Clinical trial NCT01433263: Clinical Study of BYM338 for the Treatment of Unintentional Weight Loss in Patients With Cancer of the Lung or the Pancreas
Official title: A Randomized, Double-blind, Placebo-controlled Multi-center Study of BYM338 for Treatment of Cachexia in Patients With Stage IV Non-small Cell Lung Cancer or Stage III/IV Adenocarcinoma of the Pancreas
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cachexia
Keywords: Wasting syndrome
emaciation
lung cancer
adenocarcinoma
pancreatic cancer
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-09-13
Enrollment: 57
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A safety \& efficacy clinical study of the investigational medicinal product BYM338 for the treatment of unintentional weight loss in patients with cancer of the lung or the pancreas


Eligibility criteria: Key Inclusion criteria:

1. Patients must sign an informed consent before assessment
2. Patients with pathologically and/or clinically confirmed diagnosis of stage IV non-small (squamous or non-squamous) cell lung cancer (NSCLC) or stage III/IV adenocarcinoma of the pancreas.
3. Patients with stage IV NSCLC will be receiving or completed or discontinued standard chemotherapy or be chemotherapy-naive by choice.
4. Patients with stage III/IV pancreatic adenocarcinoma will be receiving standard chemotherapy or no chemotherapy. If patients are receiving chemotherapy, a change in chemotherapy is not expected.
5. Greater than or equal to 5% unintentional weight loss over the previous 3-6 months, not explained by simple starvation. Simple starvation is considered to be excluded when weight loss is not ameliorated by standard nutritional counseling and oral supplementation over a 2 week period.
6. Body mass index (BMI) ≤ 30 kg/m2.
7. Life expectancy of at least 4 months.
8. Able to communicate well and comply with the requirements of the study, including by phone and written logs.

Key Exclusion criteria:

1. Patients who have received investigational anti-neoplastic therapy within 3 weeks of screening
2. Evidence of inadequate organ or brain function, as defined by lab tests and imaging
3. Patients with severe and/or uncontrolled medical conditions that could interfere with the study (e.g. heart conditions, high blood pressure, diabetes, infection) uncontrolled pain or any other non-stable illness
4. Pregnant or lactating women.
5. Women capable of becoming pregnant must use highly effective contraception during the study and for 8 weeks after stopping treatment. All female patients must have negative pregnancy test results throughout the study
6. Patients unwilling or unable to follow instructions.

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 8
timeFrame: Baseline, week 8
Secondary outcomes:
measure: Percentage Change in Body Weight From Baseline at Week 7 and Week 9
timeFrame: Baseline, Week 7 and Week 9
measure: Maximum Observed Serum Concentration (Cmax)
timeFrame: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Day 1 and Week 8
measure: Time to Reach the Maximum Concentration After Drug Administration (Tmax)
timeFrame: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Day 1 and Week 8
measure: Percentage Change From Baseline in Total Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo: at Week 8
timeFrame: Baseline, Week 8
measure: Percentage Change From Baseline of Bone Mineral Density (BMD) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo at Week 8
timeFrame: Baseline, Week 8
measure: Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Number of Steps Taken Compared to Placebo at Week 4 and 7
timeFrame: Baseline, Week 4 and Week 7
measure: Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Sedentary Taken Compared to Placebo at Week 4 and 7
timeFrame: Baseline, Week 4 and Week 7
measure: Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Standing Compared to Placebo at Week 4 and 7
timeFrame: Baseline, Week 4 and Week 7
measure: Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Stepping Compared to Placebo at Week 4 and 7
timeFrame: Baseline, Week 4 and Week 7
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Novartis Investigative Site
state: Arizona
city: Tampa
country: United States
facility: Novartis Investigative Site
state: Florida
city: Chicago
country: United States
facility: Novartis Investigative Site
state: Illinois
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Canton
country: United States
facility: Novartis Investigative Site
state: Ohio
city: San Antonio
country: United States
facility: Novartis Investigative Site
state: Texas
city: Vilnius
country: Lithuania
facility: Novartis Investigative Site
city: Bucharest
country: Romania
facility: Novartis Investigative Site
city: St. Gallen
country: Switzerland
facility: Novartis Investigative Site
city: Edinburgh
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: