DFT383 Treatment for Nephropathic Cystinosis in Young Kids
DFT383 in Pediatric Participants With Nephropathic Cystinosis
Recruiting
Phase 1
Phase 2
Interventional Study
Nephropathic Cystinosis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 2 – 5
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 30 (estimated)
- Sponsor
- Novartis Pharmaceuticals · Industry
Who this trial is looking for
This trial is looking for children aged 2 to 5 years who have nephropathic cystinosis. Participants will receive a treatment called DFT383 to see if it's safe and effective.
Are You a Good Fit for This Trial?
You may be able to join if
- I am between 2 to 5 years old.
- I weigh at least 10 kg.
- I have been taking cysteamine for at least 6 months.
- I have been diagnosed with nephropathic cystinosis.
- I have laboratory evidence of renal fanconi syndrome.
- I have relatively preserved kidney function.
- I have received all age-appropriate vaccinations.
- I have written consent from my parent or legal guardian.
You may not be able to join if
- I have had a kidney transplant.
- I have had bone marrow or stem cell treatment before.
- I have a history of cancer.
- I have a severe or uncontrolled medical condition.
- I have had major surgery in the past 90 days.
Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase.
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. The study consists of a Core Phase and a …
Trial Locations
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Eligibility Criteria
Key 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 ≥ th…
Contacts
Novartis Pharmaceuticals
CONTACT