Skeletal Muscle Mitochondrial Dysfunction in the ElderlyPhase 2MOTIONNCT02245620
A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly
A Phase 2 study testing Elamipretide in people with Skeletal Muscle Mitochondrial Dysfunction in the Elderly, run by Stealth BioTherapeutics Inc. at 1 site in United States.
Trial summary
This was a Phase 2, randomized, double-blind, placebo-controlled study, enrolling 41 elderly subjects with previous evidence of mitochondrial dysfunction to evaluate whether the administration of MTP-131 (elamipretide) will change either hand skeletal muscle energetics or muscle performance in age-related skeletal… Read more about this study
- Who is it for?
- Ages 60 to 85 years
- Conditions the study is for
- Skeletal Muscle Mitochondrial Dysfunction in the Elderly
- What is being tested?
- Elamipretide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jan 2015to 6 Jul 2016
- 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 60 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Are male and female adults aged ≥60 and ≤85 years
- Female subjects must be post-menopausal
- Have in vivo phosphorus-31 (31P) Magnetic Resonance Spectroscopy (MRS) and (Optical Spectra Scan (OPS) determined maximum adenosine triphosphate synthetic rate (ATPmax) < 0.70 milliMol/second (mM/sec)
- Have in vivo 31P MRS and OPS determined P/O (Phosphate/Oxygen ratio) < 1.9
- Are ambulatory and able to perform activities of daily living without assistance
- Had sufficient venous access for study drug administration and clinical testing.
- Could speak and read English fluently.
- Provided informed consent.
Who cannot join
- Had significant disease(s) or condition(s) which, in the opinion of the Investigator, may have put the subject at risk because of their participation in the study or may have influenced either the results of the study or the subject's ability to participate in the study.
- Had a history of rhabdomyolysis.
- Had been hospitalized within 3 months prior to Screening for major atherosclerotic events (e.g., myocardial infarction, target-vessel revascularization, coronary bypass surgery or stroke) or other major medical condition (as deemed by the Primary Investigator).
- Had any metal implants that could not be removed from the body that in the opinion of the Investigator were a contra-indication for undergoing the MRS procedure or any other protocol-related procedure.
- Had an implanted cardiac pacemaker or other implanted cardiac device.
- Had a serum sodium level <136 milliequivalents per litre (mEq/L) at Screening or Pre-infusion.
Show all 27Show fewer
- Had a hemoglobin level <12 g/dL at Screening or Pre-infusion.
- Had chronic, uncontrolled hypertension as judged by the Investigator (e.g., Baseline systolic blood pressure [SBP] >140 mm Hg, diastolic blood pressure [DBP] > 90 mm Hg) or a SBP >150 mm Hg or DBP >95 mm Hg at the time of Screening or Baseline (if the initial blood pressure [BP] reading was above these values, the reading may have been repeated one time within 20 minutes of the initial reading).
- Had a body mass index (BMI) of <16 or >35 kg/m2.
- Had a creatinine clearance <45 mL/min as calculated by the Cockcroft Gault equation.
- Had a 12-lead electrocardiogram (ECG) demonstrating severe bradycardia (heart rate < 40 bpm) or average corrected QC interval (QTc) >450 ms for males and > 470 ms for females, and in the opinion of the Investigator was clinically significant. (If on the initial ECG, QTc exceeded 450 ms for males or 470 ms for females, the ECG was to be repeated 2 more times and the average of the 3 QTc values was to be used to determine the subject's eligibility).
- Had a neurologic disorder that in the opinion of the Investigator was a contra-indication for enrollment into the study.
- Had any symptoms consistent with or a current diagnosis of peripheral neuropathy, such as numbness, tingling, pain, or altered sensation of hands or feet.
- Had an active, systemic autoimmune disease other than autoimmune thyroid disease (e.g., diabetes, lupus, rheumatoid arthritis) that currently required treatment or was likely to require treatment during the study.
- Subject's right hand had a history of mobility impairment, fractures, arthritis, hand surgery, muscle disease or other injury that may interfere with any study procedure.
- Had any symptoms consistent with or a current diagnosis of claustrophobia.
- Had a history of cancer, unless subject had documentation of completed curative treatment.
- Had a history of or risk factors (e.g., significant family history, concomitant medical condition) for deep vein thrombosis or pulmonary embolism.
- Had a history of serious mental illness as judged by the Investigator.
- Had a body temperature > 37.5°C at the time of planned dosing.
