Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05994534




Registration number
NCT05994534
Ethics application status
Date submitted
8/08/2023
Date registered
16/08/2023
Date last updated
8/02/2024

Titles & IDs
Public title
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Scientific title
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Secondary ID [1] 0 0
C-23-01
Universal Trial Number (UTN)
Trial acronym
INCA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystinosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Cysteamine Bitartrate
Treatment: Drugs - N-Acetylcysteine Amide

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.


Treatment: Drugs: Cysteamine Bitartrate
Single dose, tablets at current therapeutic dose

Treatment: Drugs: N-Acetylcysteine Amide
Single dose, oral solution
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Concentration of Cystine Levels Over Time
Timepoint [1] 0 0
1 day

Eligibility
Key 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).
Minimum age
10 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/10/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
21/06/2024
Actual
Sample size
Target
12
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Nacuity Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis
patients compared with cysteamine.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05994534
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Hugh McCarthy, PhD, FRACP
Address 0 0
Sydney Children's Health Network
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Jami Kern, PhD
Address 0 0
Country 0 0
Phone 0 0
+1-817-336-3000
Fax 0 0
Email 0 0
info@nacuity.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05994534