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PK and PD Study of NPI-001 and Cysteamine Bitartrate

PHASE1PHASE2RECRUITING

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

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

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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.

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Eligibility

Age eligible for study : 10 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2023-10-29

Primary completion: 2024-05-21

Study completion finish: 2024-06-21

Study type

TREATMENT

Phase

PHASE1

PHASE2

Trial ID

NCT05994534

Intervention or treatment

DRUG: Cysteamine Bitartrate

DRUG: N-Acetylcysteine Amide

Conditions

• Cystinosis

Condition: Cystinosis
DRUG: Cysteamine Bitartrate and other drugs
Westmead, New South Wales, Australia
Sponsor: Nacuity Pharmaceuticals, Inc.

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Find a site

Closest Location:

Children's Hospital at Westmead

Research sites nearby

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Children's Hospital at Westmead
Westmead, New South Wales, Australia

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 Single dose, tablets in current treatment dose	DRUG: Cysteamine Bitartrate Single dose, tablets at current therapeutic dose
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

What is the study measuring?

Primary outcome

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

Secondary outcome

Frequently Asked Questions

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References

Clinical Trials Gov: PK and PD Study of NPI-001 and Cysteamine Bitartrate