A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 10
Healthy Volunteers: f
View:
• 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).
Locations
Other Locations
Australia
Children's Hospital at Westmead
RECRUITING
Westmead
Contact Information
Primary
Jami Kern, PhD
info@nacuity.com
+1-817-336-3000
Time Frame
Start Date: 2023-10-29
Estimated Completion Date: 2026-01
Participants
Target number of participants: 12
Treatments
Active_comparator: cysteamine
Single dose, tablets in current treatment dose
Experimental: NPI-001
Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.
Related Therapeutic Areas
Sponsors
Leads: Nacuity Pharmaceuticals, Inc.