Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis

NCT02250443 | Completed Phase 2 Results

• 1 other identifier: CBYM338X2205E1
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

This study is an open-label, long-term study for those patients who participated in the prior proof-of-concept protocol, in which the preliminary efficacy for BYM338 in patients with sIBM was demonstrated after a single 30 mg/kg i.v. dose of BYM338. This study is designed to confirm the efficacy, safety and tolerability of BYM338 in sIBM with long-term dosing. However due to lack of efficacy in patients with sIBM, the study was terminated early.

Conditions

Sporadic Inclusion Body Myositis (sIBM)

Keywords

Inclusion Body Myositis (IBM)

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events as a Measure of Safety and Tolerability

  Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity

  Up to 29 month

Secondary Outcomes (9)

• Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)

  Baseline, Day 1, 57, 113, 169, 365, 533, and day 729

• Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing

  Day 29, 85, 169, 253, 337, 421, 505, 589, 673, 757, 1177

• Changes From Baseline in Physical Function Reported by Patients

  Baseline, Week 104

• Changes From Baseline in Muscle Strength.

  Baseline, Day 1, 113, 169, 365, 533, 729

• Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)

  Baseline,Day 1, 113, 169, 365, 533, 729

Study Arms (1)

BYM338

EXPERIMENTAL

BYM338 Group

Drug: BYM338 (Bimagrumab)

Interventions

BYM338 (Bimagrumab) DRUG

BYM338

Eligibility Criteria

• Age: 40 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent must be obtained before any assessment is performed.
• Patients who participated in the CBYM338X2205 study. A patient is defined as participating in the study if they were enrolled and received study medication.
• Able to communicate well with the investigator, to understand and comply with the requirements of the study.

You may not qualify if:

• Patients for whom the treating physician considers it inappropriate for continuation into the study, including consideration of physical and laboratory assessment of the patient at screening.
• Patients who were non-compliant or demonstrated a serious protocol deviation in the previous study.
• Use of other investigational drugs at the time of enrollment, within 30 days or 5 half-lives of enrollment, or until the expected PD effect has returned to baseline, whichever is longer; or longer if required by local regulations.
• History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes
• Swallowing difficulty or other reason that precludes adequate intake of energy and protein, defined as at least 20 kcal/kg/day and 0.6 g protein/kg/day as determined by the investigators assessment.
• On the Columbia-Suicide Severity Rating Scale, a score of 4 or 5 on the Suicidal Ideation item or any "yes" on the Suicidal Behavior item that is related to suicidal behavior occurring during the last 2 years.
• Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, must use highly effective contraception during the study and for 5 half-lives (14 weeks) after stopping treatment. Highly effective contraception is defined as either:
• Total abstinence: When this is in line with the preferred and usual lifestyle of the subject. \[Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception\].
• Sterilization: have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
• Male partner sterilization (with the appropriate post-vasectomy documentation of the absence of sperm in the ejaculate). \[For female study subjects, the vasectomized male partner should be the sole partner for that patient\].
• Use of a combination of any two of the following (a+b or a+c or b+c):
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• Use of oral, injected or implanted hormonal methods of contraception.
• Placement of an intrauterine device (IUD) or intrauterine system (IUS).

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (2)

Novartis Investigative Site

Phoenix, Arizona, 85013, United States

Location

Novartis Investigative Site

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• Sivakumar K, Cochrane TI, Sloth B, Ashar H, Laurent D, Tanko LB, Amato AA. Long-term safety and tolerability of bimagrumab (BYM338) in sporadic inclusion body myositis. Neurology. 2020 Oct 6;95(14):e1971-e1978. doi: 10.1212/WNL.0000000000010417. Epub 2020 Jul 20.

  PMID: 32690797 DERIVED

Related Links

• A Plain Language Trial Summary is available on novartisclinicaltrials.com

MeSH Terms

Conditions

Myositis, Inclusion Body

Interventions

bimagrumab

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceutical

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2014
• First Posted: September 26, 2014
• Study Start: March 11, 2014
• Primary Completion: August 23, 2016
• Study Completion: August 23, 2016
• Last Updated: December 30, 2020
• Results First Posted: April 10, 2018

Record last verified: 2019-03

Locations

US (2)