DFT383 in Pediatric Participants With Nephropathic Cystinosis
ConditionNephropathic Cystinosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age2-5
SponsorNovartis Pharmaceuticals
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 long-term Extension Phase. 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). Participants in Cohort 0 will not receive study treatment and will only participate in the Core Phase of the study. The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.
Eligibility criteria
Qualifiers
Informed consent in writing from parent(s) or legal guardian(s) must be provided
2 to 5 years of age (including 5 years and 364 days old) at Screening
Weight-for-stature is ≥ the third percentile, and is ≥ 10 kg
Oral cysteamine therapy for at least 6 months
Disqualifiers
A history of kidney transplantation
A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy
History of malignancy
A severe or uncontrolled medical disorder
Trial design
Treatments tested in this trial
- DFT383
Treatment groups
30 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov