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Phase 2 N=67 Randomized Quadruple-blind Supportive Care

BYM338 in Chronic Obstructive Pulmonary Disease (COPD) Patients With Cachexia

Chronic Obstructive Pulmonary Disease (COPD) With Cachexia

Enrolled (actual)
67
Serious AEs
10.5%
Results posted
May 2016
Primary outcome: Primary: Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 4, 8, 16, and 24 — 5.87; 0.02; 7.01; -0.65 Percentage Change of TMV — p=<0.001

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Placebo (Drug); BYM338 (Drug)
Age
Adult, Older Adult · 40+ yrs
Sex
All
Sponsor
Novartis Pharmaceuticals
Primary completion
Dec 2014

Outcome Measures

OutcomeResultp-value
PRIMARY
Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 4, 8, 16, and 24
5.87; 0.02; 7.01; -0.65; 7.84; -0.88 <0.001 sig
SECONDARY
Change in 6 Minute Walk Distance Compared to Placebo
364.6; 388.5; 373.9; 387.4; 383.0; 385.6
SECONDARY
Maximum Observed Serum Concentration (Cmax)
614; 580
SECONDARY
Time to Reach the Maximum Concentration After Drug Administration (Tmax)
2.22; 2.23
SECONDARY
AUC0-56 and AUClast
4540; 5790; 7480

Summary

This study will assess the pharmacodynamics, pharmacokinetics, safety and tolerability of BYM338 in patients with COPD and cachexia. The primary outcome will be a change in thigh muscle volume compared to placebo. The study will last for approximately 24 weeks.

Eligibility Criteria

Inclusion criteria

  • Written informed consent must be obtained before any assessment is performed.
  • Males and females ages 40 to 80 years
  • Smoking history of at least 10 pack-years
  • Diagnosis of COPD according to GOLD guidelines (GOLD, 2010), with a post-bronchodilator FEV¬1 < 80% predicted and FEV1/FVC ratio < 0.70
  • BMI <20 kg/m2 or skeletal muscle mass index by DXA < 7.25 kg/m2 for men or <5.45 kg/m2 for women.
  • In general stable health, including managed COPD, by past medical history, physical examination, vital signs at baseline as determined by the investigator.

Exclusion criteria

  • Patients with MRC dyspnoea grade 5 (i.e. patients too breathless to leave the house or breathless when dressing)
  • Plans for lung transplantation or lung reduction surgery within four months of enrollment
  • Patients participating in a formal pulmonary rehabilitation program within 3 months of dosing
  • History of malignancy of any organ system (other than excised non-melanomatous carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
  • Diseases other than cancer known to cause cachexia or muscle atrophy, including but not limited to congestive heart failure of any stage, chronic kidney disease (estimated GFR < 30 mL/min using the MDRD equation), rheumatoid arthritis, primary myopathy, stroke, HIV infection, tuberculosis or other chronic infection, uncontrolled diabetes mellitus, etc.
  • Inflammatory bowel disease, celiac disease, short bowel syndrome, pancreatic insufficiency
  • Use of any prescription drugs known to affect muscle mass, including androgen supplements, anti-androgens (such as LHRH agonists), anti-estrogens (tamoxifen, etc.) recombinant human growth hormone (rhGH), insulin, oral beta agonists, megestrol acetate, dronabinol, metformin, etc.
  • Hemoglobin concentration below 11.0 g/dL at screening.
  • Liver disease or liver injury.
  • Use of other investigational drugs at the time of enrollment, or within 30 days and for any other limitation of participation in an investigational trial based on local regulations.
  • Women of child-bearing potential.

Other protocol-defined inclusion/exclusion criteria may apply

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01669174). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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