Nephropathic CystinosisPhase 1/2CYStemNCT06910813
DFT383 in Pediatric Participants With Nephropathic Cystinosis
A Phase 1/2 study testing DFT383 in people with Nephropathic Cystinosis, run by Novartis Pharmaceuticals at 6 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 5 years
- Conditions the study is for
- Nephropathic Cystinosis
- What is being tested?
- DFT383
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 6 sitesUnited States
- Study dates
- 2 Jun 2025to 28 May 2044
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants eligible for inclusion in this study must meet all the following 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
Show all 9Show fewer
- Laboratory evidence of of renal fanconi syndrome (RFS)
- Relatively preserved kidney function (eGFR ≥ 60mL/min/1.73m2)
- Received all age-appropriate vaccinations
Who cannot join
- 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. Indomethacin within 2 weeks prior to Screening
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)
Atlanta, Georgia, 30322
Laurence (Larry) Greenbaum, 404-712-6374, lgreen6@emory.edu
Laurence (Larry) Greenbaum
- Recruiting
Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)
Houston, Texas, 77030
Ewa Elenberg, 832-824-3800, Email_elenberg@bcm.edu
Ewa Elenberg
- Recruiting
Children's Hospital of Philadelphia (Recruiting Cohort 0)
Philadelphia, Pennsylvania, 19104
Melissa Cadnapaphornchai, 215-590-2449
Melissa Cadnapaphornchai
- Recruiting
Phoenix Children's Hospital (Recruiting Cohort 0 and 1)
Phoenix, Arizona, 85016
Erica Sieber, 602-933-0171, esieber@phoenixchildrens.com
Shanna White
- Recruiting
University of California at San Diego - Rady Children's Hospital (Recruiting Cohort 0 and 1)
San Diego, California, 92123
Mieko Pretlow, 858-966-1700 ext. Ext 226010, mpretlow@rchsd.org
Nadine Benador nbenador@health.ucsd.edu
- Recruiting
Stanford University - Stanford Children's Health (Recruiting Cohort 0 and 1)
Stanford, California, 94305
Research Nurse, (650) 725-9032, scgt_clinical_trials_office@lists.stanford.edu
Alice Bertaina
About this study
Official title: An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase
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.
Read the detailed description
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 nephropathic cystinosis, followed by a long-term extension phase.
The study includes two Treatment Groups (Cohort 1 and Cohort 0) and consists of a Core Phase and a long-term Extension Phase.
Participants in Cohort 1 will receive DFT383 and participate in both the Core and Extension Phase. Participants in Cohort 0 will not receive study treatment and will participate in the Core Phase only.
The two cohorts will be run in parallel. Investigational sites may participate in one or both cohorts.
Cohort 1 Approximately 15 participants will receive treatment with DFT383 in 3 (sub) cohorts (1A, 1B and 1C) dosed in a staggered approach. The total study duration for a participant in Cohort 1 will be up to 32 months in the core phase and up to 13 years for the long-term extension phase.
Cohort 0 Approximately 15 participants meeting similar inclusion/exclusion criteria and receiving SoC will be enrolled. The Schedule of Activities will be reduced for this Cohort. This cohort 0 is not a direct control but will provide essential context for interpreting the results observed in the participants receiving DFT383. The total study duration for a participant in Cohort 0 will be up to 24 months.
