Status:
RECRUITING
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Lead Sponsor:
Nacuity Pharmaceuticals, Inc.
Conditions:
Cystinosis
Eligibility:
All Genders
10+ years
Phase:
PHASE1
PHASE2
Brief Summary
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 a...
Eligibility Criteria
Inclusion
- 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).
Exclusion
- 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.
Key Trial Info
Start Date :
October 29 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2026
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT05994534
Start Date
October 29 2023
End Date
January 1 2026
Last Update
December 17 2025
Active Locations (1)
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1
Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145