Health TrialsbyHealthPanel

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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • DFT383

    Genetic

    DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.

Treatment groups

30 Participants
are divided into 2 treatment groups
Group A: Cohort 1 (DFT383)Experimental treatment 1 intervention
Group B: Cohort 0 (SoC)No intervention 0 interventions

Trial outcomes

Primary outcomes

1

Core Phase - Incidence of adverse events (Cohort 1)

Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)

Time frame
Up to 32 months
2

Core Phase - Number of participants with hematological reconstitution (Cohort 1)

Hematological reconstitution by Day 42 post-DFT383 infusion

Time frame
42 days post DFT infusion
3

Core Phase - Proportion of participants with reversal of renal Fanconi syndrome (RFS)

Proportion of participants with reversal of renal Fanconi syndrome (RFS)

Time frame
Up to 32 months

Secondary outcomes

1

Core Phase - Number of participants independent from cysteamine

Independence from oral and ophthalmic cysteamine

Time frame
up to 24 months
2

Core Phase - Health-related quality of life (HRQOL)

Health-related quality of life (HRQOL) as measured by QUALIFY (cystinosis-specific PRO)- Currently under development

Time frame
Up to 32 months
3

Core Phase - Time from infusion to reversal of RFS (Cohort 1)

Time from infusion to reversal of renal Fanconi syndrome (RFS)

Time frame
Up to 24 months
4

Core Phase - Time from screening to reversal of RFS (Cohort 0)

Time from screening to reversal of renal Fanconi syndrome (RFS)

Time frame
Up to 24 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Novartis Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
Apply now