PK and PD Study of NPI-001 and Cysteamine Bitartrate

Cystinosis Clinical Trial

— INCA  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05994534
• Other study ID #: C-23-01
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 29, 2023
• Est. completion date: June 21, 2024

Study information

• Verified date: February 2024
• Source: Nacuity Pharmaceuticals, Inc.
• Contact: Jami Kern, PhD
• Phone: +1-817-336-3000
• Email: info[@]nacuity.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: June 21, 2024
• Est. primary completion date: May 21, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystinosis

Intervention

Drug:
• Cysteamine Bitartrate
Single dose, tablets at current therapeutic dose
• N-Acetylcysteine Amide
Single dose, oral solution

Locations

Country	Name	City	State
Australia	Children's Hospital at Westmead	Westmead	New South Wales

Sponsors (1)

Lead Sponsor	Collaborator
Nacuity Pharmaceuticals, Inc.

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Concentration of Cystine Levels Over Time	White Blood Cell cystine concentration over 6 hours	1 day