- Subjects who in the opinion of the Investigator abused alcohol or drugs.
- Had donated or received blood or blood products within the past 30 days.
- Investigator site personnel directly affiliated with this study and/or their immediate families. Immediate family was defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
- Sponsor employees and/or their immediate families. Immediate family was defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
- Were currently enrolled in a clinical study involving an investigational product or non-approved use of a drug or device or concurrently enrolled in any other type of medical research judged to be scientifically or medically incompatible with this study.
- Had participated, within the last 30 days, in a clinical study involving an investigational product. If the previous investigational product had a long half-life, 3 months or 5 half-lives (whichever was longer) should have passed.
- Had previously been randomized into any study investigating elamipretide or been exposed to elamipretide for any reason.
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Washington Medical Center
Seattle, Washington, 98104
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Impact of a Single Intravenous Dose of MTP-131 (Bendavia™) on Skeletal Muscle Function in the Elderly
This was a Phase 2, randomized, double-blind, placebo-controlled study, enrolling 41 elderly subjects with previous evidence of mitochondrial dysfunction to evaluate whether the administration of MTP-131 (elamipretide) will change either hand skeletal muscle energetics or muscle performance in age-related skeletal muscle mitochondrial dysfunction.
Read the detailed description
This was a Phase 2, randomized, double-blind, placebo-controlled study, enrolling 41 elderly subjects with previous evidence of mitochondrial dysfunction to evaluate whether the administration of MTP-131 (elamipretide) will change either hand skeletal muscle energetics or muscle performance in age-related skeletal muscle mitochondrial dysfunction.
Subjects were randomized 1:1 to receive either elamipretide at 0.25 mg/kg/hr intravenously at a rate of 60 mL/hr for 2 hours, or placebo (lyophilized excipients without elamipretide) intravenously at a rate of 60 mL/hr for 2 hours. Each treatment group went through three distinct periods: Screening (up to 28 days), Treatment (1day), and Observation (7 days).
- Being tested
- Elamipretide, Placebo
- Keywords
- Skeletal muscle dysfunction, MTP-131, Bendavia™, elamipretide
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElamipretideDrug
Also called MTP-131, Bendavia™
Elamipretide 0.25 mg/kg/hour administered as an intravenous infusion at the rate of 60 mL/hour for 2 hours
PlaceboDrug
Also called elamipretide
Placebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours
| Study group | Role | What they receive |
|---|---|---|
| Elamipretide | Experimental | Elamipretide given as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours. Receives: Drug: Elamipretide |
| Placebo | Placebo comparator | Placebo (lyophilized excipients without elamipretide) given as an intravenous infusion at a rate of 60 mL/hr for 2 hours. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in ATPmax (Maximal ATP Synthetic Rate)
Maximal ATP synthetic rate (phosphorylation capacity per unit muscle volume) as determined by a muscle fatigue test.
Measured: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Secondary outcomes (3)
Mean Change From Baseline in Phosphate/Oxygen (P/O) Ratio
As a measure of mitochondrial hand skeletal muscle energetics, mitochondrial coupling, or Phosphate/Oxygen ratio (P/O) was assessed at Baseline, Day 1 Hour 2, and Day 7.
Measured: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Mean Change From Baseline in Nicotine Adenine Dinucleotide (NAD)
Measured: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Mean Change From Baseline in Muscle Force-Time-Integral (FTI)
Muscle Force-Time-Integral was measured as mean change from baseline at Day 1 Hour 2, Day 3, and Day 7.
Measured: From Baseline to Day 1 Hour 2, Day 3, and Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2014 (estimated)
- Study start15 Jan 2015
- Primary completion6 Jul 2016
- Study completion6 Jul 2016
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.
- Other trials for Skeletal Muscle Mitochondrial Dysfunction in the ElderlyStudies of the same condition, open and under way.
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jim Carr, Study director, Stealth BioTherapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02245620
- Sponsor's protocol ID
- SPITM-201
- Acronym
- MOTION
- Status last verified
- Jun 2020
- First submitted
- 17 Sep 2014
- Last updated
- 23 Jun 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Impact of a Single Intravenous Dose of MTP-131 (Bendavia™) on Skeletal Muscle Function in the Elderly
- ClinicalTrials.gov ID
- NCT02245620
- Acronym / sponsor ID
- MOTION / SPITM-201
- Keywords
- Skeletal muscle dysfunction, MTP-131, Bendavia™, elamipretide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Stealth BioTherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Jan 2015 (actual)
- Primary completion
- 6 Jul 2016 (actual)
- Study completion
- 6 Jul 2016 (actual)
- First posted
- 19 Sep 2014 (estimated)
- Results
- Posted 23 Jun 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a Phase 2, randomized, double-blind, placebo-controlled study, enrolling 41 elderly subjects with previous evidence of mitochondrial dysfunction to evaluate whether the administration of MTP-131 (elamipretide) will change either hand skeletal muscle energetics or muscle performance in age-related skeletal muscle mitochondrial dysfunction.
