Primary Mitochondrial MyopathyPhase 3MMPOWER-3NCT03323749
A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
A Phase 3 study testing elamipretide and elamipretide open label treatment in people with Primary Mitochondrial Myopathy, run by Stealth BioTherapeutics Inc. at 27 sites in 7 countries.
Trial summary
This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension. Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Primary Mitochondrial Myopathy
- What is being tested?
- elamipretide and elamipretide open label treatment
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 218 participants
- Where
- 27 sitesin 7 countries
- Study dates
- 9 Oct 2017to 10 Feb 2020
- Phase
- Phase 3
- 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 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willing and able to provide a signed informed consent form prior to participation in any trial-related procedures
- Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system
- Subject is ≥ 16 and ≤ 80 years of age
- Diagnosed with PMM in the opinion of the investigator and confirmed by an Adjudication Committee
- Woman of childbearing potential must agree to use a highly effective method of birth control
Who cannot join
- Subject has myopathic signs and or/symptoms due to a neuropathic process or gait problem that would interfere with the 6 minute walk test (6MWT), in the opinion of the Investigator
- Female who are pregnant, planning to become pregnant, or breastfeeding/lactating
- At Screening, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m^2
- Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
- Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, in the opinion of the Investigator.
- Subject has QTc elongation (using the correction factor utilized at the clinical site) defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
Show all 26Show fewer
- ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
- First degree Atrioventricular bock (AV-block)
- Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
- Right bundle branch block
- Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements
- Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements.
- Active malignancy or any other cancer from which the subject has been disease-free for < 2 years.
- Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator.
- Subject has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection.
- Subject has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x10^6/L at the Screening Visit.
- Subject is currently participating or has participated in an interventional clinical trial (i.e.,investigational product or device, stem cell therapy, gene therapy) within 30 days of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding with results of the current trial (e.g., exercise therapy trial).
- Subject has previously received elamipretide (MTP-131), for any reason.
- Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator.
- Subject has 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 trial requirements.
- PART 2:
- Continuation Criteria:
- Subjects must continue to be able and willing to adhere to the trial requirements.
- Subject is appropriate to continue in Part 2 (i.e. subject was compliant in Part 1), in the opinion of the Investigator.
- Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE) attributed to the elamipretide delivery system.
- Subject has not permanently discontinued the elamipretide delivery system.
Trial locations and contacts
27 sites in 7 countries.
27 sites in 7 countries
United States13 sites
Akron Children's Hospital
Akron, Ohio, 44308
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Children's Hospital Colorado
Aurora, Colorado, 80045
Massachusetts General Hospital
Boston, Massachusetts, 02114
Cleveland Clinical Neurological Institute
Cleveland, Ohio, 44195
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, 77030
University of Texas Health Science Center
Houston, Texas, 77030
University of California San Diego
La Jolla, California, 92093
Columbia University Medical Center
New York, New York, 10032
Stanford University
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Seattle Children's Hospital
Seattle, Washington, 98105
Italy6 sites
IRCCS Institute of Neorological Sciences of Bologna, Bellaria Hospital
Bologna, 40139
Azienda Ospedaliero Universitaria Policlinico G. Martino
Messina, 98125
Istituto Nazionale Neurologico Carlo Besta
Milan, 20133
Dipartimento Ambientale di Neuroscienze
Pisa, 56126
Ospedale Pediatrico Bambin Gesù
Rome, 00165
Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli
Rome, 00168
Canada2 sites
McMaster University Medical Center
Hamilton, Ontario
Adult Metabolic Diseases Clinic
Vancouver, British Colombia
Germany2 sites
University Hospital of Bonn
Bonn, 53105
Klinikum der Universität München, Friedrich-Baur Institute
Munich, 80336
United Kingdom2 sites
MRC Centre for Neuromuscular Diseases
London
Royal Victoria Infirmary
Newcastle upon Tyne
About this study
Official title: A Phase 3 Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension
This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.
Read the detailed description
Part 11 is a 24-week, randomized, double-blind, parallel-group, placebo-controlled assessment of the efficacy and safety of single daily subcutaneous (SC) doses of 40 mg elamipretide (vs placebo) administered with the elamipretide delivery system as a treatment for subjects with primary mitochondrial myopathy (PMM). Part 2 was to assess the long-term safety and tolerability of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for up to 144 weeks.
