CystinosisPhase 1/2INCANCT05994534
PK and PD Study of NPI-001 and Cysteamine Bitartrate
A Phase 1/2 study testing Cysteamine Bitartrate and N-Acetylcysteine Amide in people with Cystinosis, run by Nacuity Pharmaceuticals, Inc. at 1 site in Australia.
Trial summary
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Cystinosis
- What is being tested?
- Cysteamine Bitartrate and N-Acetylcysteine Amide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteAustralia
- Study dates
- 29 Oct 2023to 26 May 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males or females, any race, ≥ 10 years of age.
- Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.
- Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol.
- 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).
Who cannot join
- Have undergone kidney transplantation.
- Are receiving dialysis treatment.
- History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution.
- Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1.
- Inability to provide blood samples, including difficulty with venous access.
- Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.
Trial locations and contacts
1 site in 1 country.
Australia1 site
Children's Hospital at Westmead
Westmead, New South Wales, 2145
About this study
Official title: A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
Read the detailed description
This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce cystine will be assessed and compared with cysteamine.
- Conditions
- Cystinosis
- Being tested
- Cysteamine Bitartrate, N-Acetylcysteine Amide
- Medical subject headings
- Cystinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cysteamine BitartrateDrug
Also called Cystagon
Single dose, tablets at current therapeutic dose
N-Acetylcysteine AmideDrug
Also called NPI-001
Single dose, oral solution
| Study group | Role | What they receive |
|---|---|---|
| cysteamine | Active comparator | Single dose, tablets in current treatment dose Receives: Drug: Cysteamine Bitartrate |
| NPI-001 | Experimental | Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose. Receives: Drug: N-Acetylcysteine Amide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Concentration of Cystine Levels Over Time
Cystine concentration over 6 hours
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2023
- Study start29 Oct 2023
- Primary completion26 May 2026
- Study completion26 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CystinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nacuity Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hugh McCarthy, PhD, FRACP, Principal investigator, Sydney Children's Health Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05994534
- Sponsor's protocol ID
- C-23-01
- Acronym
- INCA
- Status last verified
- Oct 2026
- First submitted
- 8 Aug 2023
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data will not be made available to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
- ClinicalTrials.gov ID
- NCT05994534
- Acronym / sponsor ID
- INCA / C-23-01
- Conditions
- Cystinosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Nacuity Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Oct 2023 (actual)
- Primary completion
- 26 May 2026 (actual)
- Study completion
- 26 May 2026 (actual)
- First posted
- 16 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
Detailed description
This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce cystine will be assessed and compared with cysteamine.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| cysteamine | Active comparator | Drug: Cysteamine BitartrateSingle dose, tablets in current treatment dose |
| NPI-001 | Experimental | Drug: N-Acetylcysteine AmideSingle dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Males or females, any race, ≥ 10 years of age.
- Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.
- Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol.
- 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).
Key exclusion criteria (6 listed)
- Have undergone kidney transplantation.
- Are receiving dialysis treatment.
- History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution.
- Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1.
- Inability to provide blood samples, including difficulty with venous access.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cysteamine BitartrateAlso: CystagonSingle dose, tablets at current therapeutic dose | Drug | cysteamine |
| N-Acetylcysteine AmideAlso: NPI-001Single dose, oral solution | Drug | NPI-001 |
Primary outcome
Concentration of Cystine Levels Over Time
Time frame: 1 day
Cystine concentration over 6 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Individual participant dataSharing planned: No. Individual participant data will not be made available to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hugh McCarthy, PhD, FRACP principal investigator
Sydney Children's Health Network
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Cysteamine Bitartrate and N-Acetylcysteine Amide in people with Cystinosis, run by Nacuity Pharmaceuticals, Inc. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Concentration of Cystine Levels Over Time (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.