Detailed description
This was a Phase 2, randomized, double-blind, placebo-controlled study, enrolling 41 elderly subjects with previous evidence of mitochondrial dysfunction to evaluate whether the administration of MTP-131 (elamipretide) will change either hand skeletal muscle energetics or muscle performance in age-related skeletal muscle mitochondrial dysfunction.
Subjects were randomized 1:1 to receive either elamipretide at 0.25 mg/kg/hr intravenously at a rate of 60 mL/hr for 2 hours, or placebo (lyophilized excipients without elamipretide) intravenously at a rate of 60 mL/hr for 2 hours. Each treatment group went through three distinct periods: Screening (up to 28 days), Treatment (1day), and Observation (7 days).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Elamipretide | Experimental | Drug: ElamipretideElamipretide given as an intravenous infusion of 0.25 mg/kg/hr at a rate of 60 mL/hr for 2 hours. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo (lyophilized excipients without elamipretide) given as an intravenous infusion at a rate of 60 mL/hr for 2 hours. |
Who may qualify
- Ages
- 60 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Are male and female adults aged ≥60 and ≤85 years
- Female subjects must be post-menopausal
- Have in vivo phosphorus-31 (31P) Magnetic Resonance Spectroscopy (MRS) and (Optical Spectra Scan (OPS) determined maximum adenosine triphosphate synthetic rate (ATPmax) < 0.70 milliMol/second (mM/sec)
- Have in vivo 31P MRS and OPS determined P/O (Phosphate/Oxygen ratio) < 1.9
- Are ambulatory and able to perform activities of daily living without assistance
Key exclusion criteria (27 listed)
- Had significant disease(s) or condition(s) which, in the opinion of the Investigator, may have put the subject at risk because of their participation in the study or may have influenced either the results of the study or the subject's ability to participate in the study.
- Had a history of rhabdomyolysis.
- Had been hospitalized within 3 months prior to Screening for major atherosclerotic events (e.g., myocardial infarction, target-vessel revascularization, coronary bypass surgery or stroke) or other major medical condition (as deemed by the Primary Investigator).
- Had any metal implants that could not be removed from the body that in the opinion of the Investigator were a contra-indication for undergoing the MRS procedure or any other protocol-related procedure.
- Had an implanted cardiac pacemaker or other implanted cardiac device.
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 |
|---|---|---|
| ElamipretideAlso: MTP-131, Bendavia™Elamipretide 0.25 mg/kg/hour administered as an intravenous infusion at the rate of 60 mL/hour for 2 hours | Drug | Elamipretide |
| PlaceboAlso: elamipretidePlacebo administered as intravenous infusion at a rate of 60 mL/hour for 2 hours | Drug | Placebo |
Primary outcome
Change From Baseline in ATPmax (Maximal ATP Synthetic Rate)
Time frame: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Maximal ATP synthetic rate (phosphorylation capacity per unit muscle volume) as determined by a muscle fatigue test.
3 secondary outcomes
Mean Change From Baseline in Phosphate/Oxygen (P/O) Ratio
Time frame: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Mean Change From Baseline in Nicotine Adenine Dinucleotide (NAD)
Time frame: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7
Mean Change From Baseline in Muscle Force-Time-Integral (FTI)
Time frame: From Baseline to Day 1 Hour 2, Day 3, and Day 7
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.
Jim Carr study director
Stealth BioTherapeutics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Elamipretide in people with Skeletal Muscle Mitochondrial Dysfunction in the Elderly, run by Stealth BioTherapeutics Inc. at 1 site 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 8 inclusion and 27 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 23 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Yes. Results are posted in the registry (first posted 23 Jun 2020). 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?
Change From Baseline in ATPmax (Maximal ATP Synthetic Rate) (time frame: From Baseline, Day 1 Hour 2 (2 hours after the start of infusion, or end of infusion) and Day 7). 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 23 Jun 2020.