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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


Phase 1/Phase 2
12 Months
18 Years
Open (Enrolling)
Both
Medulloblastoma,, Rhabdomyosarcoma,, Neuroblastoma,, Hepatoblastoma,, High Grade Glioma,, Astrocytoma

Thank you

Trial Information

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


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

Type of Study:

Interventional

Study Design:

Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Phase I - Determine the maximum tolerated dose of and characterize the dose limiting toxicities of LDE225.

Outcome Time Frame:

6 weeks

Safety Issue:

Yes

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CLDE225X2104

NCT ID:

NCT01125800

Start Date:

February 2011

Completion Date:

February 2014

Related Keywords:

  • Medulloblastoma,
  • Rhabdomyosarcoma,
  • Neuroblastoma,
  • Hepatoblastoma,
  • High Grade Glioma,
  • Astrocytoma
  • Recurrent,
  • refractory,
  • medulloblastoma,
  • rhabdomyosarcoma,
  • neuroblastoma,
  • hepatoblastoma,
  • astrocytoma,
  • children,
  • pediatric,
  • hedgehog pathway inhibitor, adult
  • Astrocytoma
  • Glioma
  • Medulloblastoma
  • Neuroblastoma
  • Rhabdomyosarcoma
  • Hepatoblastoma

Name

Location

Seattle Children's Hospital CPKC412A2114Seattle, Washington  98105
Memorial Sloan Kettering Cancer Center Sloan KetteringNew York, New York  10021
Duke University Medical Center Duke - BakerDurham, North Carolina  27710
Childrens National Medical Center SCWashington, District of Columbia  20010-2120
H. Lee Moffitt Cancer Center/University of South Florida H LeeTampa, Florida  33612
Children's Healthcare of Atlanta Childern Hosp - ATLAtlanta, Georgia  30342
Sidney Kimmel Comprehensive Cancer Center/Johns Hopkins Med. John HopkinsBaltimore, Maryland  21231
Dana Farber Cancer Institute DFCI (3)Boston, Massachusetts  02115
MD Anderson Cancer Center/University of Texas MD LDEX2104Houston, Texas  77030-4009