DFT383 in Pediatric Participants With Nephropathic Cystinosis

Description

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) treated with Standard of care (SoC). The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.

Conditions

Nephropathic Cystinosis

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Study Overview

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Study Details

Study overview

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) treated with Standard of care (SoC). The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.

An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis

DFT383 in Pediatric Participants With Nephropathic Cystinosis

Condition
Nephropathic Cystinosis
Intervention / Treatment

-

Contacts and Locations

Atlanta

Emory University School of Medicine-Winship Cancer Institute (recuiting Cohort 0), Atlanta, Georgia, United States, 30322

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • 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
  • 1. Indomethacin within 2 weeks prior to Screening

Ages Eligible for Study

2 Years to 5 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novartis Pharmaceuticals,

Study Record Dates

2030-08-26