salford, Manchester, M6 8HD

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Netherlands2 sites

Novartis Investigative Site
Amsterdam
Novartis Investigative Site
Leiden, 2333 ZA

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Denmark1 site

Novartis Investigative Site
Copenhagen, 2100

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France1 site

Novartis Investigative Site
Paris, 75013

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Switzerland1 site

Novartis Investigative Site
Zurich, 8091

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About this study

Official title: A Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel Group, Dose-finding, Pivotal, Phase 2b/3 Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous BYM338 at 52 Weeks on Physical Function, Muscle Strength, and Mobility and Additional Long Term Safety up to 2 Years in Patients With Sporadic Inclusion Body Myositis

Conditions: Sporadic Inclusion Body Myositis
Being tested: BYM338/bimagrumab, Placebo
Keywords: sporadic inclusion body myositis,, myositis,, muscle wasting,, controlled clinical trial,, randomized,, body mass,, muscle function,, strength,, performance,, physical function
Medical subject headings: Myositis, Inclusion Body, Myositis, Muscular Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BYM338/bimagrumabDrug
BYM338, a 150 mg/mL concentrate for solution for i.v. infusion, was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.
PlaceboDrug
Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.

Study groups in NCT01925209
Study group	Role	What they receive
BYM338/bimagrumab 10 mg/kg	Experimental	Participants received study medication with BYM338 at 10 mg/kg from Day 1 to Week 52 and up to Week 104, administered by intravenous (i.v.) infusion every 4 weeks. Receives: Drug: BYM338/bimagrumab
BYM338/bimagrumab 3 mg/kg	Experimental	Participants received study medication with BYM338 at 3 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks. Receives: Drug: BYM338/bimagrumab
BYM338/bimagrumab 1 mg/kg	Experimental	Participants received study medication with BYM338 at 1 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks. Receives: Drug: BYM338/bimagrumab
Placebo	Placebo comparator	Participants received matching placebo to BYM338 from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.
Measured: Baseline, Week 52

Secondary outcomes (5)

Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52
LBM was measured via dual energy x-ray absorptiometry (DXA) and calculated as (LBM at Week 52/LBM at baseline)*100 . A positive change from baseline indicates improvement.
Measured: Baseline, Week 52
Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.
Measured: Baseline, Week 52
Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52
Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration.
Measured: Baseline, Week 52
Estimated Annual Number of Falls Per Patient Within Treatment Group
Participants documented any fall occurrences in a paper diary during the study.
Measured: Week 52
Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52
The SPPB evaluated lower extremities function by testing gait speed, ability to keep standing balance and time to rise from a chair five times. The sub-score for each test ranged from 0 to 4. The summary score, which was a summation of scores from the 3 tests, ranged from 0 to 12. An increase in score indicates improvement in physical performance. A negative change from baseline indicates deterioration.
Measured: Baseline, Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

26 Sep 20136 Jan 2016

First posted19 Aug 2013 (estimated)
Study start26 Sep 2013
Primary completion6 Jan 2016
Study completion6 Jan 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Sporadic Inclusion Body MyositisStudies of the same condition, open and under way.
More trials run by Novartis 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 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 251 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01925209
Sponsor's protocol ID: CBYM338B2203
Acronym: RESILIENT
Status last verified: Aug 2017
First submitted: 15 Aug 2013
Last updated: 11 Aug 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Hanna MG, Badrising UA, Benveniste O, Lloyd TE, Needham M, Chinoy H, Aoki M, Machado PM, Liang C, Reardon KA, de Visser M, Ascherman DP, Barohn RJ, Dimachkie MM, Miller JAL, Kissel JT, Oskarsson B, Joyce NC, Van den Bergh P, Baets J, De Bleecker JL, Karam C, David WS, Mirabella M, Nations SP, Jung HH, Pegoraro E, Maggi L, Rodolico C, Filosto M, Shaibani AI, Sivakumar K, Goyal NA, Mori-Yoshimura M, Yamashita S, Suzuki N, Katsuno M, Murata K, Nodera H, Nishino I, Romano CD, Williams VSL, Vissing J, Auberson LZ, Wu M, de Vera A, Papanicolaou DA, Amato AA; RESILIENT Study Group. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial. Lancet Neurol. 2019 Sep;18(9):834-844. doi: 10.1016/S1474-4422(19)30200-5.
PubMed 31397289

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Sporadic Inclusion Body Myositis trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel Group, Dose-finding, Pivotal, Phase 2b/3 Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous BYM338 at 52 Weeks on Physical Function, Muscle Strength, and Mobility and Additional Long Term Safety up to 2 Years in Patients With Sporadic Inclusion Body Myositis
ClinicalTrials.gov ID: NCT01925209
Acronym / sponsor ID: RESILIENT / CBYM338B2203
Conditions: Sporadic Inclusion Body Myositis
Keywords: sporadic inclusion body myositis,, myositis,, muscle wasting,, controlled clinical trial,, randomized,, body mass,, muscle function,, strength,
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Completed
Enrolment: 251 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 26 Sep 2013 (actual)
Primary completion: 6 Jan 2016 (actual)
Study completion: 6 Jan 2016 (actual)
First posted: 19 Aug 2013 (estimated)
Results: Posted 12 May 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 4

Arm	Type	Receives
BYM338/bimagrumab 10 mg/kg	Experimental	Drug: BYM338/bimagrumabParticipants received study medication with BYM338 at 10 mg/kg from Day 1 to Week 52 and up to Week 104, administered by intravenous (i.v.) infusion every 4 weeks.
BYM338/bimagrumab 3 mg/kg	Experimental	Drug: BYM338/bimagrumabParticipants received study medication with BYM338 at 3 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
BYM338/bimagrumab 1 mg/kg	Experimental	Drug: BYM338/bimagrumabParticipants received study medication with BYM338 at 1 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
Placebo	Placebo comparator	Drug: PlaceboParticipants received matching placebo to BYM338 from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.

