The Novartis stem cell trial is moving forward! We are excited to inform you that one arm of the Novartis CYStem stem cell and gene therapy trial is officially recruiting! This arm of the study is for Cohort 0 which is the group of patients that will NOT be treated with the stem cells. Cohort 0 will receive the Standard of Care (SoC) which is our current treatment of cysteamine, etc. Dr. Larry Greenbaum at Emory University is the Principal Investigator of this arm of the trial.
The other sites that will be treating patients (at UC San Diego and Stanford) should be formally recruiting soon although if you are interested, your child’s physician can contact Novartis at any time about participating.
We have come so far and although it sometimes feels like it has taken a long time, this FDA approved clinical trial has moved relatively quickly and against all odds. CRF research grants, our drive and passion have ensured that the stem cell treatment, once a wish, is now a reality.
Our community came together in 2007 and raised millions to fund the stem cell research. We had patients volunteer for the very early studies and we had five brave adults who risked their lives to prove the stem cell treatment was safe (and efficacious) and here we are today, the next phase (and we hope the last) approved to treat our youngest children.
As we learned from Novartis at the CRF family conference, this phase of the trial will evolve as the first cohort of patients are treated. If the first cohorts do well, there is a chance that the trial will be opened to additional cohorts in other age groups. This is exactly what our advocacy with Novartis has accomplished – confirmation that the trial will move as fast as possible and will eventually and hopefully, include additional cohorts giving access to a broader group of patients!
CRF has worked closely with Novartis for two years to champion the mission of CRF and this trial. In our advisory role with Novartis, we ensured that Novartis and the FDA understood the current, demanding 24/7 treatment protocol for cystinosis, the tremendous burden of disease, and the impact the medications have on quality of life. CRF has had one goal when it comes to this trial – we want this treatment available to every person of every age with cystinosis and as soon as possible.
We are on our way, and we will continue to work with Novartis, Dr. Paul Grimm, Dr. Larry Greenbaum, and the person who made this all possible – the brilliant Dr. Stéphanie Cherqui – to represent the entire cystinosis community as this trial advances. We are proud of our efforts, and thank you for your love and support throughout the process. We will cross the finish line united in our purpose!
A bit more information from clinicaltrials.gov:
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. The study consists of participants receiving DFT383 in Cohort 1 and Standard of Care (SoC) in Cohort 0. 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 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.
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.
https://clinicaltrials.gov/study/NCT06910813?cond=cystinosis&aggFilters=status:not&rank=1#contacts-and-locations
