#1 Clinical Trials Search in Europe

Cpk850

A groundbreaking clinical trial is underway to investigate the safety and effectiveness of CPK850, a novel gene therapy for patients with retinitis pigmentosa caused by mutations in the RLBP1 gene. This open-label, first-in-human study aims to evaluate the potential of subretinal administration of CPK850 in improving visual function and quality of life for individuals affected by this rare genetic eye disorder.

Table of Contents

What is CPK850?

CPK850 is an innovative gene therapy being developed to treat a specific type of retinitis pigmentosa, an inherited eye disorder that causes vision loss. This therapy is currently being studied in clinical trials to evaluate its safety and effectiveness.[1]

CPK850 is classified as an advanced therapy medicinal product, specifically a gene therapy. It uses a replication-deficient recombinant adeno-associated viral (AAV) vector to deliver genetic material to the eye. This vector carries a gene called hRLBP1, which is important for normal vision.[1]

Target Condition: Retinitis Pigmentosa due to RLBP1 Gene Mutations

CPK850 is designed to treat retinitis pigmentosa caused by mutations in the RLBP1 gene. This condition is also known by other names, including:[1]

  • Bothnia dystrophy
  • Newfoundland rod-cone dystrophy

These conditions are characterized by progressive vision loss due to the degeneration of light-sensitive cells in the retina (the back part of the eye). Patients with this condition often experience difficulty seeing in low light (night blindness) and a gradual loss of peripheral vision.

How CPK850 Works

CPK850 is administered through a subretinal injection, which means it’s injected directly under the retina of the eye. The therapy aims to deliver a functioning copy of the RLBP1 gene to the cells in the retina.[1]

By providing a correct version of the gene, CPK850 may help the eye produce the necessary proteins for normal visual function. This could potentially slow down or stop the progression of vision loss in patients with this form of retinitis pigmentosa.

Clinical Trial Details

The ongoing clinical trial for CPK850 is an open-label, first-in-human study. This means it’s the first time this therapy is being tested in humans, and both the researchers and participants know which treatment is being given.[1]

Key aspects of the trial include:

  • It’s a Phase 1-2 trial, designed to assess both safety and initial signs of effectiveness.
  • The study uses a single ascending dose approach, meaning different groups of patients may receive increasing doses of the therapy to find the optimal amount.
  • Patients will receive a single dose of CPK850 in one eye, allowing comparisons between the treated and untreated eyes.

Eligibility Criteria

To participate in the CPK850 trial, patients must meet certain criteria, including:[1]

  • Age between 18 and 70 years
  • Confirmed diagnosis of retinitis pigmentosa with mutations in the RLBP1 gene
  • Specific levels of visual acuity (a measure of how well you can see)
  • Presence of certain retinal structures as seen on imaging tests

There are also several factors that may exclude a person from participating, such as certain eye conditions, active infections, or other health issues that could interfere with the treatment or its evaluation.

Safety and Efficacy Measures

The trial will closely monitor participants for any side effects or adverse events related to the treatment. Additionally, researchers will assess various measures of visual function to determine if the therapy is working. These include:[1]

  • Dark adaptation: How well the eye adjusts to low light conditions
  • Visual acuity: The clarity or sharpness of vision
  • Visual field: The entire area that can be seen without moving the eyes
  • Contrast sensitivity: The ability to distinguish between finer and finer increments of light versus dark
  • Reading speed
  • Mobility tests: Assessing how well patients can navigate in different environments

Potential Benefits

While it’s important to note that CPK850 is still in the experimental stage, the therapy aims to potentially:[1]

  • Improve rod and cone function in the retina, which are essential for vision in different light conditions
  • Enhance overall visual function, including visual acuity and field of vision
  • Improve patients’ ability to perform daily activities and navigate their environment
  • Ultimately, slow down or stop the progression of vision loss in patients with this form of retinitis pigmentosa

As with any experimental treatment, the actual benefits and risks of CPK850 will become clearer as the clinical trial progresses and more data is collected.

Aspect Details
Study Type Open-label, first-in-human, single ascending dose study
Condition Retinitis pigmentosa caused by RLBP1 gene mutations
Intervention CPK850 gene therapy (subretinal administration)
Primary Objectives Evaluate safety and efficacy in rod system recovery
Secondary Objectives Assess cone system recovery, visual function changes, and impact on daily activities
Key Eligibility Criteria Adults 18-70 years, specific visual acuity range, confirmed RLBP1 mutations
Primary Endpoints Adverse events, responders in dark adaptation
Secondary Endpoints Changes in visual field, contrast sensitivity, reading speed, mobility, and quality of life measures

FAQ

  • What is CPK850 and how does it work? CPK850 is a gene therapy that uses a modified virus to deliver a healthy copy of the RLBP1 gene to the retina. It aims to correct the genetic defect causing retinitis pigmentosa in patients with RLBP1 mutations.
  • Who is eligible for this clinical trial? The trial is open to adults aged 18-70 with retinitis pigmentosa caused by RLBP1 gene mutations. Participants must meet specific visual acuity criteria and have visible photoreceptor and retinal pigment epithelium layers on OCT scans.
  • How is CPK850 administered? CPK850 is administered through a subretinal injection, which means it is delivered directly beneath the retina during a surgical procedure.
  • What are the main objectives of the trial? The primary objectives are to evaluate the safety of CPK850 and its efficacy in improving rod system recovery in treated eyes. Secondary objectives include assessing cone system recovery, changes in visual function, and impact on daily activities.
  • How long will participants be monitored after treatment? The trial will monitor participants for at least one year after treatment to assess long-term safety and efficacy of CPK850.

Ongoing Clinical Trials on Cpk850

  • Study on the Safety and Effects of CPK850 Gene Therapy for Patients with Retinitis Pigmentosa Due to RLBP1 Gene Mutations

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Sweden

Glossary

  • Retinitis pigmentosa: A group of rare genetic disorders that involve a breakdown and loss of cells in the retina, which can lead to vision loss.
  • RLBP1 gene: A gene that provides instructions for making a protein important for normal vision. Mutations in this gene can cause certain forms of retinitis pigmentosa.
  • Gene therapy: A technique that uses genes to treat or prevent disease. In this case, it involves delivering a healthy copy of the RLBP1 gene to correct the genetic defect.
  • Subretinal administration: A method of delivering medication directly beneath the retina, typically through a surgical procedure.
  • Optical Coherence Tomography (OCT): A non-invasive imaging test that uses light waves to take cross-section pictures of the retina.
  • Dark adaptation: The process by which the eye adjusts to low light conditions, primarily involving the rod cells in the retina.
  • Visual acuity: The clarity or sharpness of vision, often measured using an eye chart.
  • Vitrectomy: A surgical procedure that involves removing the gel-like substance (vitreous) from the eye.
  • Adeno-associated viral (AAV) vector: A modified virus used to deliver genetic material into cells, commonly used in gene therapy.
  • Microperimetry: A technique used to measure visual sensitivity at specific locations in the retina.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-cpk850-gene-therapy-for-patients-with-retinitis-pigmentosa-due-to-rlbp1-gene-mutations/