DFT383 for Cystinosis

(CYStem Trial)

Recruiting at 9 trial locations

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called DFT383 to determine its safety and effectiveness for young children with nephropathic cystinosis, a rare condition that causes harmful cystine buildup in the body. The study consists of two parts: one group will receive DFT383, while another will continue their usual treatment for comparison. Children aged 2 to 5 years who have been on oral cysteamine therapy for at least 6 months and have nephropathic cystinosis may be suitable candidates. As a Phase 1 trial, this research aims to understand how the new gene therapy works in people, offering participants a chance to be among the first to receive this innovative treatment.

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 there any evidence suggesting that DFT383 is likely to be safe for humans?

Research has shown that DFT383 is a new type of gene therapy being tested for safety in young children with nephropathic cystinosis, a rare condition. Since testing is still in the early stages, there is limited information about its safety in humans. The study is in its first and second phases, focusing primarily on assessing safety for the children involved. These early phases typically ensure the treatment does not cause excessive side effects.

Gene therapies like DFT383 are new and may function differently from other treatments, so researchers carefully monitor for any issues. The study aims to identify side effects and evaluate how well children tolerate the treatment. Although specific data is not yet available, its inclusion in a clinical trial suggests scientists believe it could be safe. More detailed safety information will likely emerge as the study progresses. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cystinosis?

DFT383 is unique because it offers a new approach to treating cystinosis compared to the current standard of care, which typically involves cysteamine. Researchers are excited about DFT383 because it may work through a different mechanism, potentially improving how cystine is reduced in cells. This could mean better management of cystinosis symptoms and possibly fewer side effects, giving patients a new hope for improved quality of life.

What evidence suggests that DFT383 might be an effective treatment for cystinosis?

Research has shown that DFT383 is a gene therapy under investigation for treating nephropathic cystinosis, a rare disease affecting kidney function. Early studies suggest that DFT383 targets the genetic issues causing the disease, potentially reducing the buildup of cystine in cells. This buildup can damage the kidneys and other organs over time. In this trial, participants will either receive DFT383 or continue with the standard of care, cysteamine. Although information on DFT383's effectiveness in people remains limited, the therapy aims to address the genetic root of the problem, potentially offering longer-lasting effects than current treatments. As more results from ongoing trials become available, they will provide clearer insights into its potential benefits. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. novartis.com novartis.com/clinicaltrials/study/nct06910813

DFT383 in Pediatric Participants With Nephropathic ... 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.

2. clinicaltrials.ucsd.edu clinicaltrials.ucsd.edu/trial/NCT06910813

UCSD Cystinosis Trial → DFT383 in Pediatric Participants ... 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.

3. cystinosisresearch.org cystinosisresearch.org/novartis-cystem-stem-cell-and-gene-therapy-trial-is-moving-forward-and-recruiting/

Novartis CYStem Stem Cell and Gene Therapy Trial Is ... This study is an open-label, multi-center, phase I/II study to assess the safety, tolerability, and efficacy of DFT383 in participants aged 2 to 5 years with ...

4. trial.medpath.com trial.medpath.com/clinical-trial/35806699ca6018d9/nct06910813-dft383-safety-efficacy-pediatric-nephropathic-cystinosis

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.

5. en.wikipedia.org en.wikipedia.org/wiki/DFT383

DFT383 DFT383 is an investigational gene therapy under development by Novartis for the treatment of cystinosis, a rare autosomal recessive lysosomal storage disorder.

6. cystinosisresearch.org cystinosisresearch.org/current-clinical-trials/

Current Clinical Trials The purpose of the study is to investigate if DFT383 is safe and effective in children aged 2-5 years who have nephropathic cystinosis. DFT383 is a cellular ...

7. withpower.com withpower.com/trial/phase-2-cystinosis-2-2025-2302c

DFT383 for Cystinosis (CYStem Trial) 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 ...