Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
12 participants
INTERVENTIONAL
2023-10-29
2026-01-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
CROSSOVER
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
cysteamine
Single dose, tablets in current treatment dose
Cysteamine Bitartrate
Single dose, tablets at current therapeutic dose
NPI-001
Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.
N-Acetylcysteine Amide
Single dose, oral solution
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Cysteamine Bitartrate
Single dose, tablets at current therapeutic dose
N-Acetylcysteine Amide
Single dose, oral solution
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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.
10 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Nacuity Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Hugh McCarthy, PhD, FRACP
Role: PRINCIPAL_INVESTIGATOR
Sydney Children's Health Network
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hospital at Westmead
Westmead, New South Wales, Australia
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
C-23-01
Identifier Type: -
Identifier Source: org_study_id