Nephropathic Cystinosis study: DFT383
Official title DFT383 in Pediatric Participants With Nephropathic Cystinosis
ClinicalTrials.gov ID: NCT06910813
What this study is testing
What is DFT383?
an investigational autologous hematopoietic stem cell gene therapy being studied as a potential treatment for nephropathic cystinosis
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether DFT383 is safe, tolerable, and works in children 2 to 5 with nephropathic cystinosis.
- Who it's for
- You may be able to join if you are 2 to 5 years old and have nephropathic cystinosis.
- What's involved
- You would receive a one‑time DFT383 gene therapy infusion and have regular check‑ups. Some children will not get the therapy and will only have check‑ups.
- Time commitment
- The core part lasts several months and may continue for years in a long‑term extension, with visits at the start and later check‑ins.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 5
You may be able to join if
- 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
- Historic clinical diagnosis of nephropathic cystinosis
You likely can't join if
- for Cohort 1 and 0
- 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
- Major surgery within 90 days Additional Key exclusion criteria for Cohort 1 - The following exclusion criterion applies to Cohort 1 only as it is...
See the full eligibility criteria
- 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
- Historic clinical diagnosis of nephropathic cystinosis
- Laboratory evidence of of renal fanconi syndrome (RFS)
- Relatively preserved kidney function (eGFR ≥ 60mL/min/1.73m2)
- Received all age-appropriate vaccinations Key
- for Cohort 1 and 0
- 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
- Major surgery within 90 days Additional Key exclusion criteria for Cohort 1 - The following exclusion criterion applies to Cohort 1 only as it is related to DFT383 treatment: 1\.
The study team makes the final eligibility decision.
Where it is enrolling
- Phoenix, Arizona, United States
- San Diego, California, United States
- Stanford, California, United States
- Atlanta, Georgia, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 5 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Phoenix, Arizona, United States; San Diego, California, United States; Stanford, California, United States; Atlanta, Georgia, United States; Philadelphia, Pennsylvania, United States; Houston, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.