- Conditions
- Primary Mitochondrial Myopathy
- Being tested
- elamipretide, placebo comparator, elamipretide open label treatment
- Keywords
- Myopathy, PMD, Primary Mitochondrial Disease, MTP-131, elamipretide
- Medical subject headings
- Muscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elamipretideCombination product
Also called MTP-131
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
placebo comparatorCombination product
Also called Placebo
40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
elamipretide open label treatmentCombination product
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Elamipretide | Experimental | 40 mg (0.5mL) elamipretide subcutaneous (SC) daily Receives: Combination Product: elamipretide |
| Part 1: Placebo | Placebo comparator | Placebo SC daily Receives: Combination Product: placebo comparator |
| Part 2: Elamipretide open label | Experimental | Elamepretide 40 mg (0.5 mL) SC daily Receives: Combination Product: elamipretide open label treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Six-minute Walk Test (6MWT)
Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
Measured: Baseline to 24 weeks
Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Change from Baseline in Total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) by visit. Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
Measured: Baseline to 24 weeks
Secondary outcomes (4)
Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).
Change from baseline in Fatigue During Activities. Fatigue During Activities is the sum of question 2 (tiredness during activities) and question 4 (muscle weakness during activities.) The four response options are: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Raw scores for each subject range from 2-8. A lower score means a better outcome, with less fatigue. A higher score means a worse outcome, with more fatigue.
Measured: Baseline to 24 weeks
Neuro-QoL Fatigue Activities of Daily Living
Change From Baseline in Neuro-QoL Fatigue Activities of Daily Living by Visit. Each individual item score ranges from 1-5. Total raw score for the entire item bank ranges from 19-95. Raw scores will be calibrated using Item Response Theory Model. Lower values represent a better outcome. Individual items will be summed to calculate total scores.
Measured: Baseline to 24 weeks
Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment
The item score rangers from 1 (none) to 4 (severe). Lower values represent a better outcome. The most bothersome score is the average of the identified most bothersome symptom of the Primary Mitochondrial Myopathy Symptom Assessment by each subject.
Measured: Baseline to 24 weeks
Neuro-QoL Fatigue Short Form Score
Change From Baseline in Neuro-QoL Fatigue - Short Form: Total T-Scores by Visit. The Neuro-QoL Fatigue Short Form is comprised of the sum of the first 8 questions of the Neuro-QoL Item Bank v1.0 - Fatigue. Each question is scored as following: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, and 5=Always. The questions include: I felt exhausted, I felt that I had no energy, I felt fatigued, I was too tired to do my household chores, I was too tired to leave the house, I was frustrated by being too tired to do the things I wanted to do, I felt tired, and I had to limit my social activity because I was tired. T-scores are calculated from the short form scoring table provided by the instrument authors (Neuro-QoL User Manual, 2015). T-score distributions rescale raw scores into standardized scores with a mean of 50 and a standard deviation (SD) of 10. Change from baseline: Negative numbers mean less fatigue, better outcome, positive score means more fatigue, worse outcome.
Measured: 24 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2017
- Study start9 Oct 2017
- Primary completion10 Feb 2020
- Study completion10 Feb 2020
Why it stopped: Part1,double blind portion of the trial did not meet the primary end points
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 (2)Papers the sponsor has linked to this study.
- Other trials for Primary 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 218 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
- NCT03323749
- Sponsor's protocol ID
- SPIMM-301
- Acronym
- MMPOWER-3
- Status last verified
- Jan 2022
- First submitted
- 12 Oct 2017
- Last updated
- 24 Jan 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Karaa A, Bertini E, Carelli V, Cohen B, Ennes GM, Falk MJ, Goldstein A, Gorman G, Haas R, Hirano M, Klopstock T, Koenig MK, Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Abbruscato A, Brown DA, Sullivan A, Shiffer JA, Mancuso M; MMPOWER-3 Trial Investigators. Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial. Orphanet J Rare Dis. 2024 Nov 21;19(1):431. doi: 10.1186/s13023-024-03421-5.
PubMed 39574155Karaa A, Bertini E, Carelli V, Cohen BH, Enns GM, Falk MJ, Goldstein A, Gorman GS, Haas R, Hirano M, Klopstock T, Koenig MK, Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Brown DA, Shiffer JA, Mancuso M; MMPOWER-3 Trial Investigators. Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial. Neurology. 2023 Jul 18;101(3):e238-e252. doi: 10.1212/WNL.0000000000207402. Epub 2023 Jun 2.
