Stanford, CANCT06910813Now EnrollingIRB Ready

Nephropathic Cystinosis Clinical Trial in Stanford, CA

Access cutting-edge nephropathic cystinosis treatment through this clinical trial at a research site in Stanford. Study-provided care at no cost to qualified participants.

Sponsored by Novartis Pharmaceuticals

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Expert Care in Stanford

Access nephropathic cystinosis specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related nephropathic cystinosis treatment provided free

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Check if you qualify for this nephropathic cystinosis clinical trial in Stanford, CA

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Why Participate?

  • No-Cost Study Care

  • Local to Stanford

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Stanford site if eligible
  4. 4Begin participation

About This Nephropathic Cystinosis Study in Stanford

An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis. The purpose of this clinical study is to assess safety, tolerability, and efficacy of DFT383 in participants aged 2 to ≤ 5 years with nephropathic cystinosis. DFT383 is a cellular gene therapy. This study includes an active arm (Cohort 1) of participants treated with study treatment DFT383 and a concurrent reference arm (Cohort 0) treat

Sponsor: Novartis Pharmaceuticals

Who Can Participate

Inclusion Criteria

Participants eligible for inclusion in this study must meet all the following criteria: 1. Informed consent in writing from parent(s) or legal guardian(s) must be provided 2. 2 to 5 years of age (including 5 years and 364 days old) at Screening 3. Weight-for-stature is ≥ the third percentile is ≥ 10 kg 4. Oral cysteamine therapy for at least 6 months 5. Historic clinical diagnosis of nephropathic cystinosis 6. Laboratory evidence of of renal fanconi syndrome (RFS) 7. Preserved kidney function (eGFR ≥ 90mL/min/1.73m2) 8. Received all age-appropriate vaccinations Key

Exclusion Criteria

for Cohort 1 and 0 1. A history of kidney transplantation 2. A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy 3. History of malignancy 4. A severe or uncontrolled medical disorder 5. Major surgery within 90 days Additional Key exclusion criteria for Cohort 1 - The following exclusion criteria only apply to Cohort 1 only as they ar

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Stanford?

Yes, this clinical trial (NCT06910813) has an active research site in Stanford, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Nephropathic Cystinosis Treatment Options in Stanford, CA

If you're searching for nephropathic cystinosis treatment options in Stanford, CA, this clinical trial (NCT06910813) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Stanford research site is actively enrolling participants for this clinical trial. You'll receive care from experienced nephropathic cystinosis specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all nephropathic cystinosis clinical trials near you to find additional studies recruiting in your area.

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