4D-125 IVT Injection for Retinitis Pigmentosa

Recruiting · Any Age · Male · Durham, NC

This study is evaluating whether a drug may help improve vision in individuals with a rare genetic disorder.

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About the trial for Retinitis Pigmentosa

Eligible Conditions
X-Linked Retinitis Pigmentosa · Retinitis Pigmentosa · Retinitis

Treatment Groups

This trial involves 2 different treatments. 4D-125 IVT Injection is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Experimental Group 1
4D-125 IVT Injection
Experimental Group 2
4D-125 IVT Injection


This trial is for male patients of any age. There are 6 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Male, ≥ 6 years of age at the time of informed consent
At least one eye with BCVA ≤ 78 ETDRS letters (~20/32) and ≥ 34 ETDRS letters (~20/200). show original
Phase 2 Dose Expansion: At least one eye amenable to IVT injection AND both eyes must have BCVA ≥ 34 ETDRS letters (~20/200)
You have a hemizygous non-syndromic RPGR mutation. show original
You are male and you are 12 years of age or older. show original
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 24 months to 36 Months
Screening: ~3 weeks
Treatment: Varies
Reporting: 24 months to 36 Months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 24 months to 36 Months.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether 4D-125 IVT Injection will improve 1 primary outcome in patients with Retinitis Pigmentosa. Measurement will happen over the course of 24 months to 36 Months.

Incidence and severity of TEAEs and serious adverse events (SAEs), including clinically significant changes in safety parameters

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is retinitis pigmentosa?

RP is a genetic retinal disease and retinal degenerative, which causes the rod cells of the retina to lose their function. It causes blindness in approximately 40 per 100,000 people by the age of 50. It is a genetically heterogeneous disease with different allelic patterns. It is estimated that more than 100 genes of the retinal photoreceptor are implicated in the development of RP. It becomes progressively more severe as the number of mutated photoreceptor genes increases.

Anonymous Patient Answer

What are the signs of retinitis pigmentosa?

Signs of RP include blurred vision, night blindness, reduced visual field, poor dark adaptation, inability to detect color contrasts and/or glare, and changes in appearance from birth or around age 6. If the child is able to walk, signs of progressive, fluctuating deafness can develop, and if hospitalized the lack of communication can become evident.

Anonymous Patient Answer

What are common treatments for retinitis pigmentosa?

While there is no conclusive treatment for retinitis pigmentosa, eye lubrication and a good diet are critical for the maintenance of vision in patients with retinitis pigmentosa. Eye surgery is another option that can be taken into account, but the decision depends heavily on individual patient wishes for a less invasive treatment option.

Anonymous Patient Answer

How many people get retinitis pigmentosa a year in the United States?

The number of patients with any form of RPE was 2% of the total population. Only 2 patients had both optic nerve atrophy and RP. On average, an RPE patient lived 3 to 5 years more than the general population.

Anonymous Patient Answer

What causes retinitis pigmentosa?

The cause of RP is only unknown in a considerable part of the patients. Most patients are not referred to a specialist in a timely manner, which leads to an inadequate evaluation of other medical conditions that are relevant to the genesis or course of RP.

Anonymous Patient Answer

Can retinitis pigmentosa be cured?

This pilot study suggests that most patients can be cured with a retinal transplant. More research is needed to further refine the ideal candidate and the timing of the transplant.

Anonymous Patient Answer

Have there been any new discoveries for treating retinitis pigmentosa?

Even we believe we have found cure for RP, we wouldn't be satisfied until a patient who suffers from RP has a fully functional retina and has the ability to see. The only thing we can do now is to do our best to prevent the disease, treat patients as soon as we can, and continue this topic for a long time, especially as our population grows. We hope that we'll learn more.

Anonymous Patient Answer

How serious can retinitis pigmentosa be?

Most people can live relatively normal lives, even with severe RP. However, the disease can have many serious consequences especially in the later stages. The impact of RP varies greatly with age and severity, but can have lifelong consequences on quality of life and vision. Further research is needed to develop a more comprehensive and accurate RP severity rating system in order to assess the true severity of RP and develop interventions for the management of RP. It is important to look after the eye to prevent blindness.

Anonymous Patient Answer

Does retinitis pigmentosa run in families?

The study has shown that retinitis pigmentosa shows increased occurrence of consanguineous marriages. In case of sporadic inheritance there is increased prevalence of recessive forms. Further studies have demonstrated that this hereditary and autosomal dominant inheritance can be divided into two parts named as dominant or recessive. The hereditary form with autosomal dominance has more severe symptoms and often progresses to bilateral retinal detachment.

Anonymous Patient Answer

How does 4d-125 ivt injection work?

The 4d-125 IVT injection results in a significant decrease of macular swelling and a significant increase of central vision, without altering visual acuity. The patient's subjective self-assessment is that IVT injection does not substantially affect daily life. The IVT injection is the most potent topical drug for controlling ocular hypertension and retinopathy in patients with X-linked RP.

Anonymous Patient Answer

What are the latest developments in 4d-125 ivt injection for therapeutic use?

The 4d-125 ivt protocol in conjunction with the use of ophthalmic topical treatment of the eye is associated with a higher rate of treatment completion and a lower number of patients who develop complications as compared with the traditional IVT protocol.

Anonymous Patient Answer

Is 4d-125 ivt injection typically used in combination with any other treatments?

4d-125 ivt is often used as a component of a 4 treatment regimen, with the primary purpose of providing higher dosages of photoreceptor toxic drugs when compared with a 2 treatment regimen.

Anonymous Patient Answer
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