CachexiaPhase 2NCT01433263

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

A Phase 2 study testing BYM338 active drug in people with Cachexia, run by Novartis Pharmaceuticals at 10 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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 Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Cachexia
What is being tested?: BYM338 active drug
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 57 participants
Where: 10 sitesin 5 countries
Study dates: Aug 2011to Apr 2014
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients must sign an informed consent before assessment
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.
Patients with stage IV NSCLC will be receiving or completed or discontinued standard chemotherapy or be chemotherapy-naive by choice.
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.
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.
Body mass index (BMI) ≤ 30 kg/m2.
Life expectancy of at least 4 months.
Able to communicate well and comply with the requirements of the study, including by phone and written logs.

Who cannot join

Patients who have received investigational anti-neoplastic therapy within 3 weeks of screening
Evidence of inadequate organ or brain function, as defined by lab tests and imaging
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
Pregnant or lactating women.
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
Patients unwilling or unable to follow instructions.
Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

10 sites in 5 countries.

Country

City, postcode or hospital

10 sites in 5 countries

United States6 sites

Novartis Investigative Site
Boston, Massachusetts, 02114
Novartis Investigative Site
Canton, Ohio, 44718
Novartis Investigative Site
Chicago, Illinois, 60611-3308
Novartis Investigative Site
San Antonio, Texas, 78217
Novartis Investigative Site
Tampa, Florida, 33647
Novartis Investigative Site
Tucson, Arizona, 85715

Other trials in United States

Lithuania1 site

Novartis Investigative Site
Vilnius, LT 08661

Other trials in Lithuania

Romania1 site

Novartis Investigative Site
Bucharest, Sector 5

Other trials in Romania

Switzerland1 site

Novartis Investigative Site
Sankt Gallen, 9007

Other trials in Switzerland

United Kingdom1 site

Novartis Investigative Site
Edinburgh, EH10 5HF

Other trials in United Kingdom

About this study

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

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

Conditions: Cachexia
Being tested: BYM338 active drug, Placebo
Keywords: Wasting syndrome, emaciation, lung cancer, adenocarcinoma, pancreatic cancer
Medical subject headings: Cachexia, Wasting Syndrome, Emaciation, Lung Neoplasms, Adenocarcinoma, Pancreatic Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BYM338 active drugDrug
PlaceboDrug

Study groups in NCT01433263
Study group	Role	What they receive
30mg/kg BYM338	Experimental	Not described Receives: Drug: BYM338 active drug
Placebo / late 30mg/kg BYM338	Placebo comparator	Not described Receives: Drug: BYM338 active drug; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline, week 8

Secondary outcomes (9)

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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, Week 8
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.
Measured: 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.
Measured: 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
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.
Measured: 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
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.
Measured: 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
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.
Measured: Baseline, Week 4 and Week 7

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2011Apr 2014

First posted13 Sep 2011 (estimated)
Study startAug 2011
Primary completionApr 2014
Study completionApr 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for CachexiaStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 57 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01433263
Sponsor's protocol ID: CBYM338X2202
Status last verified: Feb 2016
First submitted: 24 Aug 2011
Last updated: 2 Mar 2016
Results posted: Yes, on ClinicalTrials.gov

Publications

Epstein SA, Doles JD, Dasgupta A. KLF10: a point of convergence in cancer cachexia. Curr Opin Support Palliat Care. 2024 Sep 1;18(3):120-125. doi: 10.1097/SPC.0000000000000711. Epub 2024 Jul 15.
PubMed 39007915

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cachexia trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT01433263
Acronym / sponsor ID: CBYM338X2202
Conditions: Cachexia
Keywords: Wasting syndrome, emaciation, lung cancer, adenocarcinoma, pancreatic cancer
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 57 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Aug 2011
Primary completion: Apr 2014 (actual)
Study completion: Apr 2014 (actual)
First posted: 13 Sep 2011 (estimated)
Results: Posted 13 May 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
30mg/kg BYM338	Experimental	Drug: BYM338 active drug
Placebo / late 30mg/kg BYM338	Placebo comparator	Drug: BYM338 active drug, Drug: Placebo

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

Patients must sign an informed consent before assessment
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.
Patients with stage IV NSCLC will be receiving or completed or discontinued standard chemotherapy or be chemotherapy-naive by choice.
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.
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.

Key exclusion criteria (7 listed)

Patients who have received investigational anti-neoplastic therapy within 3 weeks of screening
Evidence of inadequate organ or brain function, as defined by lab tests and imaging
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
Pregnant or lactating women.
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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BYM338 active drug	Drug	30mg/kg BYM338, Placebo / late 30mg/kg BYM338
Placebo	Drug	Placebo / late 30mg/kg BYM338

Primary outcome

Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 8
Time frame: Baseline, 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.

9 secondary outcomes

Percentage Change in Body Weight From Baseline at Week 7 and Week 9
Time frame: Baseline, Week 7 and Week 9
Maximum Observed Serum Concentration (Cmax)
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)
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
Time frame: Baseline, Week 8
Percentage Change From Baseline of Bone Mineral Density (BMD) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo at Week 8
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
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

Results and safety

Results posted to the registry on 13 May 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
30mg/kg BYM338	29	10
Placebo / Late 30mg/kg BYM338	28	16

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

Time frame: Baseline, week 8. Mean (Standard Deviation). Unit: Percentage Change of TMV

Measure	30mg/kg BYM338 n = 15	Placebo / Late 30mg/kg BYM338 n = 22
Value	2.0 (8.094)	0.65 (8.239)

Population analysed: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data. However, for a given time frame, analyzed participants had values at both baseline and the corresponding time frame, i.e. week 8

9 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Core - 30mg/kg BYM338	18 of 29 (62.1%)	19 of 29 (65.5%)	Not reported
Core - Placebo	4 of 28 (14.3%)	19 of 28 (67.9%)	Not reported
Follow-up - 30mg/kg BYM338	7 of 19 (36.8%)	12 of 19 (63.2%)	Not reported
Follow-up - Placebo	1 of 3 (33.3%)	2 of 3 (66.7%)	Not reported
Follow-up - 30mg/kg BYM338 Late	7 of 21 (33.3%)	13 of 21 (61.9%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 39007915Epstein SA, Doles JD, Dasgupta A. KLF10: a point of convergence in cancer cachexia. Curr Opin Support Palliat Care. 2024 Sep 1;18(3):120-125. doi: 10.1097/SPC.0000000000000711. Epub 2024 Jul 15.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

10 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BYM338 active drug in people with Cachexia, run by Novartis Pharmaceuticals at 10 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in United States, Lithuania, Romania, Switzerland, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 13 May 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 8 (time frame: Baseline, week 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01433263, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2016.