Mitochondrial MyopathyPhase 1/2MMPOWERNCT02367014
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
A Phase 1/2 study testing elamipretide (low dose), elamipretide (intermediate dose) and elamipretide (high dose) in people with Mitochondrial Myopathy, run by Stealth BioTherapeutics Inc. at 4 sites in United States.
Trial summary
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this… Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Mitochondrial Myopathy
- What is being tested?
- elamipretide (low dose), elamipretide (intermediate dose) and elamipretide (high dose)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 4 sitesUnited States
- Study dates
- Feb 2015to Apr 2016
- Phase
- Phase 1/2
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of mitochondrial disease believed to impair the mitochondrial respiratory chain.
- Eligibility requires prior genetic confirmation of mitochondrial disease.
- Diagnosis of mitochondrial myopathy judged by the Investigators to be due to existing mitochondrial disease.
- Must be able to complete a Screening Visit 6MWT.
- Body mass index (BMI) score >15.0 and <35.0 kg/m2 at Screening Visit.
- Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the ICF until two months after the last dose of study drug.
Who cannot join
- Any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all study requirements.
- Had any exclusionary Newcastle Mitochondrial Disease Adult Scale (NMDAS) scores at Screening Visit.
- Hospitalized (admitted as in-patient) within 1 month prior to the Baseline Visit.
- A history of type 1 diabetes mellitus (T1DM).
- Uncontrolled Type 1 (T1DM) or Type 2 diabetes mellitus (T2DM), in the opinion of the investigator.
- A creatinine clearance <45 mL/min as calculated by the Cockcroft Gault equation.
Show all 13Show fewer
- Requires pacemaker, defibrillator, or has undergone cardiac surgery within 2 years of Screening Visit.
- QTc elongation defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
- Uncontrolled hypertension (>160 mmHg systolic or >100 mmHg diastolic) at Screening Visit.
- History of rhabdomyolysis defined as an acute rise in the serum creatine phosphokinase (CPK) value that, in the opinion of the investigator, caused clinically significant symptoms.
- Serum sodium more than 5 meq/L below the reference lower limit of normal at Screening Visit.
- Participated in another interventional clinical trial within 3 months of the screening visit or is currently enrolled in a non-interventional clinical trial judged by the Investigator to be incompatible with the current trial.
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Akron Children's Hospital
Akron, Ohio, 44308
Massachusetts General Hospital
Boston, Massachusetts, 02114
Children's Hospital of Pittsburg of UPMC
Pittsburgh, Pennsylvania, 15213
University of California
San Diego, California, 92093
About this study
Official title: Phase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial Disease
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this patient population.
Read the detailed description
This multi-center, randomized, double-blind, placebo-controlled study enrolled 36 subjects into 3 cohorts of 12 subjects each to evaluate treatment with 3 ascending doses of intravenous elamipretide (0.01, 0.10, and 0.25 mg/kg/hr infused for 2 hours). After each cohort, a Safety Monitoring Board (SMB) determined if dose escalation to the next higher dose of elamipretide was warranted. Each cohort went through 3 distinct periods: Screening, Treatment, and Follow-up.
The Screening Period started with informed consent and may have lasted up to 40 days. During this period, screening procedures to determine subject eligibility for the study occurred, including confirmation of disease, which incorporated a committee review of the investigator-submitted diagnosis and genetic results. The Treatment Period began on Day 1 (Visit 2) and lasted for 5 days (until Day 5 [Visit 6]). Within each cohort, 9 subjects were randomized to active drug and 3 subjects were randomized to placebo on Day 1 and subjects received treatment once a day for 5 consecutive days. Safety, tolerability, and efficacy measures were performed at pre-specified times. The Follow-up Period began at the time of discharge on Day 5. Subjects returned to the study center for the Follow-up Visit on Day 7 (+1 day).