Who may qualify

Ages: 36 to 85 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (2 listed)

Diagnosed with sporadic inclusion body myositis;
Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);

Key exclusion criteria (5 listed)

Must not have other conditions that significantly limit ability to move around;
Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months;
Must meet cardiovascular requirements;
Must not be pregnant or nursing;
Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc.);

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
BYM338/bimagrumabBYM338, a 150 mg/mL concentrate for solution for i.v. infusion, was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.	Drug	BYM338/bimagrumab 1 mg/kg, BYM338/bimagrumab 10 mg/kg, BYM338/bimagrumab 3 mg/kg
PlaceboMatching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.	Drug	Placebo

Primary outcome

Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
Time frame: Baseline, Week 52
The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.

5 secondary outcomes

Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52
Time frame: Baseline, Week 52
Estimated Annual Number of Falls Per Patient Within Treatment Group
Time frame: Week 52
Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52
Time frame: Baseline, Week 52

Results and safety

Results posted to the registry on 12 May 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BYM338/Bimagrumab 10 mg/kg	63	54
BYM338/Bimagrumab 3 mg/kg	63	55
BYM338/Bimagrumab 1 mg/kg	63	56
Placebo	62	57

Primary outcome: Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52

Time frame: Baseline, Week 52. Least squares mean (Standard Error). Unit: meters

Measure	BYM338/Bimagrumab 10 mg/kg n = 61	BYM338/Bimagrumab 3 mg/kg n = 63	BYM338/Bimagrumab 1 mg/kg n = 63	Placebo n = 62
Value	8.63 (10.934)	9.63 (10.770)	-10.27 (10.718)	-8.96 (10.765)

BYM338/Bimagrumab 10 mg/kg vs Placebo: Mean Difference (Net): 17.59 (99% CI -19.63 to 54.80), p = 0.2210, mixed model repeated measures
BYM338/Bimagrumab 3 mg/kg vs Placebo: Mean Difference (Net): 18.59 (99% CI -18.21 to 55.40), p = 0.1909, mixed model repeated measure
BYM338/Bimagrumab 1 mg/kg vs Placebo: Mean Difference (Net): -1.31 (99% CI -37.97 to 35.36), p = 0.9263, mixed models repeated measures

Population analysed: The Full Analysis Set (FAS), which included all randomized participants who had received at least one dose of study drug and had at least one post-baseline efficacy assessment, was considered for the analysis. Only those participants from the FAS who had both baseline and week 52 6MWD measurements were analyzed.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: up to 2 years. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BYM338/Bimagrumab 10 mg/kg	21 of 63 (33.3%)	63 of 63 (100%)	Not reported
BYM338/Bimagrumab 3 mg/kg	11 of 63 (17.5%)	63 of 63 (100%)	Not reported
BYM338/Bimagrumab 1 mg/kg	18 of 63 (28.6%)	63 of 63 (100%)	Not reported
Placebo	20 of 62 (32.3%)	61 of 62 (98.4%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 31397289Hanna MG, Badrising UA, Benveniste O, Lloyd TE, Needham M, Chinoy H, Aoki M, Machado PM, Liang C, Reardon KA, de Visser M, Ascherman DP, Barohn RJ, Dimachkie MM, Miller JAL, Kissel JT, Oskarsson B, Joyce NC, Van den Bergh P, Baets J, De Bleecker JL, Karam C, David WS, Mirabella M, Nations SP, Jung HH, Pegoraro E, Maggi L, Rodolico C, Filosto M, Shaibani AI, Sivakumar K, Goyal NA, Mori-Yoshimura M, Yamashita S, Suzuki N, Katsuno M, Murata K, Nodera H, Nishino I, Romano CD, Williams VSL, Vissing J, Auberson LZ, Wu M, de Vera A, Papanicolaou DA, Amato AA; RESILIENT Study Group. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial. Lancet Neurol. 2019 Sep;18(9):834-844. doi: 10.1016/S1474-4422(19)30200-5.

Related trials for Sporadic Inclusion Body Myositis

Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
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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.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

38 sites in 10 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing BYM338/bimagrumab in people with Sporadic Inclusion Body Myositis, run by Novartis Pharmaceuticals at 38 sites in 10 countries.

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 2 inclusion and 5 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 11 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 38 sites in United States, Japan, Italy, Australia, Belgium and 5 more countries. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 May 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 6 Minute Walking Distance (6MWD) Test at Week 52 (time frame: Baseline, Week 52). 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 NCT01925209, 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 11 Aug 2017.