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Elamipretide Trihydrochloride

Elamipretide Trihydrochloride is currently being studied in clinical trials as a potential treatment for dry age-related macular degeneration (dry AMD). This article explores the ongoing research, focusing on a Phase 3 clinical trial called ReNEW, which aims to evaluate the efficacy, safety, and pharmacokinetics of subcutaneous injections of Elamipretide in patients with dry AMD.

Table of Contents

What is Elamipretide?

Elamipretide, also known by its scientific name elamipretide trihydrochloride or its product code MTP-131, is an investigational drug being developed by Stealth BioTherapeutics Inc.[1] It is classified as an aromatic-cationic tetrapeptide, which is a type of small protein molecule. Elamipretide represents a new class of drugs with a unique mechanism of action, making it a promising candidate for treating various conditions, particularly those affecting the eyes.[1]

Target Condition: Dry Age-Related Macular Degeneration

The primary focus of the current clinical trial is to evaluate elamipretide’s effectiveness in treating dry age-related macular degeneration (dry AMD).[1] Dry AMD is a common eye condition that affects older adults, causing a gradual loss of central vision. It occurs when the central part of the retina, called the macula, deteriorates over time. This can significantly impact a person’s ability to read, recognize faces, and perform daily activities.

Clinical Trial Details

The clinical trial for elamipretide, known as “ReNEW,” is a Phase 3 study designed to evaluate the drug’s efficacy, safety, and pharmacokinetics in patients with dry AMD.[1] Here are some key details about the trial:

  • It is a randomized, double-masked, placebo-controlled study, which means participants are randomly assigned to either the elamipretide group or a placebo group, and neither the patients nor the researchers know who is receiving the actual drug.
  • The trial will last for approximately 96 weeks (about 2 years).
  • Participants will be randomized in a 2:1 ratio, meaning two-thirds of the participants will receive elamipretide, while one-third will receive a placebo.

How Elamipretide is Administered

Elamipretide is administered as a subcutaneous injection, which means it is injected just under the skin.[1] The drug comes in the form of a solution for injection. Patients or their caregivers will be trained to administer the drug daily and will be required to keep a dosing diary to record the time and location of each injection.

Eligibility Criteria

To participate in the trial, patients must meet certain criteria, including:[1]

  • Being 55 years of age or older
  • Having at least one eye with dry AMD and photoreceptor loss, as determined by specific imaging techniques
  • Women of childbearing potential and men with partners of childbearing potential must agree to use effective birth control methods
  • Being able to administer the study drug and comply with all trial procedures

Certain conditions may exclude a person from participating, such as having concurrent eye diseases or other medical conditions that might interfere with the study results.

Potential Benefits

The main goals of the study are to determine if elamipretide can:[1]

  • Slow down the rate of photoreceptor loss in the macula, which could potentially slow the progression of dry AMD
  • Improve low-light visual acuity, which is often affected in patients with dry AMD

These potential benefits could significantly impact patients’ quality of life by preserving their vision for longer periods.

Safety Considerations

As with any clinical trial, the safety and tolerability of elamipretide are being closely monitored.[1] The study includes a 4-week safety follow-up period after the 96-week treatment period to assess any potential long-term effects of the drug. It’s important to note that as an investigational drug, not all potential side effects of elamipretide may be known at this time.

Other Potential Applications of Elamipretide

While this trial focuses on dry AMD, elamipretide is being studied for other conditions as well. Previous and ongoing studies have explored its potential in treating:[1]

  • Heart conditions, including heart failure and reperfusion injury following heart attacks
  • Primary mitochondrial diseases, which are genetic disorders affecting cellular energy production
  • Primary mitochondrial myopathy, a type of muscle disease

These diverse applications suggest that elamipretide may have broad potential in treating conditions related to cellular energy dysfunction.

Aspect Details
Drug Name Elamipretide Trihydrochloride
Trial Name ReNEW (Phase 3 Clinical Trial)
Condition Studied Dry Age-Related Macular Degeneration (Dry AMD)
Administration Method Daily subcutaneous injections
Trial Duration 96 weeks
Primary Objective Evaluate efficacy in slowing photoreceptor loss
Secondary Objectives Assess safety, tolerability, and pharmacokinetics
Key Inclusion Criteria Adults ≥55 years with dry AMD and photoreceptor loss
Primary Endpoint Rate of change in macular area of photoreceptor loss at Week 48
Key Secondary Endpoints Changes in photoreceptor loss at Weeks 72 and 96, improvements in low luminance visual acuity

FAQ

  • What is Elamipretide Trihydrochloride? Elamipretide Trihydrochloride is an investigational drug being studied for the treatment of dry age-related macular degeneration (dry AMD). It is administered as a subcutaneous injection and represents a first-in-class mechanism of action for retinal vascular diseases.
  • What is the ReNEW clinical trial? ReNEW is a Phase 3, randomized, double-masked, placebo-controlled clinical trial designed to evaluate the efficacy, safety, and pharmacokinetics of subcutaneous injections of Elamipretide in patients with dry AMD. The trial lasts for approximately 96 weeks and compares Elamipretide to a placebo.
  • Who can participate in the ReNEW clinical trial? The trial is open to adults 55 years of age or older with dry AMD and photoreceptor loss, as determined by the presence of extrafoveal geographic atrophy. Participants must meet specific inclusion criteria and not have any exclusion criteria, such as concurrent eye diseases or participation in other clinical trials.
  • How is Elamipretide administered in the trial? Elamipretide is administered as a daily subcutaneous injection. Participants or their caregivers are trained to administer the drug and record the location and time of administration in a dosing diary.
  • What are the main goals of the ReNEW clinical trial? The primary goal is to evaluate the efficacy of Elamipretide in slowing the progression of photoreceptor loss in dry AMD. Secondary objectives include assessing the safety and tolerability of the drug, as well as studying its pharmacokinetic profile and effects on visual acuity under low luminance conditions.

Ongoing Clinical Trials on Elamipretide Trihydrochloride

  • Study on Elamipretide for Patients with Dry Age-Related Macular Degeneration (Dry AMD)

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Germany Hungary Italy Spain

Glossary

  • Dry Age-Related Macular Degeneration (Dry AMD): A chronic eye condition that affects the macula, the central part of the retina responsible for sharp, detailed vision. In dry AMD, the macula gradually thins and deteriorates, leading to vision loss.
  • Elamipretide Trihydrochloride: An investigational drug being studied for the treatment of dry AMD, administered as a subcutaneous injection.
  • Subcutaneous Injection: A method of administering medication by injecting it into the layer of tissue between the skin and muscle.
  • Photoreceptor Loss: The deterioration or death of light-sensitive cells (rods and cones) in the retina, which can lead to vision impairment.
  • Geographic Atrophy (GA): An advanced form of dry AMD characterized by well-defined areas of retinal tissue loss.
  • Spectral Domain-Optical Coherence Tomography (SD-OCT): An imaging technique used to capture detailed cross-sectional images of the retina.
  • Ellipsoid Zone (EZ): A layer in the retina visible on OCT scans, which is associated with photoreceptor health.
  • Low Luminance Best-Corrected Visual Acuity (LL BCVA): A measure of visual acuity under low light conditions, used to assess visual function in AMD patients.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Double-Masked: A study design where both the participants and the researchers are unaware of which treatment (active drug or placebo) is being administered.

References

  1. http://clinicaltrials.eu/trial/study-on-elamipretide-for-patients-with-dry-age-related-macular-degeneration-dry-amd/