PK and PD Study of NPI-001 and Cysteamine Bitartrate

Launched by NACUITY PHARMACEUTICALS, INC. · Aug 8, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new oral solution called NPI-001 as a treatment for cystinosis, a condition that affects the kidneys. Researchers want to learn about how safe this medication is and how it works in the body compared to the standard treatment, which is cysteamine. The trial is currently looking for participants who are at least 10 years old and have been diagnosed with nephropathic cystinosis. To join the study, participants must be able to stop their current cysteamine treatment for two days and must agree to use contraception if they are of childbearing age.

If you or someone you know is eligible and decides to participate, you'll need to visit a clinical research unit for up to three days. During this time, you'll receive the study medication and undergo tests to monitor its effects. The trial is open to all genders, and the researchers want to make sure that everyone understands the study and agrees to participate. It's an important opportunity to help advance treatment options for cystinosis!

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Males or females, any race, ≥ 10 years of age.
• • 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:
• • 1. Have undergone kidney transplantation.
• • 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.