Clinical trial · Interventional
A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB
A Phase I/II Study of LDE225 in Pediatric Patients With Recurrent or Refractory Medulloblastoma or Other Tumors Potentially Dependent on the Hedgehog-signaling Pathway and Adult Patients With Recurrent or Refractory Medulloblastoma
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway. Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.
Conditions
Conditions (6)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Astrocytoma | Astrocytoma | CURATED_BROADER | 0.80 |
| Glioma | Glioma | ONTOLOGY_EXACT | 0.98 |
| Hepatoblastoma | Hepatoblastoma | ONTOLOGY_EXACT | 0.98 |
| Medulloblastoma | Medulloblastoma | CURATED_BROADER | 0.80 |
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
| Rhabdomyosarcoma | Rhabdomyosarcoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| LDE225 | Drug | Sonidegib | ALIAS |
Design
Arms and outcomes
Arms (5)
- type
- EXPERIMENTAL
- label
- LDE225 233mg/m2 daily dose
- description
- Pediatric dose.
- interventionNames
- Drug: LDE225
- type
- EXPERIMENTAL
- label
- LDE225 372mg/m2 daily dose
- description
- Pediatric dose.
- interventionNames
- Drug: LDE225
- type
- EXPERIMENTAL
- label
- LDE225 425 mg/m2 daily dose
- description
- Pediatric dose.
- interventionNames
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 12 Months
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Phase I - Patients aged ≥12 months and \<18 years, Phase II - Patients ≥12 months * Phase I - Histologically confirmed diagnosis of medulloblastoma, rhabdomyosarcoma, neuroblastoma, hepatoblastoma, high grade glioma, or osteosarcoma, that has progressed despite treatment with standard therapies, or for which no standard treatments are available (patients with brainstem gliomas are excluded). Phase II - Histologically confirmed diagnosis of recurrent or relapsed medulloblastoma with at least one measurable lesion. * Performance Status: Karnofsky ≥60% for patients \>10 yrs, Lansky ≥50 for patients less than or equal to 10 yrs * Protocol-defined renal , liver and bone marrow function * Negative pregnancy test before starting study treatment. If of child bearing potential must use 'highly effective' methods of contraception. * All patients must consent to provide a tumor sample Exclusion Criteria: * Systemic anti-cancer treatment within 2 weeks prior to first dose (6 weeks for nitrosourea, mitomycin and monoclonal antibodies). * Focal radiotherapy within 4 weeks prior to first dose, or full spinal radiotherapy within 3 months of first dose. * Unresolved toxicity greater than CTCAE grade 1 from previous anti-cancer therapy or radiotherapy (excluding neurotoxicity, alopecia, ototoxicity, lymphopenia or other specifications in the eligibility criteria for this study), or incomplete recovery from previous surgery, unless agreed by Novartis and the Principal Investigator (PI) and documented. * Major surgery, serious illness or traumatic injury within 2 weeks of starting study therapy. Patients anticipated to require major surgery within the first 2 cycles of treatment. * Patients requiring a nasogastric tube for drug administration (G-tubes are permitted) * Impaired cardiac function * Pregnant or breast-feeding females * Impairment of gastrointestinal (GI) function or GI disease Other protocol-defined inclusion/exclusion criteria may apply
References
Publications (0)
Data not yet available