Study of efficacy and safety of BYM338 in sporadic inclusion body myositis patients

• Register: EUCTR
• Last refreshed on: 30 June 2019
• Main ID: EUCTR2013-000705-23-DK
• Date of registration: 10/03/2014
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Study of efficacy and safety of BYM338 in sporadic inclusion body myositis patients
• Scientific title: A randomized, double-blind, placebo-controlled, multicenter, parallel group, dose-finding, pivotal, phase IIb/III 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 - RESILIENT
• Date of first enrolment: 21/03/2014
• Target sample size: 240
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-000705-23
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 4
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Belgium, Denmark, France, Germany, Italy, Japan, Netherlands, Poland, Switzerland, United Kingdom, United States

Contacts

• Name: Medicinsk Information
• Affiliation: Novartis Healthcare A/S

• Name: Medicinsk Information
• Affiliation: Novartis Healthcare A/S

Key inclusion & exclusion criteria

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

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 170

Exclusion criteria:
•No 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);

Other protocol-defined inclusion/exclusion criteria may apply



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Sporadic Inclusion Body Myositis
MedDRA version: 18.1 Level: PT Classification code 10066407 Term: Inclusion body myositis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Intervention(s)

Product Code: BYM338
Pharmaceutical Form: Solution for injection/infusion
CAS Number: 1356922-05-8
Current Sponsor code: BYM338
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Solution for injection/infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Secondary Objective: •Change from Baseline in lean body mass (LBM) at Week 52
•Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52
•Change from Baseline in Patient-Reported Physical Function at Week 52
•Rate of Fall Events
•Change from Baseline in Short Physical Performance Battery score at Week 52
•Dose-response relationship in the change from Baseline in 6MWD meters to Week 52

Timepoint(s) of evaluation of this end point: 52 weeks

Main Objective: To demonstrate that at least one dose regimen of BYM338 will increase the 6 minute walking distance test relative to placebo at week 52.

Primary end point(s): Change from Baseline in 6 Minute Walking Distance Test (6MWD) meters to Week 52

Secondary Outcome(s)

Secondary end point(s): •Change from Baseline in lean body mass (LBM) at Week 52
•Change from Baseline in quadriceps Quantitative Muscle Testing (QMT) at Week 52
•Change from Baseline in Patient-Reported Physical Function at Week 52
•Rate of Fall Events
•Change from Baseline in Short Physical Performance Battery score at Week 52
•Dose-response relationship in the change from Baseline in 6MWD meters to Week 52

Timepoint(s) of evaluation of this end point: Week 52 for LBM, QMT, patient-reporter physical function, short physical performance battery and dose-response relationship for 6MWD and measured during entire study for falls , safety and tolerability

Secondary ID(s)

NCT01925209

CBYM338B2203

2013-000705-23-IT

Source(s) of Monetary Support

Novartis Pharma Services AG

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 21/01/2017
• Date Completed: 06/01/2016
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-000705-23/results