Clareo Health | 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
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Keep this study
Lead
Novartis Pharmaceuticals
Age
1–18
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