- Conditions
- Mitochondrial Myopathy
- Being tested
- elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose), Placebo
- Keywords
- Mitochondrial Myopathy, Primary Mitochondrial Disease, Bendavia™, elamipretide
- Medical subject headings
- Mitochondrial Myopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elamipretide (low dose)Drug
Also called MTP-131
elamipretide (0.01 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days
elamipretide (intermediate dose)Drug
Also called MTP-131
elamipretide (0.10 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days
elamipretide (high dose)Drug
Also called MTP-131
elamipretide (0.25 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days
PlaceboDrug
placebo (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days
| Study group | Role | What they receive |
|---|---|---|
| Low Dose | Experimental | elamipretide 0.01 mg/kg/hr infused for 2 hours for 5 days Receives: Drug: elamipretide (low dose) |
| Intermediate dose | Experimental | elamipretide 0.10 mg/kg/hr infused for 2 hours for 5 days Receives: Drug: elamipretide (intermediate dose) |
| High dose | Experimental | elamipretide 0.25 mg/kg/hr infused for 2 hours for 5 days Receives: Drug: elamipretide (high dose) |
| Placebo | Placebo comparator | In each cohort, subjects received either IV elamipretide given once daily for 2 hours for 5 days or matching placebo. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)
Change in distance walked as measured by meters on the 6-minute walk test from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Assessed at Baseline, Day 5 (end-of-treatment visit)
Secondary outcomes (27)
Change in Maximum Oxygen Uptake (ml/kg/Min)
Change in maximum oxygen uptake as measured by mL/kg/min from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Ventilatory Efficiency (VE/VCO2 Slope)
Change in ventilatory efficiency as measured by the VE/VCO2 slope from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio)
Change in aerobic efficiency as measured by ΔO2 consumption/Δ work ratio from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio)
Change in oxygen utilization as measured by ΔVO2/ΔlogVE ratio from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds)
Change in oxygen uptake kinetics (mean response time) as measured by seconds from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Pre-exercise Lactate Levels (mg/dL)
Change in pre-exercise lactate levels as measured by mg/dL from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Post-exercise Lactate Levels (mg/dL)
Change in post-exercise lactate levels as measured by mg/dL from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Respiratory Exchange Ratio (VCO2/VO2)
Change in peak respiratory exchange ratio as measured by VCO2/VO2 from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Respiratory Rate (Breaths/Min)
Change in peak respiratory rate as measured by breaths/min from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Ventilation (L/Min)
Change in peak ventilation as measured by L/min from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Heart Rate (Beats/Min)
Change in peak heart rate as measured by beats per minute from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Oxygen Saturation (% O2-saturated Hemoglobin)
Change in peak oxygen saturation as measured by percentage of O2-saturated hemoglobin from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Systolic Blood Pressure (mmHg)
Change in peak systolic blood pressure as measured by mmHg from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Diastolic Blood Pressure (mmHg)
Change in peak diastolic blood pressure as measured by mmHg from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Peak Borg Dyspnea
Change in peak Borg dyspnea as measured by 0-10 with 0 meaning no breathlessness and 10 meaning maximal breathlessness from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in VO2 Anaerobic Threshold (mL)
Change in VO2 anaerobic threshold as measured by mL from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Watts
Change in watts from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Temperature (°C)
Change in temperature as measured by units of Celsius from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in ECG-PR Interval (Msec)
Change in PR interval as measured by ECG in milliseconds from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in ECG-QRS Complex (Msec)
Change in QRS complex as measured by ECG in msec from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in ECG-QT Interval (Msec)
Change in QT interval as measured by ECG in msec from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in ECG-QTc Interval (Msec)
Change in QTc interval as measured by ECG in msec from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Number of Participants Who Had Suicide Ideation, Suicidal Behavior, or Non-suicidal Self-injurious Behavior Post-screening.
Number of participants with suicide ideation, suicidal behavior, or non-suicidal self-injurious behavior post-screening as measured on Days 1-5, and Day 7 on the Columbia Suicide Severity Rating Scale (CSSRS). A yes/no binary response is utilized in the following ten categories: 1 - Wish to be Dead; 2 - Non-specific Active Suicidal Thoughts; 3 - Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act; 4 - Active Suicidal Ideation with Some Intent to Act, without Specific Plan; 5 - Active Suicidal Ideation with Specific Plan and Intent; 6 - Preparatory Acts or Behavior; 7 - Aborted Attempt; 8 - Interrupted Attempt; Category 9 - Actual Attempt (non-fatal); 10 - Completed Suicide. A yes/no binary response is also utilized in assessing self-injurious behavior without suicidal intent. A lower score means a better outcome whereas a higher score means a worse outcome.
