Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms. Phase 1 Monotherapy consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B). Phase 2 Monotherapy is a randomized, open-label, crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 Monotherapy is a randomized open-label crossover study comparing the final fixed dose of oral ASTX030 to SC azacitidine. Phase 1 Combination Therapy is an open-label, multicenter, randomized, exploratory study comparing ASTX030 and SC azacitidine in combination with venetoclax in participants with treatment-naïve AML. Phase 2 Combination Therapy is an open-label, single arm, study evaluating the efficacy, safety, pharmacokinetics (PK), and drug interactions of ASTX030 in combination with venetoclax in participants with treatment-naïve AML. The duration of this multi-phase study is approximately 8 years.
RNA-lipid Particle (RNA-LP) Vaccines for Recurrent/Progressive Medulloblastoma (MB)
This is not medical advice. AI-assisted translation — inaccuracies may occur. Always verify the original and consult your oncologist before taking any steps.
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About the trial
The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in pediatric patients with recurrent/progressive Medulloblastoma (MB)
Original English text from ClinicalTrials.gov
Who can (and can't) join
✓ Qualifies
- •Wiek od 3 do 39 lat.
- •Potwierdzony lub podejrzewany nawrotowy/postępujący medulloblastoma po pierwszym lub drugim nawrocie.
- •Pacjent musi otrzymać radioterapię jako część wcześniejszego leczenia.
- •Musiała być pobrana próbka guza do przygotowania szczepionki.
- •Pacjent musi być w pełni zdrowy po wcześniejszym leczeniu przeciwnowotworowym (bez ostrych skutków ubocznych).
- •Od ostatniego leczenia przeciwnowotworowego musi minąć określony czas (np. 90 dni po radioterapii miejscowej).
- •Badanie rezonansem magnetycznym (MRI) głowy i kręgosłupa wykonane przed badaniem.
- •Ogólny stan sprawności pacjenta (oceniany przed badaniem).
✗ Disqualifies
- •Pacjenci, którzy otrzymali wcześniejszą terapię ukierunkowaną na pp65 lub jakąkolwiek terapię RNA-LP.
Simplified criteria — AI translation
Trial details
- Minimum age
- 4 Years
- Maximum age
- 39 Years
- Last updated (source)
- September 29, 2026
- Sex
- No restrictions
Therapies / drugs in trial
Locations (1)
UF Health Shands Children's Hospital
Gainesville, United States
Share this trial
Data from ClinicalTrials.gov. AI-assisted translation, last sync: 7/3/2026.
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