- Conditions
- Nephropathic Cystinosis
- Being tested
- DFT383
- Keywords
- Cystinosis, Nephropathic cystinosis, Lysosomal storage disorder, CTNS gene, DFT383, Cellular gene therapy, Cysteamine, Renal Fanconi syndrome
- Medical subject headings
- Cystinosis, Lysosomal Storage Diseases, Fanconi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DFT383Genetic
DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (DFT383) | Experimental | Treatment with DFT383 Receives: Genetic: DFT383 |
| Cohort 0 (SoC) | No intervention | No study treatment, will continue with standard of care (cysteamine). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Core Phase - Incidence of adverse events (Cohort 1)
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Measured: Up to 32 months
Core Phase - Number of participants with hematological reconstitution (Cohort 1)
Hematological reconstitution by Day 42 post-DFT383 infusion
Measured: 42 days post DFT infusion
Core Phase - Proportion of participants with reversal of renal Fanconi syndrome (RFS)
Proportion of participants with reversal of renal Fanconi syndrome (RFS)
Measured: Up to 32 months
Secondary outcomes (32)
Core Phase - Number of participants independent from cysteamine
Independence from oral and ophthalmic cysteamine
Measured: up to 24 months
Core Phase - Health-related quality of life (HRQOL)
Health-related quality of life (HRQOL) as measured by QUALIFY (cystinosis-specific PRO)- Currently under development
Measured: Up to 32 months
Core Phase - Time from infusion to reversal of RFS (Cohort 1)
Time from infusion to reversal of renal Fanconi syndrome (RFS)
Measured: Up to 24 months
Core Phase - Time from screening to reversal of RFS (Cohort 0)
Time from screening to reversal of renal Fanconi syndrome (RFS)
Measured: Up to 24 months
Core Phase - Duration of reversal of RFS
Duration of reversal of renal Fanconi syndrome (RFS)
Measured: Up to 24 months
Core Phase - Change from baseline on urine protein to creatinine ratio (UPr/CR)
Change from baseline on urine protein to creatinine ratio (UPr/CR)
Measured: Up to 27 months
Core Phase - Change from baseline on urine amino acids
Change from baseline on urine amino acids
Measured: Up to 27 months
Core Phase - Change from baseline on urinary glucose to creatinine ratio
Change from baseline on urinary glucose to creatinine ratio
Measured: Up to 27 months
Core Phase - Change from baseline on tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate ratio (TmP/GFR)
Change from baseline on tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate ratio
Measured: Up to 27 months
Core Phase - Change from baseline on urine retinol-binding protein/creatinine ratio (RBP/Cr)
Change from baseline on urine retinol-binding protein/creatinine ratio
Measured: Up to 27 months
Core Phase - Number of participants with improvement of proximal tubular function
Improvement of proximal tubular function as defined by 2-fold change from Baseline of at least 4 out of 5 RFS parameters (urine protein to creatinine ratio, urine amino acids, urinary glucose to creatinine ratio, tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate, urine retinol-binding protein/creatinine ratio). Improvement will also be considered if the change from Baseline is less than 2-fold but the value falls within the definition for normalization.
Measured: Up to 27 months
Core Phase - Corneal cystine crystal content
Corneal crystal content by anterior segment optical coherence tomography (AS-OCT)
Measured: Up to 27 months
Core Phase - Number of participants with clinically significant changes in vital signs, physical examinations, laboratories, and ECG (Cohort 1)
Vital signs, physical examinations, laboratories (eg., chemistry, hematology, liver function tests), and ECG
Measured: Up to 27 months
Core Phase - Number of participants with presence/emergence of replication-competent lentivirus (Cohort 1)
Number of participants with presence/emergence of replication-competent lentivirus
Measured: Up to 27 months
Core Phase - Number of participants with malignancy (Cohort 1)
Number of participants with malignancy
Measured: Up to 27 months
Core Phase - Time to hematological reconstitution (Cohort 1)
Time to hematological reconstitution
Measured: Up to 24 months
Core Phase - Time to platelet engraftment (Cohort 1)
Time to platelet engraftment
Measured: Up to 24 months
Core Phase - Incidence of Adverse Events (Cohort 0)
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Measured: up to 24 months
