PK and PD Study of NPI-001 and Cysteamine Bitartrate
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
Study details:
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 cystine will be assessed and compared with cysteamine.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
- Males or females, any race, ≥ 10 years of age.
- Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.
- Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol.
- 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
- Have undergone kidney transplantation.
- Are receiving dialysis treatment.
- History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution.
- Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1.
- Inability to provide blood samples, including difficulty with venous access.
- Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.
Eligibility
Age eligible for study : NA
Healthy volunteers accepted : No
Gender eligible for study: ALL
Things to know
Study dates
Study start:
Primary completion:
Study completion finish:
Study type
TREATMENT
Phase
- PHASE1PHASE2
Study ID
NCT05994534
Intervention or treatment
DRUG: Cysteamine Bitartrate
DRUG: N-Acetylcysteine Amide
Conditions
- Cystinosis
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Research sites nearby
Closest Location:
Children's Hospital at Westmead
Select from list below to view details:
Children's Hospital at Westmead
Westmead, New South Wales, Australia
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Who is running the clinical trial?
Nacuity Pharmaceuticals, Inc.
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?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
ACTIVE_COMPARATOR: cysteamine
| Drug: Cysteamine Bitartrate |
EXPERIMENTAL: NPI-001
| Drug: N-Acetylcysteine Amide |
What is the study measuring?
Primary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Concentration of Cystine Levels Over Time | Cystine concentration over 6 hours | 1 day |
Secondary Outcome
References
Clinical Trials Gov: PK and PD Study of NPI-001 and Cysteamine BitartrateOther trials to consider
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