Retinitis Pigmentosa study: NPI-001
Official title A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
ClinicalTrials.gov ID: NCT07710196
What this study is testing
What is NPI-001?
An investigational oral tablet being studied for retinitis pigmentosa in adults with Usher syndrome.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study wants to find out if NPI-001 can slow the loss of photoreceptor cells and to learn about any medical problems caused by the drug.
- Who it's for
- Adults who have both Usher syndrome and retinitis pigmentosa.
- What's involved
- Participants will take NPI-001 or a placebo twice a day for 24 months and go to the clinic 9 times for check‑ups and tests.
- Time commitment
- The trial lasts 24 months and requires 9 clinic visits.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Able to comprehend and willing to sign an informed consent form (OCF) and adhere to the study protocol.
- Diagnosed with RP associated with Usher syndrome.
- EZ width ≥ 500 microns, which includes the fovea in each eye at Visit 1.
- All edges of the EZ area in both eyes can be visualized at Visit 1 (Screening).
- Have at least 20 detectable points on the MAIA grid in at least one eye at the Screening and Baseline visits (same eye for both visits).
You likely can't join if
- Concurrent retinal pathologies that result in vision loss or inability to fixate.
- Intraocular surgery within the last two months or capsulotomy within the last month.
- Current or history of uveitis, Coat's disease, diabetic retinopathy, glaucoma, herpes simplex of the eye, or currently has a cataract that prevents...
- Likely to require cataract surgery within the next 12 months.
- Unstable fixation during microperimetry in either eye at either Screening or Baseline visits.
- Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment.
See the full eligibility criteria
- Able to comprehend and willing to sign an informed consent form (OCF) and adhere to the study protocol.
- Diagnosed with RP associated with Usher syndrome.
- EZ width ≥ 500 microns, which includes the fovea in each eye at Visit 1.
- All edges of the EZ area in both eyes can be visualized at Visit 1 (Screening).
- Have at least 20 detectable points on the MAIA grid in at least one eye at the Screening and Baseline visits (same eye for both visits).
- On stable dose of medications associated with other conditions for at least one month.
- Willing to use contraception during study (for anyone of childbearing potential).
- Concurrent retinal pathologies that result in vision loss or inability to fixate.
- Intraocular surgery within the last two months or capsulotomy within the last month.
- Current or history of uveitis, Coat's disease, diabetic retinopathy, glaucoma, herpes simplex of the eye, or currently has a cataract that prevents visualization of the posterior pole.
- Likely to require cataract surgery within the next 12 months.
- Unstable fixation during microperimetry in either eye at either Screening or Baseline visits.
- Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment.
- Use of N-acetylcysteine containing products in the previous 30 days prior to the baseline visit or unwilling to refrain from such supplements for the duration of the study.
- Chronic liver or kidney disease, cystic fibrosis, severe asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause, or other blood dyscrasia.
- Suspected liver dysfunction determined by having alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin values \> 1.5 X the upper limit of normal (ULN) at screening.
- Platelet or hemoglobin values \< 100 at screening.
- History of known sensitivity to N-acetylcysteine or similar thiol compounds or any ingredients of NPI-001.
- History of hypersensitivity to any medication or food resulting in systemic symptoms.
- History of cancer (other than non-melanoma skin cancer) diagnosed or requiring treatment within the past 2 years.
- Pregnant women or women planning to become pregnant in the next 25 months or men with partners planning to become pregnant in the next 25 months.
- Lactating women who are breast-feeding.
- Potential participant lives in the same household as a current participant in this study.
- people who are unwilling or unable to refrain from donation of blood from 30 days prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening until 56 days following the last...
- Inability to provide blood samples, including difficulty with venous access.
- Any reason, in the opinion of the Principal Investigator, the participant should not participate.
The study team makes the final eligibility decision.
Where it is enrolling
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Dallas, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.