Skeletal MusclePhase 2NCT01601600
A Multi-center Study to Assess the Effects of BYM338 on Skeletal Muscle in Sarcopenic Adults
A Phase 2 study testing BYM338 in people with Skeletal Muscle, run by Novartis Pharmaceuticals at 5 sites in United States.
Trial summary
The purpose of this Proof of Concept study is to determine the effects of BYM338 on skeletal muscle volume, mass, and strength and patient function (gait speed) in non-demented elderly adults with sarcopenia and mobility limitations. Read more about this study
- Who is it for?
- Ages 65 years and older
- Conditions the study is for
- Skeletal Muscle
- What is being tested?
- BYM338
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 5 sitesUnited States
- Study dates
- Jan 2012to Dec 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 65 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Men or women aged 65 or older with difficulty standing up from a chair or walking for longer than 10 minutes on a flat surface or climbing a flight of stairs.
- Written informed consent must be obtained before any laboratory or physical assessment is performed to establish eligibility. Answering initial questions for qualification, whether in person or over the phone does not require written consent.
- At the onsite screening, candidates should have gait speed measured over 4 meters of <1.0 m/s but ≥0.6 m/s.
- Appendicular skeletal muscle index (skeletal muscle in kg/ height in m2) by DXA ≤ 7.25 kg/m2 for men and ≤ 5.67 kg/m2 for women to be assessed during screening if the gait speed criterion in #3 is met.
- At screening and baseline, vital signs (systolic and diastolic blood pressure and pulse rate) will be assessed in the supine position followed by assessment in standing within three (3) minutes of changing position. Investigators can be guided by the following ranges for the supine measurement and position change:
- oral body temperature between 35.0-37.5 °C
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- systolic blood pressure, 80-160 mm Hg and a reduction of >20mmHg
- diastolic blood pressure, 50-90 mm Hg and a reduction of >10mmHg
- pulse rate, 50 - 100 bpm
- Patients must weigh at least 35 kg to participate in the study, and must have a body mass index (BMI) within the range of 18 - 32 kg/m2, inclusive. (See Appendix 4) of this protocol for BMI ranges.
- Be able to communicate well with the investigator, to understand and comply with the requirements of the study.
Who cannot join
- Use of other investigational drugs at the time of enrollment, or within 30 days or 5 halflives of enrollment, whichever is longer; or longer if required by local regulations, and for any other limitation of participation in an investigational trial based on local regulations.
- History of hypersensitivity to antibodies.
- A history of clinically significant ECG abnormalities, which, in the opinion of the investigator, may indicate active cardiac disease.
- History of malignancy of any organ system (other than localized nonmelanomatous cell 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, COPD, 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.
- Diseases known to cause malabsorption of protein or energy, including inflammatory bowel disease, celiac disease, short bowel syndrome, pancreatic insufficiency, etc.
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- Liver disease or liver injury as indicated by abnormal liver function tests such as SGOT (AST), SGPT (ALT), γ-GT, alkaline phosphatase, or serum bilirubin (except Gilbert's
- Disease). The Investigator should be guided by the following criteria:
- Any single transaminase listed above may not exceed 3x upper limit of normal (ULN).
- If the total bilirubin concentration is increased above 1.5 x ULN, total bilirubin should be differentiated into the direct and indirect reacting bilirubin. In any case, serum bilirubin should not exceed the value of 1.6 mg/dL (27 μmol/L). 8. 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, etc. 9. Donation or loss of 400 mL or more of blood within eight (8) weeks prior to initial dosing, or longer if required by local regulation. 10. Plasma donation (> 250 mL) within 14 days prior to first dosing. 11. Hemoglobin concentration below 11.0 g/dL at screening. 12. Significant illness within two (2) weeks prior to initial dosing. 13. Patients with known claustrophobia, presence of pacemaker and/or ferromagnetic material in their body that would prohibit administration of MRI assessments. 14. Patient smokes more than one cigarette, pipe or cigar a month
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Novartis Investigative Site
Brighton, Massachusetts, 02135
Novartis Investigative Site
Hot Springs, Arkansas, 71913
Novartis Investigative Site
Little Rock, Arkansas, 72201
Novartis Investigative Site
Miami, Florida, 33143
Novartis Investigative Site
Miami, Florida, 33169
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Multi-center Study to Assess the Effects of BYM338 on Skeletal Muscle in Sarcopenic Adults With Mobility Limitations
The purpose of this Proof of Concept study is to determine the effects of BYM338 on skeletal muscle volume, mass, and strength and patient function (gait speed) in non-demented elderly adults with sarcopenia and mobility limitations. In addition, this study will generate data on the safety, tolerability, and pharmacokinetics of BYM338 in older adults and its response on additional physical function measures in this population. Furthermore, the extended study duration will provide information on the stability of BYM-induced changes in skeletal muscle and patient function in this subgroup of the older population.
