Evaluating NPI-001 for treating cystinosis
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
This study is testing a new oral treatment called NPI-001 to see if it can lower cystine levels in people aged 10 and older with cystinosis better than the current standard treatment.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 10 Years and up |
| Sex | All |
| Sponsor | Nacuity Pharmaceuticals, Inc. Industry-sponsored |
| Locations | 1 site (Westmead, New South Wales) |
| Trial ID | NCT05994534 on ClinicalTrials.gov |
What this trial studies
This study investigates the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in patients with cystinosis aged 10 years and older. Participants will be assessed for the ability of NPI-001 to reduce cystine levels compared to the standard treatment, cysteamine. The study aims to provide insights into the effectiveness and safety profile of NPI-001, which may offer a new therapeutic option for managing cystinosis.
Who should consider this trial
Good fit: Ideal candidates are males and females aged 10 years or older diagnosed with nephropathic cystinosis who can temporarily stop cysteamine therapy.
Not a fit: Patients who have undergone kidney transplantation or are currently receiving dialysis treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a more effective treatment option for patients with cystinosis.
How similar studies have performed: Other studies have explored treatments for cystinosis, but the specific approach of using NPI-001 is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Westmead, New South Wales
- Children's Hospital at Westmead — Westmead, New South Wales, Australia (Recruiting)
Study contacts
- Principal investigator: Hugh McCarthy, PhD, FRACP — Sydney Children's Health Network
- Study coordinator: Jami Kern, PhD
- Email: info@nacuity.com
- Phone: +1-817-336-3000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.