Actively Recruiting
Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Patients with Cystinosis
Led by Nacuity Pharmaceuticals, Inc. · Updated on 2026-04-13
12
Participants Needed
1
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in patients with cystinosis who are aged 10 years and older. This study compares the ability of NPI-001 to reduce cystine levels with the current treatment using cysteamine. The research aims to understand how these treatments behave in the body and their effects on cystine reduction in this patient group. Participants will receive a single dose of either the current cysteamine treatment in tablet form at their usual dose or a single dose of NPI-001 (N-acetylcysteine amide) oral solution at a molar equivalent dose. The study design is non-randomized and open-label, meaning participants and researchers know which treatment is given. This phase 1/2 study focuses on comparing these two treatments directly. During the study, participants will be monitored for cystine levels over one day to assess treatment effects. They must be able to stop cysteamine therapy for two days before dosing. Safety and pharmacokinetic data will be collected, including blood samples. Participants need to stay at a clinical phase 1 unit or similar setting for up to 3 days for assessments and monitoring. The study is sponsored by Nacuity Pharmaceuticals, Inc. and is expected to continue until August 2026.
CONDITIONS
Brief Title
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females aged 10 years or older
- Diagnosis of nephropathic cystinosis
- Able to stop cysteamine therapy for 2 days
- Females must be nonpregnant and nonlactating
- Females of childbearing potential and males must agree to use contraception
- Able to understand and willingly sign informed consent or assent
- Willing to follow study restrictions including travel and stay at clinical unit for up to 3 days
You will not qualify if you...
- Have undergone kidney transplantation
- Currently receiving dialysis treatment
- History of significant allergy to NAC or any ingredient in NPI-001 oral solution
- Participation in another investigational drug study within 30 days before Day 1
- Unable to provide blood samples or have difficulty with venous access
- Any other condition that the investigator or sponsor considers unsuitable for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Participants receive a single dose of either NPI-001 oral solution or cysteamine bitartrate tablets. The study compares the pharmacokinetics and pharmacodynamics of these treatments by measuring cystine levels over one day.
Participants stay at the clinical unit for up to 3 days for dosing and monitoring
Trial Site Locations
Total: 1 location
1
Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145
Actively Recruiting
Research Team
J
Jami Kern, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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