- Conditions
- Skeletal Muscle
- Being tested
- BYM338, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BYM338Drug
BYM338
PlaceboDrug
BYM338 Placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| BYM338 | Experimental | BYM338 active drug Receives: Drug: BYM338 |
What is being measured
The primary outcome is the main question the study is designed to answer.
muscle volume of the thigh (measurement gathered using MRI, magnetic resonance imaging)
Measured: baseline to 24 weeks
Secondary outcomes (6)
Safety and tolerability will be measured by physical examination, checking vital signs, ECG evaluation and taking standard laboratory tests. Clinical safety data will be compiled by Adverse events reports.
Measured: 24 weeks
Pharmacokinetic profile will measure the (Composite of Pharmacokinetics)area under curve (AUC)
Measured: pre-dose, 2, 6 hrs postdose, 1,2,4,6,8,10,12,16,20,40 weeks postdose
Muscle function measured by evaluating physical performance tests
Measured: 24 weeks
Health status measured by evaluating patient reports.
Measured: 24 weeks
Duration of drug-induced changes in body composition
Measured: 24 hours
Duration of drug-induced physical function
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2012 (estimated)
- Study startJan 2012
- Primary completionDec 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Skeletal MuscleStudies 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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 40 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
- NCT01601600
- Sponsor's protocol ID
- CBYM338X2201
- Status last verified
- Nov 2016
- First submitted
- 17 Feb 2012
- Last updated
- 17 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Multi-center Study to Assess the Effects of BYM338 on Skeletal Muscle in Sarcopenic Adults With Mobility Limitations
- ClinicalTrials.gov ID
- NCT01601600
- Acronym / sponsor ID
- CBYM338X2201
- Conditions
- Skeletal Muscle
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jan 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 18 May 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this Proof of Concept study is to determine the effects of BYM338 on skeletal muscle volume, mass, and strength and patient function (gait speed) in non-demented elderly adults with sarcopenia and mobility limitations. In addition, this study will generate data on the safety, tolerability, and pharmacokinetics of BYM338 in older adults and its response on additional physical function measures in this population. Furthermore, the extended study duration will provide information on the stability of BYM-induced changes in skeletal muscle and patient function in this subgroup of the older population.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| BYM338 | Experimental | Drug: BYM338BYM338 active drug |
Who may qualify
- Ages
- 65 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (11 listed)
- Men or women aged 65 or older with difficulty standing up from a chair or walking for longer than 10 minutes on a flat surface or climbing a flight of stairs.
- Written informed consent must be obtained before any laboratory or physical assessment is performed to establish eligibility. Answering initial questions for qualification, whether in person or over the phone does not require written consent.
- At the onsite screening, candidates should have gait speed measured over 4 meters of <1.0 m/s but ≥0.6 m/s.
- Appendicular skeletal muscle index (skeletal muscle in kg/ height in m2) by DXA ≤ 7.25 kg/m2 for men and ≤ 5.67 kg/m2 for women to be assessed during screening if the gait speed criterion in #3 is met.
- At screening and baseline, vital signs (systolic and diastolic blood pressure and pulse rate) will be assessed in the supine position followed by assessment in standing within three (3) minutes of changing position. Investigators can be guided by the following ranges for the supine measurement and position change:
Key exclusion criteria (10 listed)
- Use of other investigational drugs at the time of enrollment, or within 30 days or 5 halflives of enrollment, whichever is longer; or longer if required by local regulations, and for any other limitation of participation in an investigational trial based on local regulations.
- History of hypersensitivity to antibodies.
- A history of clinically significant ECG abnormalities, which, in the opinion of the investigator, may indicate active cardiac disease.
- History of malignancy of any organ system (other than localized nonmelanomatous cell 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, COPD, 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.
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 |
|---|---|---|
| BYM338BYM338 | Drug | BYM338 |
| PlaceboBYM338 Placebo | Drug | Placebo |
Primary outcome
muscle volume of the thigh (measurement gathered using MRI, magnetic resonance imaging)
Time frame: baseline to 24 weeks
6 secondary outcomes
Safety and tolerability will be measured by physical examination, checking vital signs, ECG evaluation and taking standard laboratory tests. Clinical safety data will be compiled by Adverse events reports.
Time frame: 24 weeks
Pharmacokinetic profile will measure the (Composite of Pharmacokinetics)area under curve (AUC)
Time frame: pre-dose, 2, 6 hrs postdose, 1,2,4,6,8,10,12,16,20,40 weeks postdose
Muscle function measured by evaluating physical performance tests
Time frame: 24 weeks
Health status measured by evaluating patient reports.
Time frame: 24 weeks
Duration of drug-induced changes in body composition
Time frame: 24 hours
Duration of drug-induced physical function
Time frame: 24 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2020.
- Results for CBYM338X2201 from the Novartis Clinical Trials Results WebsiteLink provided by the sponsor.
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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
5 sites in 1 country: 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 Skeletal Muscle, run by Novartis Pharmaceuticals at 5 sites in United States.
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 11 inclusion and 10 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 17 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
muscle volume of the thigh (measurement gathered using MRI, magnetic resonance imaging) (time frame: baseline to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Dec 2020.