Clinical Study of BYM338 for the Treatment of Unintentional Weight Loss in Patients With Cancer of the Lung or the Pancreas

Novartis Pharmaceuticals57 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Cachexia

Interventions

BYM338 active drugDRUG

PlaceboDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (10)

Novartis Investigative Site

Tucson, Arizona, United States

Novartis Investigative Site

Tampa, Florida, United States

Novartis Investigative Site

Chicago, Illinois, United States

Outcomes

Primary Outcomes

Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 8

Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was no more than or equal to 2% at Week 8 was considered responders.

Time frame: Baseline, week 8

Secondary Outcomes

Percentage Change in Body Weight From Baseline at Week 7 and Week 9

Percentage Change in body weight from baseline in killograms (kg) at week 7 and week 9

Time frame: Baseline, Week 7 and Week 9

Maximum Observed Serum Concentration (Cmax)

Blood samples for pharmacokinetic (PK) evaluation were drawn on Day 1 30mg/kg BYM338 (Core)or week 8 Late 30mg/kg BYM338 (when placebo subjects were rolled over to active). PK parameters were calculated from plasma concentration-time data using non-compartmental methods.

Time frame: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Day 1 and Week 8

Time to Reach the Maximum Concentration After Drug Administration (Tmax)

Blood samples for pharmacokinetic (PK) evaluation were drawn on Day 1 30mg/kg BYM338 (Core)or week 8 Late 30mg/kg BYM338 (when placebo subjects were rolled over to active). Tmax was directly determined from the raw serum concentration-time data.

Time frame: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Day 1 and Week 8

Percentage Change From Baseline in Total Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo: at Week 8

total lean body mass (LBM) is measured by dual energy x-ray absorptiometry (DXA).Percent Change = \[(LBM at Visit - LBM at Baseline) / LBM at Baseline\] \* 100.

Time frame: Baseline, Week 8

Data from ClinicalTrials.gov

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Novartis Investigative Site

Boston, Massachusetts, United States

Novartis Investigative Site

Canton, Ohio, United States

Novartis Investigative Site

San Antonio, Texas, United States

Novartis Investigative Site

Vilnius, Lithuania

Novartis Investigative Site

Bucharest, Romania

Novartis Investigative Site

Sankt Gallen, Switzerland

Novartis Investigative Site

Edinburgh, United Kingdom

Percentage Change From Baseline of Bone Mineral Density (BMD) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo at Week 8

Bone Mineral Density (BMD)is measured by dual energy x-ray absorptiometry (DXA).Percent Change = \[(BMD at Visit - BMD at Baseline) / BMD at Baseline\] \* 100.

Time frame: Baseline, Week 8

Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Number of Steps Taken Compared to Placebo at Week 4 and 7

Each patient was required to wear the ActivPal™ for a span of 6 days at Week 4 and Week 7 for patient home activity recording. The ActivPal™ was given to patients in clinic to wear for 6 consecutive days. The ActivPAL™ records periods spent sitting, standing and walking, sit-to-stand transitions, step count and rate of stepping (cadence) over a maximum period of 10 days with a fully charged new battery.

Time frame: Baseline, Week 4 and Week 7

Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Sedentary Taken Compared to Placebo at Week 4 and 7

Time frame: Baseline, Week 4 and Week 7

Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Standing Compared to Placebo at Week 4 and 7

Time frame: Baseline, Week 4 and Week 7

Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Stepping Compared to Placebo at Week 4 and 7

Time frame: Baseline, Week 4 and Week 7