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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05162768




Registration number
NCT05162768
Ethics application status
Date submitted
8/12/2021
Date registered
17/12/2021
Date last updated
12/12/2023

Titles & IDs
Public title
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
Scientific title
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 Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) NuPower
Secondary ID [1] 0 0
SPIMD-301
Universal Trial Number (UTN)
Trial acronym
NuPower
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mitochondrial Myopathies 0 0
Mitochondrial Pathology 0 0
Mitochondrial DNA Mutation 0 0
Mitochondrial Diseases 0 0
Mitochondrial DNA Deletion 0 0
Mitochondrial DNA Depletion 0 0
Mitochondrial Metabolism Defect 0 0
Mitochondrial Complex I Deficiency 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Elamipretide
Treatment: Drugs - Placebo

Experimental: Elamipretide - 0.75 mL of 80mg/mL solution of elamipretide for a single daily SC dose of 60mg elamipretide

Placebo comparator: Placebo - 0.75 mL of 80mg/mL solution of matching placebo for a single daily SC dose of 60mg


Treatment: Drugs: Elamipretide
60 mg of elamipretide administered as once daily 0.75 mL subcutaneous injections for 48 weeks

Treatment: Drugs: Placebo
Placebo administered as once daily 0.75 mL subcutaneous injections for 48 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Six-minute walk test (6MWT)
Timepoint [1] 0 0
Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)
Secondary outcome [1] 0 0
5 times sit-to-stand test (5XSST)
Timepoint [1] 0 0
Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)
Secondary outcome [2] 0 0
Triple Timed up-and-go test (3TUG)
Timepoint [2] 0 0
Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)
Secondary outcome [3] 0 0
Patient Global Impression of Severity (PGI-S) Scale
Timepoint [3] 0 0
Baseline, Weeks 12, 24, 36, 48

Eligibility
Key inclusion criteria
A subject must meet all of the following inclusion criteria at the Screening and Baseline Visit (unless otherwise specified) to be eligible for inclusion in the SPIMD-301 trial:

1. Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures.
2. Agrees and is able to adhere to the trial requirements for the length of the trial, including administration of assigned treatment.
3. Is =18 years and = 70 years of age at the time of screening.
4. Diagnosed with nPMD with a predominant clinical manifestation of myopathy, which must include progressive external ophthalmoplegia (PEO) and exercise intolerance and/or skeletal muscle weakness, with genetic confirmation of either:

1. Nuclear DNA mutation of the mitochondrial replisome (replisome-related mutations), which include the following genes:

* POLG 1/2
* TWINKLE (C10ORF2)
* TYMP
* DGUOK
* TK2
* RRM2B
* RNASEH1
* SSBP
* MGME1
* DNA2
* ANT1 (SLC25A4)
* SUCLG1
* SUCLA2
* MPV17 or
2. Other pathogenic mutations specific to nuclear DNA.
5. Women of childbearing potential must agree to use one of the following methods of birth control from the date they sign the ICF until 28 days after the last dose of IMP:

1. Abstinence, when it is in line with the preferred and usual lifestyle of the subject. Subject agrees to use a highly effective method of contraception should they become sexually active.
2. Relationships with male partners who have been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days prior to the Screening Visit).
3. Barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or injectable) or an intrauterine device or system.

Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit).
6. Male subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception from the date they sign the ICF until 28 days after the last dose of IMP.
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Is unable to perform the 6MWT, 3TUG, or 5XSST functional tests. The use of a gait assist device is allowed; however, use should remain consistent for the entire duration of the trial.
2. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating.
3. Walks < 150 meters or > 450 meters during the 6MWT (Screening Visit only).
4. The estimated glomerular filtration rate (eGFR) is < 30 mL/min/1.73 m2, using the Modification of Diet in Renal Disease (MDRD) Study equation (Screening Visit only).
5. Has undergone an in-patient hospitalization within 30 days prior to screening or has a planned hospitalization or a surgical procedure during the trial, unless, in the opinion of the Investigator, it is concluded that it will not impact the outcome measurements of the trial.
6. Has clinically significant respiratory disease and/or cardiac disease that would interfere with trial assessments, in the opinion of the Investigator.
7. Has had any prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening.
8. Has history of or current severe neurologic impairment, severe epilepsy, severe ataxia, or severe neuropathy that may interfere with their ability to complete all trial requirements, in the opinion of the Investigator.
9. Active malignancy or any other cancer from which the subject has been disease-free for < 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to screening.
10. Has had a solid organ transplant.
11. Has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection.
12. Has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x106/L at the Screening Visit.
13. 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 prior to current trial; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial (e.g., exercise therapy trial).
14. Has received elamipretide (MTP-131) within the past one year of the Screening Visit.
15. Has a history of active substance abuse during the year prior, in the opinion of the Investigator.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/04/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2024
Actual
Sample size
Target
Accrual to date
Final
102
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal North Shore Hospital Neurology - Sydney
Recruitment hospital [2] 0 0
Calvary Health Care Bethlehem - Parkdale
Recruitment postcode(s) [1] 0 0
2065 - Sydney
Recruitment postcode(s) [2] 0 0
3162 - Parkdale
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Germany
State/province [9] 0 0
Bavaria
Country [10] 0 0
Germany
State/province [10] 0 0
Dresden
Country [11] 0 0
Germany
State/province [11] 0 0
Tübingen
Country [12] 0 0
Hungary
State/province [12] 0 0
Budapest
Country [13] 0 0
Hungary
State/province [13] 0 0
Pecs
Country [14] 0 0
Italy
State/province [14] 0 0
Brescia
Country [15] 0 0
Italy
State/province [15] 0 0
Lazio
Country [16] 0 0
Italy
State/province [16] 0 0
Bologna
Country [17] 0 0
Italy
State/province [17] 0 0
Messina
Country [18] 0 0
Italy
State/province [18] 0 0
Milano
Country [19] 0 0
Italy
State/province [19] 0 0
Pisa
Country [20] 0 0
Netherlands
State/province [20] 0 0
Nijmegen
Country [21] 0 0
New Zealand
State/province [21] 0 0
Auckland
Country [22] 0 0
Norway
State/province [22] 0 0
Bergen
Country [23] 0 0
Spain
State/province [23] 0 0
Barcelona
Country [24] 0 0
Spain
State/province [24] 0 0
Madrid
Country [25] 0 0
Spain
State/province [25] 0 0
Valencia
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Cambridge
Country [27] 0 0
United Kingdom
State/province [27] 0 0
London
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Newcastle

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Stealth BioTherapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).
Trial website
https://clinicaltrials.gov/study/NCT05162768
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05162768