A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension



Status:Recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:16 - 80
Updated:4/3/2019
Start Date:October 27, 2017
End Date:December 2020

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A Phase 3 Randomized, Double-Blind, Parallel-Group, 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.


PART 1:

Inclusion Criteria:

- 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

Exclusion Criteria:

- Subject has myopathic signs and or/symptoms due to a neuropathic process or gait
problem that would interfere with the 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.

- ECG evidence of acute ischemia, atrial fibrillation, or active conduction system
abnormalities with the exception of any of the following:

1. First degree 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.
We found this trial at
14
sites
Houston, Texas 77030
Phone: 713-500-5372
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1 Perkins Square
Akron, Ohio 44308
(330) 543-1000
Phone: 330-543-4743
Akron Children's Hospital From humble beginnings as a day nursery in 1890, Akron Children
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Phone: 617-724-7726
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Phone: 215-590-1934
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Phone: 412-692-3476
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Atlanta, Georgia 30318
Phone: 404-883-2724
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Phone: 720-777-4690
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Cleveland, Ohio 44195
Phone: 216-444-0173
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Houston, Texas
Phone: 832-822-1630
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9500 Gilman Dr
La Jolla, California 92093
(858) 534-2230
Phone: 858-246-2288
The University of California, San Diego UC San Diego is an academic powerhouse and economic...
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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Palo Alto, California 94304
Phone: 650-736-8166
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Phone: 206-987-0058
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Vancouver, British Colombia
Phone: 604-875-4111
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