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New cystinosis drug shows promise in early trial

NCT ID NCT05994534

Disease control Sponsor: Nacuity Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times

Summary

This study tests a new oral solution called NPI-001 in people with cystinosis, a rare disease that causes cystine buildup in cells. Researchers want to see if NPI-001 is safe and works better than the current drug cysteamine. About 12 participants aged 10 and older will stop their usual medicine for 2 days to take part.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2023

Expected to finish

Aug 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

10 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Males or females, any race, ≥ 10 years of age. 2. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days. 3. Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol. 4. Able to comprehend and willing to sign an informed consent /assent form and to abide by the study restrictions (travel as necessary, clinical phase 1 unit or similar for up to 3 days). Exclusion Criteria: 1. Have undergone kidney transplantation. 2. Are receiving dialysis treatment. 3. History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution. 4. Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1. 5. Inability to provide blood samples, including difficulty with venous access. 6. Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.

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Conditions

The condition(s) this trial relates to.

cystinosis nephropathic cystinosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystinosis

Contacts and locations

Locations

  • Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.