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

Completed · Phase 1/Phase 2

Conditions studied: Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma, Hepatoblastoma, Glioma, Astrocytoma

In brief

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.

Key facts

Study ID

NCT01125800

Run by

Novartis Pharmaceuticals

People needed

76

Starts

2011-02-01

Expected to finish

2014-10-01

Last updated by the study team

2017-03-20

Who can join

Age: 1 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify 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 qualify 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
• Other protocol-defined inclusion/exclusion criteria may apply

Where it is running

• Children's Healthcare of Atlanta Childern Hosp - ATL — Atlanta, Georgia, United States
• Sidney Kimmel Comprehensive Cancer Center/Johns Hopkins Med. John Hopkins — Baltimore, Maryland, United States
• Dana Farber Cancer Institute DFCI (3) — Boston, Massachusetts, United States
• Seattle Children's Hospital CPKC412A2114 — Seattle, Washington, United States
• Novartis Investigative Site — Parkville, Victoria, Australia
• Novartis Investigative Site — Toronto, Ontario, Canada
• Novartis Investigative Site — Toronto, Ontario, Canada
• Novartis Investigative Site — Paris, France
• Novartis Investigative Site — Villejuif, France
• Novartis Investigative Site — Bologna, BO, Italy
• Novartis Investigative Site — Milan, MI, Italy
• Novartis Investigative Site — Roma, RM, Italy
• Novartis Investigative Site — Sutton, Surrey, United Kingdom
• Novartis Investigative Site — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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