Chronic Obstructive Pulmonary Disease (COPD) With CachexiaPhase 2NCT01669174
BYM338 in Chronic Obstructive Pulmonary Disease (COPD) Patients With Cachexia
A Phase 2 study testing BYM338 in people with Chronic Obstructive Pulmonary Disease (COPD) With Cachexia, run by Novartis Pharmaceuticals at 9 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 40 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Chronic Obstructive Pulmonary Disease (COPD) With Cachexia
- What is being tested?
- BYM338
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 67 participants
- Where
- 9 sitesin 3 countries
- Study dates
- Sep 2012to Dec 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 40 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
United States5 sites
Novartis Investigative Site
Missoula, Montana, 59808
Novartis Investigative Site
Normal, Illinois, 61761
Novartis Investigative Site
Savannah, Georgia, 31419
Novartis Investigative Site
Spartanburg, South Carolina, 29303
Novartis Investigative Site
Torrance, California, 90502
United Kingdom3 sites
Novartis Investigative Site
Leicester, LE1 5WW
Novartis Investigative Site
London, SW 6NP
Novartis Investigative Site
Machester, M23 9QZ
About this study
Official title: A Randomized, Double Blind, Placebo Controlled, Multi-centre Study to Asses the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of BYM338 in Chronic Obstructive Pulmonary Disease Patients With Cachexia
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.
- Being tested
- Placebo, BYM338
- Keywords
- Chronic Obstructive Pulmonary Disease, COPD; cachexia; muscle wasting
- Medical subject headings
- Pulmonary Disease, Chronic Obstructive, Cachexia, Muscular Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
BYM338Drug
BYM338
| Study group | Role | What they receive |
|---|---|---|
| BYM338 | Experimental | Not described Receives: Drug: BYM338 |
| Placebo | Placebo comparator | Not described Receives: 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 4, 8, 16, and 24
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 4,8,16 and 24 was considered responders.
Measured: Baseline, Weeks 4, 8, 16, 24
Secondary outcomes (4)
Change in 6 Minute Walk Distance Compared to Placebo
Practical simple test that requires a 100-ft hallway but no exercise quipment or advanced training for technicians. Walking is an activity performed daily by all but the most severely impaired patients. This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes (the 6MWD)
Measured: Baseline, Weeks 4, 8, 16, 24
Maximum Observed Serum Concentration (Cmax)
The observed maximum plasma concentration following drug administration
Measured: 0 hour, 2 hour, Day 8, 15, 29, 57, 71, 85, 99, 113, 127, 168 post dose
Time to Reach the Maximum Concentration After Drug Administration (Tmax)
The time to reach the maximum concentration after drug administration
Measured: 24 weeks
AUC0-56 and AUClast
AUC0-56, the area under the serum concentration-time curve from the time zero to the end of the dosing interval, day 56. AUC0-56 was analyzed for dose 1 and 2. AUClast is from time zero to the last quantifiable concentration. AUClast was analyzed for dose 2 only.
Measured: 0 hour, 2 hour, Day 8, 15, 29, 57, 71, 85, 99, 113, 127, 168 post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2012 (estimated)
- Study startSep 2012
- Primary completionDec 2014
- Study completionDec 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 Chronic Obstructive Pulmonary Disease (COPD) With 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
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01669174
- Sponsor's protocol ID
- CBYM338X2204
- Other IDs
- 2011-000461-12
- Status last verified
- Apr 2016
- First submitted
- 1 Mar 2012
- Last updated
- 3 May 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Polkey MI, Praestgaard J, Berwick A, Franssen FME, Singh D, Steiner MC, Casaburi R, Tillmann HC, Lach-Trifilieff E, Roubenoff R, Rooks DS. Activin Type II Receptor Blockade for Treatment of Muscle Depletion in Chronic Obstructive Pulmonary Disease. A Randomized Trial. Am J Respir Crit Care Med. 2019 Feb 1;199(3):313-320. doi: 10.1164/rccm.201802-0286OC.
PubMed 30095981
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Placebo Controlled, Multi-centre Study to Asses the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of BYM338 in Chronic Obstructive Pulmonary Disease Patients With Cachexia
- ClinicalTrials.gov ID
- NCT01669174
- Acronym / sponsor ID
- CBYM338X2204
- Keywords
- Chronic Obstructive Pulmonary Disease, COPD; cachexia; muscle wasting
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2012
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 20 Aug 2012 (estimated)
- Results
- Posted 3 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BYM338 | Experimental | Drug: BYM338 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
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 |
|---|---|---|
| Placebo | Drug | Placebo |
| BYM338BYM338 | Drug | BYM338 |
Primary outcome
Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 4, 8, 16, and 24
Time frame: Baseline, Weeks 4, 8, 16, 24
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 4,8,16 and 24 was considered responders.
4 secondary outcomes
Change in 6 Minute Walk Distance Compared to Placebo
Time frame: Baseline, Weeks 4, 8, 16, 24
Maximum Observed Serum Concentration (Cmax)
Time frame: 0 hour, 2 hour, Day 8, 15, 29, 57, 71, 85, 99, 113, 127, 168 post dose
Time to Reach the Maximum Concentration After Drug Administration (Tmax)
Time frame: 24 weeks
AUC0-56 and AUClast
Time frame: 0 hour, 2 hour, Day 8, 15, 29, 57, 71, 85, 99, 113, 127, 168 post dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30095981Polkey MI, Praestgaard J, Berwick A, Franssen FME, Singh D, Steiner MC, Casaburi R, Tillmann HC, Lach-Trifilieff E, Roubenoff R, Rooks DS. Activin Type II Receptor Blockade for Treatment of Muscle Depletion in Chronic Obstructive Pulmonary Disease. A Randomized Trial. Am J Respir Crit Care Med. 2019 Feb 1;199(3):313-320. doi: 10.1164/rccm.201802-0286OC.
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
9 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BYM338 in people with Chronic Obstructive Pulmonary Disease (COPD) With Cachexia, run by Novartis Pharmaceuticals at 9 sites in 3 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 6 inclusion and 12 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 3 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, United Kingdom, Netherlands. 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 3 May 2016). 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 4, 8, 16, and 24 (time frame: Baseline, Weeks 4, 8, 16, 24). 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 NCT01669174, 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 3 May 2016.