A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
NCT ID: NCT03323749
Last Updated: 2022-01-24
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
218 participants
INTERVENTIONAL
2017-10-09
2020-02-10
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part 1: Elamipretide
40 mg (0.5mL) elamipretide subcutaneous (SC) daily
elamipretide
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Part 1: Placebo
Placebo SC daily
placebo comparator
40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Part 2: Elamipretide open label
Elamepretide 40 mg (0.5 mL) SC daily
elamipretide open label treatment
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
Interventions
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elamipretide
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
placebo comparator
40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
elamipretide open label treatment
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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.
* ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
1. First degree Atrioventricular bock (AV-block)
2. Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
3. 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.
16 Years
80 Years
ALL
No
Sponsors
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Stealth BioTherapeutics Inc.
INDUSTRY
Responsible Party
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Locations
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University of California San Diego
La Jolla, California, United States
Stanford University
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Columbia University Medical Center
New York, New York, United States
Akron Children's Hospital
Akron, Ohio, United States
Cleveland Clinical Neurological Institute
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, United States
University of Texas Health Science Center
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Adult Metabolic Diseases Clinic
Vancouver, British Colombia, Canada
McMaster University Medical Center
Hamilton, Ontario, Canada
Copenhagen Neuromuscular Center
Copenhagen, , Denmark
University Hospital of Bonn
Bonn, , Germany
Klinikum der Universität München, Friedrich-Baur Institute
Munich, , Germany
Institute of Genomic Medicine and Rare Disorders
Budapest, , Hungary
IRCCS Institute of Neorological Sciences of Bologna, Bellaria Hospital
Bologna, , Italy
Azienda Ospedaliero Universitaria Policlinico G. Martino
Messina, , Italy
Istituto Nazionale Neurologico Carlo Besta
Milan, , Italy
Dipartimento Ambientale di Neuroscienze
Pisa, , Italy
Ospedale Pediatrico Bambin Gesù
Rome, , Italy
Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli
Rome, , Italy
MRC Centre for Neuromuscular Diseases
London, , United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, , United Kingdom
Countries
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References
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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.
Karaa 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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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SPIMM-301
Identifier Type: -
Identifier Source: org_study_id
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