Extension Phase Primary Objective - Incidence of Adverse events (Cohort 1)
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Measured: Up to 15 years and 8 months
Extension Phase - Number of participants with malignancy (Cohort 1)
Number of participants with malignancy
Measured: Up to 15 years and 3 months
Extension Phase - Number of participants with presence/emergence of replication-competent lentivirus (Cohort 1)
Number of participants with presence/emergence of replication-competent lentivirus
Measured: Up to 15 years and 3 months
Extension Phase - Number of participants with clinically significant changes in vital signs, physical examinations, laboratories, and ECG (Cohort 1)
Vital signs, physical examinations, laboratories (eg., chemistry, hematology, liver function tests), and ECG
Measured: Up to 15 years and 3 months
Extension Phase - Change from baseline on urine protein to creatinine ratio (UPr/CR) (Cohort 1)
Change from baseline on urine protein to creatinine ratio (UPr/CR)
Measured: Up to 15 years and 3 months
Extension Phase - Change from baseline on urine amino acids (Cohort 1)
Change from baseline on urine amino acids
Measured: Up to 15 years and 3 months
Extension Phase - Change from baseline on urinary glucose to creatinine ratio (Cohort 1)
Change from baseline on urinary glucose to creatinine ratio
Measured: Up to 15 years and 3 months
Extension Phase - Change from baseline on tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate ratio (TmP/GFR) (Cohort 1)
Change from baseline on tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate ratio
Measured: Up to 15 years and 3 months
Extension Phase - Change from baseline on urine retinol-binding protein/creatinine ratio (RBP/Cr) (Cohort 1)
Change from baseline on urine retinol-binding protein/creatinine ratio
Measured: Up to 15 years and 3 months
Extension Phase - Duration of reversal of RFS (Cohort 1)
Duration of reversal of renal Fanconi syndrome (RFS)
Measured: Up to 15 years
Extension Phase - Number of participants with kidney failure (Cohort 1)
Number of participants with kidney failure
Measured: Up to 15 years
Extension Phase - Number of participants independent from cysteamine (Cohort 1)
Independence from oral and ophthalmic cysteamine
Measured: Up to 15 years
Extension Phase - Corneal cystine crystal content (Cohort 1)
Corneal crystal content by anterior segment optical coherence tomography (AS-OCT)
Measured: Up to 15 years and 3 months
Extension Phase - Health-related quality of life (HRQOL) (Cohort 1)
Health-related quality of life (HRQOL) as measured by QUALIFY (cystinosis-specific PRO)- Currently under development
Measured: Up to 15 years and 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2025
- Study start2 Jun 2025
- Primary completion28 May 2031 (estimated)
- Study completion28 May 2044 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nephropathic CystinosisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06910813
- Sponsor's protocol ID
- CDFT383A12101
- Acronym
- CYStem
- Status last verified
- Oct 2026
- First submitted
- 17 Mar 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase
- ClinicalTrials.gov ID
- NCT06910813
- Acronym / sponsor ID
- CYStem / CDFT383A12101
- Conditions
- Nephropathic Cystinosis
- Keywords
- Cystinosis, Nephropathic cystinosis, Lysosomal storage disorder, CTNS gene, DFT383, Cellular gene therapy, Cysteamine, Renal Fanconi syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 2 Jun 2025 (actual)
- Primary completion
- 28 May 2031 (estimated)
- Study completion
- 28 May 2044 (estimated)
- First posted
- 4 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 nephropathic cystinosis, followed by a long-term extension phase.
The study includes two Treatment Groups (Cohort 1 and Cohort 0) and consists of a Core Phase and a long-term Extension Phase.
Participants in Cohort 1 will receive DFT383 and participate in both the Core and Extension Phase. Participants in Cohort 0 will not receive study treatment and will participate in the Core Phase only.
The two cohorts will be run in parallel. Investigational sites may participate in one or both cohorts.
Cohort 1 Approximately 15 participants will receive treatment with DFT383 in 3 (sub) cohorts (1A, 1B and 1C) dosed in a staggered approach. The total study duration for a participant in Cohort 1 will be up to 32 months in the core phase and up to 13 years for the long-term extension phase.
