Actively Recruiting
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Led by Nacuity Pharmaceuticals, Inc. · Updated on 2026-04-13
12
Participants Needed
1
Research Sites
143 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
CONDITIONS
Official Title
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females, any race, 10 years of age 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 as detailed in the protocol
- Able to understand and willing to sign an informed consent or assent form
- Willing to follow study restrictions including travel and staying in a clinical phase 1 unit for up to 3 days
You will not qualify if you...
- Have had a kidney transplant
- Receiving dialysis treatment
- History of significant allergy to N-acetylcysteine or any ingredient in NPI-001 oral solution
- Participated in another clinical study involving an investigational drug within 30 days before Day 1
- Unable to provide blood samples or have difficulty with venous access
- Considered unsuitable for the study by the Investigator or Sponsor
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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