Measured: Days 1-5 and Day 7.
Change in Creatine Phosphokinase (IU/L)
Change in Creatine Phosphokinase as measured by IU/L from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Alanine Aminotransferase (ALT) (U/L)
Change in Alanine aminotransferase (ALT) as measured by (U/L) from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Aspartate Aminotransferase (AST) (U/L)
Change in aspartate aminotransferase (AST) as measured by U/L from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Change in Eosinophils (10^9 Cells/L)
Change in eosinophils as measured by (10^9 cells/L) from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
Measured: Baseline, Day 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2015 (estimated)
- Study startFeb 2015
- Primary completionApr 2016
- Study completionApr 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 Mitochondrial MyopathyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02367014
- Sponsor's protocol ID
- SPIMM-201
- Acronym
- MMPOWER
- Status last verified
- Dec 2019
- First submitted
- 9 Feb 2015
- Last updated
- 27 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial Disease
- ClinicalTrials.gov ID
- NCT02367014
- Acronym / sponsor ID
- MMPOWER / SPIMM-201
- Conditions
- Mitochondrial Myopathy
- Keywords
- Mitochondrial Myopathy, Primary Mitochondrial Disease, Bendavia™, elamipretide
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Stealth BioTherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2015
- Primary completion
- Apr 2016 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 20 Feb 2015 (estimated)
- Results
- Posted 27 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this patient population.
Detailed description
This multi-center, randomized, double-blind, placebo-controlled study enrolled 36 subjects into 3 cohorts of 12 subjects each to evaluate treatment with 3 ascending doses of intravenous elamipretide (0.01, 0.10, and 0.25 mg/kg/hr infused for 2 hours). After each cohort, a Safety Monitoring Board (SMB) determined if dose escalation to the next higher dose of elamipretide was warranted. Each cohort went through 3 distinct periods: Screening, Treatment, and Follow-up.
The Screening Period started with informed consent and may have lasted up to 40 days. During this period, screening procedures to determine subject eligibility for the study occurred, including confirmation of disease, which incorporated a committee review of the investigator-submitted diagnosis and genetic results. The Treatment Period began on Day 1 (Visit 2) and lasted for 5 days (until Day 5 [Visit 6]). Within each cohort, 9 subjects were randomized to active drug and 3 subjects were randomized to placebo on Day 1 and subjects received treatment once a day for 5 consecutive days. Safety, tolerability, and efficacy measures were performed at pre-specified times. The Follow-up Period began at the time of discharge on Day 5. Subjects returned to the study center for the Follow-up Visit on Day 7 (+1 day).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Low Dose | Experimental | Drug: elamipretide (low dose)elamipretide 0.01 mg/kg/hr infused for 2 hours for 5 days |
| Intermediate dose | Experimental | Drug: elamipretide (intermediate dose)elamipretide 0.10 mg/kg/hr infused for 2 hours for 5 days |
| High dose | Experimental | Drug: elamipretide (high dose)elamipretide 0.25 mg/kg/hr infused for 2 hours for 5 days |
| Placebo | Placebo comparator | Drug: PlaceboIn each cohort, subjects received either IV elamipretide given once daily for 2 hours for 5 days or matching placebo. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of mitochondrial disease believed to impair the mitochondrial respiratory chain.
- Eligibility requires prior genetic confirmation of mitochondrial disease.
- Diagnosis of mitochondrial myopathy judged by the Investigators to be due to existing mitochondrial disease.
- Must be able to complete a Screening Visit 6MWT.
- Body mass index (BMI) score >15.0 and <35.0 kg/m2 at Screening Visit.
Key exclusion criteria (13 listed)
- Any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all study requirements.
- Had any exclusionary Newcastle Mitochondrial Disease Adult Scale (NMDAS) scores at Screening Visit.
