Phase 2
N=12
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
Leber's Hereditary Optic Neuropathy
Bottom Line
View on ClinicalTrials.gov: NCT02693119 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Oct 2021
Primary outcome: Primary: Double Masked Period: Incidence of Ocular TEAEs — 3; 8; 1; 3 eyes
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- elamipretide (MTP-131) 1% topical ophthalmic solution (Drug); Vehicle topical ophthalmic solution (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Stealth BioTherapeutics Inc.
- Primary completion
- Sep 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Double Masked Period: Incidence of Ocular TEAEs |
3; 8; 1; 3; 0; 3 | — |
| PRIMARY Open Label Extension: Incidence of Ocular TEAEs |
1; 1; 1; 1; 1; 1 | — |
| PRIMARY Double Masked Period: Severity of Ocular TEAEs |
2; 0; 0; 1; 1; 0 | — |
| PRIMARY OLE: Severity of Ocular TEAEs |
5; 0; 0; 6; 0; 0 | — |
| PRIMARY Double Masked Period: Best Corrected Visual Acuity (BCVA) |
-3.1; -0.5; 0.3; 1.0; 2.0; 0.5 | — |
| PRIMARY Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale |
8.5; 6.00; 10.6; 5.4; 6.1; 6.1 | — |
| SECONDARY Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude |
0.0; 1.3; -0.2; 0.5; -3.5; -3.2 | — |
| SECONDARY Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude |
3.2; 4.0; -7.1; 1.8; 8.0; 4.9 | — |
| SECONDARY Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude |
-9.1; -4.5; -4.6; -3.4; 3.5; 14.6 | — |
| SECONDARY Open Label Extension Period: PhNR-ERG B-wave Amplitude |
-15.4; -7.4; -6.0; -13.1; -28.7; -19.9 | — |
| SECONDARY Double Masked Period: PhNR Amplitude |
-6.4; 0.3; -4.6; -3.3; -11.6; -0.7 | — |
| SECONDARY Open Label Extension Period: PhNR Amplitude |
-7.1; 0.5; -2.5; -2.2; 3.3; 1.8 | — |
| SECONDARY Double Masked Period: PhNR/B-wave Amplitude Ratio |
-0.102; -0.033; -0.036; -0.033; -0.113; 0.019 | — |
| SECONDARY Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio |
-0.097; -0.002; -0.021; -0.041; 0.012; -0.003 | — |
| SECONDARY Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude |
0.18; -0.21; -0.03; 1.08; 0.08; 0.00 | — |
| SECONDARY Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude |
-0.31; -0.70; 0.25; -0.09; -0.30; -0.13 | — |
| SECONDARY Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline |
-2.72; -4.80; 0.08; -0.08; 15.16; 15.38 | — |
| SECONDARY Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) |
-8.36; -7.92; -3.50; -10.90; -32.02; -21.64 | — |
| SECONDARY Double Masked: VFQ-39 Composite |
6.1; 5.7; 7.8 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Composite Score |
5.6; 13.9 | — |
| SECONDARY Open Label Extension Period: VFQ-39 General Health Score |
-6.8; 6.7 | — |
| SECONDARY Double Masked: VFQ-39 General Vision Score |
8.1; 1.3; 3.3 | — |
| SECONDARY Open Label Extension Period: VFQ-39 General Vision Score |
9.3; 6.7 | — |
| SECONDARY Double Masked: VFQ-39 Ocular Pain Score |
1.6; 3.1; 12.5 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Ocular Pain Score |
-16.1; -4.2 | — |
| SECONDARY Double Masked: VFQ-39 Near Activities Score |
3.6; 9.2; 11.1 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Near Activities |
8.5; 16.7 | — |
| SECONDARY Double Masked Period: Mean Retinal Nerve Fiber (RNFL) Layer Thickness |
-3.8; -2.6 | — |
| SECONDARY Open Label Extension Period: Retinal Nerve Fiber Layer Thickness |
-1.0; 1.4 | — |
| SECONDARY Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III |
1.1; 2.1; 0.5; 1.6; 0.4; 0.9 | — |
| SECONDARY Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline |
3.3; 4.0; 5.1; 4.4; 3.1; 5.5 | — |
| SECONDARY Double Masked Period: Color Discrimination - Number of Plates |
0.0; 0.1; 0.0; 0.1; 0.1; 0.3 | — |
| SECONDARY Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit |
1.3; 1.4; 1.9; 1.6; 2.8; 2.0 | — |
| SECONDARY Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit |
0.075; -0.019; 0.038; -0.028; 0.075; 0.047 | — |
| SECONDARY Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit |
0.225; 0.084; 0.300; 0.122; 0.281; 0.118 | — |
| SECONDARY Double Masked Change From Baseline in Retinal Ganglion Cell Layer Thickness |