PubMed 37268435
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension
- ClinicalTrials.gov ID
- NCT03323749
- Acronym / sponsor ID
- MMPOWER-3 / SPIMM-301
- Conditions
- Primary Mitochondrial Myopathy
- Keywords
- Myopathy, PMD, Primary Mitochondrial Disease, MTP-131, elamipretide
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Part1,double blind portion of the trial did not meet the primary end points
- Enrolment
- 218 participants (actual)
- Lead sponsor
- Stealth BioTherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Oct 2017 (actual)
- Primary completion
- 10 Feb 2020 (actual)
- Study completion
- 10 Feb 2020 (actual)
- First posted
- 27 Oct 2017 (actual)
- Results
- Posted 2 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.
Detailed description
Part 11 is a 24-week, randomized, double-blind, parallel-group, placebo-controlled assessment of the efficacy and safety of single daily subcutaneous (SC) doses of 40 mg elamipretide (vs placebo) administered with the elamipretide delivery system as a treatment for subjects with primary mitochondrial myopathy (PMM). Part 2 was to assess the long-term safety and tolerability of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for up to 144 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Part 1: Elamipretide | Experimental | Combination Product: elamipretide40 mg (0.5mL) elamipretide subcutaneous (SC) daily |
| Part 1: Placebo | Placebo comparator | Combination Product: placebo comparatorPlacebo SC daily |
| Part 2: Elamipretide open label | Experimental | Combination Product: elamipretide open label treatmentElamepretide 40 mg (0.5 mL) SC daily |
Who may qualify
- Ages
- 16 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Willing and able to provide a signed informed consent form prior to participation in any trial-related procedures
- Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system
- Subject is ≥ 16 and ≤ 80 years of age
- Diagnosed with PMM in the opinion of the investigator and confirmed by an Adjudication Committee
- Woman of childbearing potential must agree to use a highly effective method of birth control
Key exclusion criteria (26 listed)
- Subject has myopathic signs and or/symptoms due to a neuropathic process or gait problem that would interfere with the 6 minute walk test (6MWT), in the opinion of the Investigator
- Female who are pregnant, planning to become pregnant, or breastfeeding/lactating
- At Screening, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m^2
- Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
- Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, 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 |
|---|---|---|
| elamipretideAlso: MTP-13140 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system | Combination product | Part 1: Elamipretide |
| placebo comparatorAlso: Placebo40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system | Combination product | Part 1: Placebo |
| elamipretide open label treatment40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system | Combination product | Part 2: Elamipretide open label |
Primary outcome
Six-minute Walk Test (6MWT)
Time frame: Baseline to 24 weeks
Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Time frame: Baseline to 24 weeks
Change from Baseline in Total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) by visit. Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
4 secondary outcomes
Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).
Time frame: Baseline to 24 weeks
Neuro-QoL Fatigue Activities of Daily Living
Time frame: Baseline to 24 weeks
Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment
Time frame: Baseline to 24 weeks
Neuro-QoL Fatigue Short Form Score
Time frame: 24 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39574155Karaa A, Bertini E, Carelli V, Cohen B, Ennes GM, Falk MJ, Goldstein A, Gorman G, Haas R, Hirano M, Klopstock T, Koenig MK, Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Abbruscato A, Brown DA, Sullivan A, Shiffer JA, Mancuso M; MMPOWER-3 Trial Investigators. Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial. Orphanet J Rare Dis. 2024 Nov 21;19(1):431. doi: 10.1186/s13023-024-03421-5.
- Derived from this study: PubMed 37268435Karaa A, Bertini E, Carelli V, Cohen BH, Enns GM, Falk MJ, Goldstein A, Gorman GS, Haas R, Hirano M, Klopstock T, Koenig MK, Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Brown DA, Shiffer JA, Mancuso M; MMPOWER-3 Trial Investigators. Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial. Neurology. 2023 Jul 18;101(3):e238-e252. doi: 10.1212/WNL.0000000000207402. Epub 2023 Jun 2.
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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.
27 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing elamipretide and elamipretide open label treatment in people with Primary Mitochondrial Myopathy, run by Stealth BioTherapeutics Inc. at 27 sites in 7 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 5 inclusion and 26 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: stopped early, as of the sponsor's last update on 24 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, Italy, Canada, Germany, United Kingdom and 2 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 2 Apr 2021). 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?
Six-minute Walk Test (6MWT) (time frame: Baseline to 24 weeks). The study lists 2 primary outcomes in all. 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 NCT03323749, which also lists 2 linked publications.
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 24 Jan 2022.