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

NCT01925209 | Completed Phase 2 Results DMC

• 1 other identifier: CBYM338B2203
• Study Type: interventional
• Target: 251
• Locations: 10 countries
• Sites: 38

Brief 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).

Conditions

Sporadic Inclusion Body Myositis

Keywords

sporadic inclusion body myositis,; myositis,; muscle wasting,; controlled clinical trial,; randomized,; body mass,; muscle function,; strength,; performance,; physical function

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, Week 52

Secondary Outcomes (5)

• Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52

  Baseline, Week 52

• Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52

  Baseline, Week 52

• Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52

  Baseline, Week 52

• Estimated Annual Number of Falls Per Patient Within Treatment Group

  Week 52

• Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52

  Baseline, Week 52

Study Arms (4)

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.

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.

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.

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.

Drug: Placebo

Interventions

BYM338/bimagrumab DRUG

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.

BYM338/bimagrumab 1 mg/kg; BYM338/bimagrumab 10 mg/kg; BYM338/bimagrumab 3 mg/kg

Placebo DRUG

Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.

Placebo

Eligibility Criteria

• Age: 36 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);

You may not qualify if:

• 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.);

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (38)

Novartis Investigative Site

Phoenix, Arizona, 85028, United States

Location

Novartis Investigative Site

Orange, California, 92868, United States

Location

Novartis Investigative Site

Sacramento, California, 95817, United States

Location

Novartis Investigative Site

Miami, Florida, 33101, United States

Location

Novartis Investigative Site

Kansas City, Kansas, 66160, United States

Location

Novartis Investigative Site

Baltimore, Maryland, 21287, United States

Location

Novartis Investigative Site

Boston, Massachusetts, 02114, United States

Location

Novartis Investigative Site

Boston, Massachusetts, 02115, United States

Location

Novartis Investigative Site

Columbus, Ohio, 43221, United States

Location

Novartis Investigative Site

Portland, Oregon, 97239, United States

Location

Novartis Investigative Site

Dallas, Texas, 75235, United States

Location

Novartis Investigative Site

Houston, Texas, 77030, United States

Location

Novartis Investigative Site

St Leonards, New South Wales, 2065, Australia

Location

Novartis Investigative Site

Cauldfield, Victoria, 3162, Australia

Location

Novartis Investigative Site

Nedlands, Western Australia, 6009, Australia

Location

Novartis Investigative Site

Brussels, 1200, Belgium

Location

Novartis Investigative Site

Edegem, 2650, Belgium

Location

Novartis Investigative Site

Ghent, 9000, Belgium

Location

Novartis Investigative Site

Copenhagen, 2100, Denmark

Location

Novartis Investigative Site

Paris, 75013, France

Location

Novartis Investigative Site

Brescia, BS, 25123, Italy

Location

Novartis Investigative Site

Rome, Lazio, 00168, Italy

Location

Novartis Investigative Site

Messina, ME, 98125, Italy

Location

Novartis Investigative Site

Milan, MI, 20133, Italy

Location

Novartis Investigative Site

Padua, PD, 35128, Italy

Location

Novartis Investigative Site

Nagoya, Aichi-ken, 466-8560, Japan

Location

Novartis Investigative Site

Kumamoto, Kumamoto, 860-8556, Japan

Location

Novartis Investigative Site

Sendai, Miyagi, 980-8574, Japan

Location

Novartis Investigative Site

Osaka, Osaka, 534-0021, Japan

Location

Novartis Investigative Site

Tokushima, Tokushima, 770-8503, Japan

Location

Novartis Investigative Site

Kodaira, Tokyo, 187-8551, Japan

Location

Novartis Investigative Site

Wakayama, Wakayama, 641-8510, Japan

Location

Novartis Investigative Site

Amsterdam, Netherlands

Location

Novartis Investigative Site

Leiden, 2333 ZA, Netherlands

Location

Novartis Investigative Site

Zurich, 8091, Switzerland

Location

Novartis Investigative Site

Salford, Manchester, M6 8HD, United Kingdom

Location

Novartis Investigative Site

London, NW1 2BU, United Kingdom

Location

Novartis Investigative Site

Newcastle upon Tyne, NE4 5PL, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 31397289 DERIVED

MeSH Terms

Conditions

Myositis, Inclusion Body; Myositis; Muscular Atrophy

Interventions

bimagrumab

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Atrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 15, 2013
• First Posted: August 19, 2013
• Study Start: September 26, 2013
• Primary Completion: January 6, 2016
• Study Completion: January 6, 2016
• Last Updated: August 11, 2017
• Results First Posted: May 12, 2017

Record last verified: 2017-08

Locations

DK (1); IT (5); JP (7); GB (3); AU (3); BE (3); US (12); FR (1); NL (2); CH (1)