DFT383 for Cystinosis

(CYStem Trial)

Recruiting at 1 trial location

Overseen By Novartis Pharmaceuticals

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Novartis Pharmaceuticals

Must be taking: Cysteamine

Must not be taking: Indomethacin

Disqualifiers: Kidney transplant, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 32 months

30 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must have been on oral cysteamine therapy for at least 6 months. If you are taking indomethacin, you need to stop it at least 2 weeks before screening for Cohort 1.

Is DFT383 (cysteamine) safe for humans?

Cysteamine, used in treating cystinosis, has been shown to be generally safe in humans, though it can cause side effects like vomiting and upper respiratory infections. It has been used for many years to help manage cystinosis, improving growth and kidney function in patients. ^{1 2 3 4 5}

What is the purpose of 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. 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.

Eligibility Criteria

This trial is for kids aged 2 to almost 6 with nephropathic cystinosis, a rare kidney condition. They must have normal kidney function, be up-to-date on vaccines, weigh enough for their height, and have been on oral cysteamine therapy for at least half a year.

Inclusion Criteria

My parent or guardian has given written consent for me.

I have been diagnosed with nephropathic cystinosis.

I have been diagnosed with renal Fanconi syndrome.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants in Cohort 1 receive DFT383 treatment, while Cohort 0 continues with standard of care

Up to 32 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term monitoring

Participants are monitored for adverse events and other outcomes

Up to 32 months

Treatment Details

Interventions

• DFT383

Trial Overview DFT383 gene therapy is being tested in children with nephropathic cystinosis. The study has two groups: one receiving DFT383 (Cohort 1) and another getting standard care (Cohort 0). It's an open-label phase I/II study that isn't randomized.

Participant Groups

Group I: Cohort 1 (DFT383) Experimental Treatment 1 Intervention

Treatment with DFT383

Group II: Cohort 0 (SoC) Active Control 1 Intervention

No study treatment, will continue with standard of care (cysteamine).

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

References

1. United States pubmed.ncbi.nlm.nih.gov

Cysteamine bitartrate delayed-release capsules control leukocyte cystine levels and promote statural growth and kidney health in an open-label study of treatment-naïve patients [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

Early oral cysteamine therapy for nephropathic cystinosis. [2018]

3. France pubmed.ncbi.nlm.nih.gov

[Infantile cystinosis. A new Tunisian case]. [2016]

4. United States pubmed.ncbi.nlm.nih.gov

An international cohort study spanning five decades assessed outcomes of nephropathic cystinosis. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Pharmacological treatment of nephropathic cystinosis with cysteamine. [2019]