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| Diagnosis | Non-hematological tumours (solid and brain) with relevant biomarker | Study Status | Closed |
| Phase | I/II |
| Age | 12 Years and older | Randomisation | NO |
| Line of treatment | Disease relapse or progression |
| Routes of Treatment Administration | Drug: DAY101 (Oral tablet)
Drug: Pimasertib Hydrochloride (Oral capsule) - Sub-study B only.
|
| Last Posted Update | 2024-07-31 |
| ClinicalTrials.gov # | NCT04985604 |
International Sponsor
Day One Biopharmaceuticals, Inc.Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel MorgensternCentres
Study Description
This is a Phase 1b/2, multi-center, open label umbrella study of patients ≥12 years of age with recurrent or progressive solid (including CNS) tumors with alterations in the key proteins of the RAS/RAF/MEK/ERK pathway, referred to as the MAPK pathway. Study DAY101-102 (master study) and sub-studies will consist of a screening period, a treatment period, a safety follow-up period, and a long-term follow-up period where survival, status and subsequent anticancer therapies are collected.
DAY101 will be evaluated alone (sub-study A) or combined with a different targeted therapy (sub-study B) in each sub-study. The Phase 1b part of each sub-study will evaluate the safety of the combination and select the dose for the Phase 2 part. The Phase 2 part of each sub-study will evaluate anti-tumor activity.
Inclusion Criteria
- Signed assent for patients ≥ 12 up to < 18 years of age
- Patients must have a histologically confirmed diagnosis of non-hematological tumor with concurrent MAPK pathway alteration as assessed by sequencing, PCR, FISH, or another clinically accepted molecular diagnostic method recognized by local laboratory or regulatory agency
- Patients must have radiographically-recurrent or radiographically-progressive disease that is measurable using the appropriate tumor response criteria (e.g. RECIST version 1.1)
- Archival tumor tissue (preferably less than 3 years old) or fresh tumor tissue for correlative studies is required
- If brain metastases are present, they must have been previously treated and be stable as assessed by radiographic imaging
Other inclusion criteria may apply
Exclusion Criteria
- Known presence of concurrent activating mutation
- Patients with current evidence or a history of central serous retinopathy (CSR), retinal vein occlusion (RVO)
Substudy A-specific exclusion criterion:
- Prior therapy of any RAS- RAF-, MEK-, or ERK-directed inhibitor therapy
Substudy B-specific exclusion criterion:
- Prior receipt of any Type-II pan-RAF inhibitor therapy (e.g., LXH254/naporafenib, BGB- 283, BGB-3245, belvarafenib)
Other exclusion criteria may apply
