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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
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Scientific title
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
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Secondary ID [1]
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C-23-01
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Universal Trial Number (UTN)
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Trial acronym
INCA
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystinosis
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Condition category
Condition code
Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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0
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Metabolic disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Concentration of Cystine Levels Over Time
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Assessment method [1]
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White Blood Cell cystine concentration over 6 hours
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Timepoint [1]
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1 day
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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).
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Minimum age
10
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Crossover
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Other design features
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Phase
Phase 1/Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
29/10/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
21/06/2024
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Actual
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Sample size
Target
12
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Children's Hospital at Westmead - Westmead
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Recruitment postcode(s) [1]
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2145 - Westmead
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Nacuity Pharmaceuticals, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis
patients compared with cysteamine.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05994534
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Hugh McCarthy, PhD, FRACP
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Address
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Sydney Children's Health Network
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Jami Kern, PhD
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Address
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Country
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Phone
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+1-817-336-3000
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05994534
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