- Hospitalized (admitted as in-patient) within 1 month prior to the Baseline Visit.
- A history of type 1 diabetes mellitus (T1DM).
- Uncontrolled Type 1 (T1DM) or Type 2 diabetes mellitus (T2DM), in the opinion of the investigator.
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 |
|---|---|---|
| elamipretide (low dose)Also: MTP-131elamipretide (0.01 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days | Drug | Low Dose |
| elamipretide (intermediate dose)Also: MTP-131elamipretide (0.10 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days | Drug | Intermediate dose |
| elamipretide (high dose)Also: MTP-131elamipretide (0.25 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days | Drug | High dose |
| Placeboplacebo (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days | Drug | Placebo |
Primary outcome
Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT)
Time frame: Assessed at Baseline, Day 5 (end-of-treatment visit)
Change in distance walked as measured by meters on the 6-minute walk test from baseline (last assessment prior to start of study) to Day 5 (end of treatment visit).
27 secondary outcomes
Change in Maximum Oxygen Uptake (ml/kg/Min)
Time frame: Baseline, Day 5
Change in Ventilatory Efficiency (VE/VCO2 Slope)
Time frame: Baseline, Day 5
Change in Aerobic Efficiency (ΔO2 Consumption/Δ Work Ratio)
Time frame: Baseline, Day 5
Change in Oxygen Utilization (ΔVO2/ΔlogVE Ratio)
Time frame: Baseline, Day 5
Change in Oxygen Uptake Kinetics (Mean Response Time as Measured by Seconds)
Time frame: Baseline, Day 5
Change in Pre-exercise Lactate Levels (mg/dL)
Time frame: Baseline, Day 5
Change in Post-exercise Lactate Levels (mg/dL)
Time frame: Baseline, Day 5
Change in Peak Respiratory Exchange Ratio (VCO2/VO2)
Time frame: Baseline, Day 5
Change in Peak Respiratory Rate (Breaths/Min)
Time frame: Baseline, Day 5
Change in Peak Ventilation (L/Min)
Time frame: Baseline, Day 5
Change in Peak Heart Rate (Beats/Min)
Time frame: Baseline, Day 5
Change in Peak Oxygen Saturation (% O2-saturated Hemoglobin)
Time frame: Baseline, Day 5
Change in Peak Systolic Blood Pressure (mmHg)
Time frame: Baseline, Day 5
Change in Peak Diastolic Blood Pressure (mmHg)
Time frame: Baseline, Day 5
Change in Peak Borg Dyspnea
Time frame: Baseline, Day 5
Change in VO2 Anaerobic Threshold (mL)
Time frame: Baseline, Day 5
Change in Watts
Time frame: Baseline, Day 5
Change in Temperature (°C)
Time frame: Baseline, Day 5
Change in ECG-PR Interval (Msec)
Time frame: Baseline, Day 5
Change in ECG-QRS Complex (Msec)
Time frame: Baseline, Day 5
Change in ECG-QT Interval (Msec)
Time frame: Baseline, Day 5
Change in ECG-QTc Interval (Msec)
Time frame: Baseline, Day 5
Number of Participants Who Had Suicide Ideation, Suicidal Behavior, or Non-suicidal Self-injurious Behavior Post-screening.
Time frame: Days 1-5 and Day 7.
Change in Creatine Phosphokinase (IU/L)
Time frame: Baseline, Day 5
Change in Alanine Aminotransferase (ALT) (U/L)
Time frame: Baseline, Day 5
Change in Aspartate Aminotransferase (AST) (U/L)
Time frame: Baseline, Day 5
Change in Eosinophils (10^9 Cells/L)
Time frame: Baseline, Day 5
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing elamipretide (low dose), elamipretide (intermediate dose) and elamipretide (high dose) in people with Mitochondrial Myopathy, run by Stealth BioTherapeutics Inc. at 4 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 6 inclusion and 13 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 27 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 27 Dec 2019). 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 in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) (time frame: Assessed at Baseline, Day 5 (end-of-treatment visit)). 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 27 Dec 2019.