Cohort 0 Approximately 15 participants meeting similar inclusion/exclusion criteria and receiving SoC will be enrolled. The Schedule of Activities will be reduced for this Cohort. This cohort 0 is not a direct control but will provide essential context for interpreting the results observed in the participants receiving DFT383. The total study duration for a participant in Cohort 0 will be up to 24 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (DFT383) | Experimental | Genetic: DFT383Treatment with DFT383 |
| Cohort 0 (SoC) | No intervention | –No study treatment, will continue with standard of care (cysteamine). |
Who may qualify
- Ages
- 2 to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DFT383DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy. | Genetic | Cohort 1 (DFT383) |
Primary outcome
Core Phase - Incidence of adverse events (Cohort 1)
Time frame: Up to 32 months
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Core Phase - Number of participants with hematological reconstitution (Cohort 1)
Time frame: 42 days post DFT infusion
Hematological reconstitution by Day 42 post-DFT383 infusion
Core Phase - Proportion of participants with reversal of renal Fanconi syndrome (RFS)
Time frame: Up to 32 months
Proportion of participants with reversal of renal Fanconi syndrome (RFS)
32 secondary outcomes
Core Phase - Number of participants independent from cysteamine
Time frame: up to 24 months
Core Phase - Health-related quality of life (HRQOL)
Time frame: Up to 32 months
Core Phase - Time from infusion to reversal of RFS (Cohort 1)
Time frame: Up to 24 months
Core Phase - Time from screening to reversal of RFS (Cohort 0)
Time frame: Up to 24 months
Core Phase - Duration of reversal of RFS
Time frame: Up to 24 months
Core Phase - Change from baseline on urine protein to creatinine ratio (UPr/CR)
Time frame: Up to 27 months
Core Phase - Change from baseline on urine amino acids
Time frame: Up to 27 months
Core Phase - Change from baseline on urinary glucose to creatinine ratio
Time frame: Up to 27 months
Core Phase - Change from baseline on tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate ratio (TmP/GFR)
Time frame: Up to 27 months
Core Phase - Change from baseline on urine retinol-binding protein/creatinine ratio (RBP/Cr)
Time frame: Up to 27 months
Core Phase - Number of participants with improvement of proximal tubular function
Time frame: Up to 27 months
Core Phase - Corneal cystine crystal content
Time frame: Up to 27 months
Core Phase - Number of participants with clinically significant changes in vital signs, physical examinations, laboratories, and ECG (Cohort 1)
Time frame: Up to 27 months
Core Phase - Number of participants with presence/emergence of replication-competent lentivirus (Cohort 1)
Time frame: Up to 27 months
Core Phase - Number of participants with malignancy (Cohort 1)
Time frame: Up to 27 months
Core Phase - Time to hematological reconstitution (Cohort 1)
Time frame: Up to 24 months
Core Phase - Time to platelet engraftment (Cohort 1)
Time frame: Up to 24 months
Core Phase - Incidence of Adverse Events (Cohort 0)
Time frame: up to 24 months
Extension Phase Primary Objective - Incidence of Adverse events (Cohort 1)
Time frame: Up to 15 years and 8 months
Extension Phase - Number of participants with malignancy (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Number of participants with presence/emergence of replication-competent lentivirus (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Number of participants with clinically significant changes in vital signs, physical examinations, laboratories, and ECG (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Change from baseline on urine protein to creatinine ratio (UPr/CR) (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Change from baseline on urine amino acids (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Change from baseline on urinary glucose to creatinine ratio (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Change from baseline on tubular maximum reabsorption of Phosphate/Glomerular Filtration Rate ratio (TmP/GFR) (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Change from baseline on urine retinol-binding protein/creatinine ratio (RBP/Cr) (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Duration of reversal of RFS (Cohort 1)
Time frame: Up to 15 years
Extension Phase - Number of participants with kidney failure (Cohort 1)
Time frame: Up to 15 years
Extension Phase - Number of participants independent from cysteamine (Cohort 1)
Time frame: Up to 15 years
Extension Phase - Corneal cystine crystal content (Cohort 1)
Time frame: Up to 15 years and 3 months
Extension Phase - Health-related quality of life (HRQOL) (Cohort 1)
Time frame: Up to 15 years and 8 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing DFT383 in people with Nephropathic Cystinosis, run by Novartis Pharmaceuticals at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Core Phase - Incidence of adverse events (Cohort 1) (time frame: Up to 32 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.