PK and PD Study of NPI-001 and Cysteamine Bitartrate (INCA)

February 7, 2024 updated by: Nacuity Pharmaceuticals, Inc.

A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients

Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 white blood cell (WBC) cystine will be assessed and compared with cysteamine.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: cysteamine
Single dose, tablets in current treatment dose
Drug: Cysteamine Bitartrate
Single dose, tablets at current therapeutic dose
Other Names:
  • Cystagon
Experimental: NPI-001
Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.
Drug: N-Acetylcysteine Amide
Single dose, oral solution
Other Names:
  • NPI-001

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Concentration of Cystine Levels Over Time
Time Frame: 1 day
White Blood Cell cystine concentration over 6 hours
1 day

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nacuity Pharmaceuticals, Inc.

Investigators

  • Principal Investigator: Hugh McCarthy, PhD, FRACP, Sydney Children's Health Network

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 29, 2023

Primary Completion (Estimated)

May 21, 2024

Study Completion (Estimated)

June 21, 2024

Study Registration Dates

First Submitted

August 8, 2023

First Submitted That Met QC Criteria

August 8, 2023

First Posted (Actual)

August 16, 2023

Study Record Updates

Last Update Posted (Actual)

February 8, 2024

Last Update Submitted That Met QC Criteria

February 7, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C-23-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Individual participant data will not be made available to other researchers.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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