Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
50
In part 1, three different doses will be administered to 3 participants with either disease indication to establish maximum tolerated dose In part 2, the MTD established in Part-1 will be administered as six once-weekly infusions to up to 10 participants per each of four baskets (cSLE, AAV, JDM and JMG) in an outpatient setting.
H01- Children's National Hospital
Washington D.C., District of Columbia, United States
RECRUITINGH03- Washington University School of Medicine
St Louis, Missouri, United States
RECRUITINGMaximum tolerated dose in Part 1, type and frequency of treatment related SAE's in Part 2
The primary endpoint for Part-1 is the Maximum Tolerated Dose (MTD), defined as the Dose Level at which no more than 20% of the patients treated have shown Dose-Limiting Toxicity (DLT), i.e. at which 3 patients received all 6 weekly infusions without a DLT by Day 50; or 6 patients received 3 weekly infusions with no more than 1 patient having a DLT by Day 50. The primary endpoint for Part-2 is the type and frequency of treatment-related SAEs. This will be assessed on Days 22, 50 and Months 3,6,9,12
Time frame: Days 22 and 50 for Part1 , Days 22, 50 and Months 3,6,9 and 12 for part 2
Maximum Dose Tolerated
The primary endpoint for Part-1 is the Maximum Tolerated Dose (MTD), defined as the Dose Level at which no more than 20% of the patients treated have shown Dose-Limiting Toxicity (DLT), i.e. at which 3 patients received all 6 weekly infusions without a DLT by Day 50; or 6 patients received 3 weekly infusions with no more than 1 patient having a DLT by Day 50. The primary endpoint for Part-2 is the type and frequency of treatment-related SAEs.
Time frame: Days 2250 for Part 1, Days 11,50, month 3 for part 2 in addition to months, 4,6,9,12
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