-1.5; -3.6 | — |
| SECONDARY Open Label Extension Period: Change From Baseline in Retinal Ganglion Cell Layer Thickness |
-1.0; 3.4 | — |
| SECONDARY Double Masked: VFQ-39 Distance Activities Score |
9.4; 0.0; 54.2 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Distance Activities Score |
12.5; 13.9 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Social Functioning Score |
8.3; 5.6 | — |
| SECONDARY Double Masked: VFQ-39 Social Functioning Score. |
6.3; 0.0; 16.7 | — |
| SECONDARY Double Masked: VFQ-39 Mental Health Score |
12.0; 19.7; 13.3 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Mental Health Score |
16.4; 29.2 | — |
| SECONDARY Double Masked: VFQ-39 Role Difficulties Score |
4.7; 14.1; 10.4 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Role Difficulties Score |
8.0; 37.5 | — |
| SECONDARY Open Label Extension Period:VFQ-39 Dependency Score |
16.1; 27.1 | — |
| SECONDARY Double Masked: VFQ-39 Dependency Score |
9.4; -2.6; -8.3 | — |
| SECONDARY Double Masked: VFQ-39 Color Vision Score |
6.3; 6.3; 0.0 | — |
| SECONDARY Open Label Extension Period: VFQ-39 Color Vision Score |
-7.1; 8.3 | — |
| SECONDARY Double Masked: VFQ-39 Peripheral Score |
0.0; 6.3; 66.7 | — |
| SECONDARY Open Label Extension Period: Visual Function Questionnaire (VFQ-39) Peripheral Score |
0.0; 0.0 | — |
| SECONDARY Double Masked Period: Change in Intraocular Pressure (IOP) |
-0.3; -0.2; 0.0; -0.4; -0.4; -0.5 | — |
| SECONDARY Open Label Extension Period: Intraocular Pressure (IOP) |
-0.4; 0.8; 1.4; 0.4; -0.9; -0.2 | — |
| SECONDARY OLE: Slit Lamp Examination (SLE)-Vehicle Eye |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY OLE: Slit Lamp Examination (SLE)-Elamipretide Eye |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye |
1; 0; 1; 0; 1; 1 | — |
| SECONDARY Double Masked: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes |
0; 0 | — |
| SECONDARY Open Label Extension Period: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes |
0; 0 | — |
| SECONDARY Double Masked: VFQ-39 General Health Score |
-3.8; -0.6; -5.8 | — |
Summary
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic solution in this patient population. At the conclusion of 52 weeks of treatment, subjects will be offered the opportunity to enter an Open Label Extension for up to 48 additional weeks of treatment.
Eligibility Criteria
Inclusion Criteria
- Adults ≥18 and ≤ 50 years old at the time of loss of vision in the second eye.
- Able to provide informed consent and willing to comply with all study visits and examinations
- Diagnosis of LHON based on clinical and ophthalmic functional/anatomic test findings, and satisfactory documentation of the mitochondrial DNA mutation m.11778G>A
- Loss of vision in both eyes of ≥1 year and ≤10 years at the time of the Screening Visit and current clinically stable visual function (as assessed by the Investigator)
- Able to self-administer eye drops as demonstrated at screening or having a care provider who can do so
- Documentation of having satisfactorily completed at least two previous Humphrey automated visual field tests prior to screening.
- Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form
Exclusion Criteria
- Any other ocular pathology requiring treatment with prescription topical ophthalmic drops (e.g., glaucoma, dry eye)
- Cup to disc ratio of > 0.8 in either eye
- Media opacity, suboptimal pupillary dilatation, or refractive error that interferes with adequate retinal imaging
- Known to be immunocompromised or receiving systemic immunosuppression
- Any disease or medical condition that in the opinion of the Investigator would prevent the subject from participating in the study or might confound study results
- Participation in other investigational drug or device clinical trials within 30 days prior to enrollment, or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion
- Women who are pregnant or lactating
Other protocol-defined inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT02693119). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.