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Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1) (MACS0977)

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01365468
First received: May 27, 2011
Last updated: January 6, 2014
Last verified: January 2014
  Purpose

This study will evaluate the antitumor activity and safety of RAD001 in patients with Plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1).

The aim of the study is to :

  1. determine whether RAD001, administrated orally daily on a continuous dosing schedule might:

    1. Increases time to disease progression (TTP) based on volumetric MRI measurements in children and adults with NF1 in inoperable documented progressive PN (stratum 1).
    2. Results in objective radiographic responses based on volumetric MRI measurements in children and adults with NF1 and inoperable PN in the absence of documented radiographic progression at the trail entry (stratum
  2. To evaluate the tolerability and toxicity of chronic RAD001 administration in this patient population as assessed by the NCI Common Toxicity Criteria, version 4.0.

Condition Intervention Phase
Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
Drug: Everolimus
Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • time to disease progression (TTP) based on change in volumetric MRI measurements in children and adults [ Time Frame: Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • objective radiographic responses based on volumetric MRI measurements in children and adults with NF1 and inoperable PN [ Time Frame: Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days) ] [ Designated as safety issue: No ]
  • feasibility and toxicity of chronic RAD001 administration in this patient population as assessed by the NCI Common Toxicity Criteria, version 4.0 [ Time Frame: From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months) ] [ Designated as safety issue: Yes ]
  • evaluate the clinical effect of RAD001 on clinical response by quality of life questionnaire and improvement in function or performance scale [ Time Frame: Screening, Day 1, every 3 courses, End of Study ] [ Designated as safety issue: Yes ]
  • evaluate the effect of RAD001 on skin lesions as assessed by Digital Photographs [ Time Frame: Screening, after course #3, #6, #12, #18, #24, End of Treatment ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: May 2012
Estimated Study Completion Date: May 2016
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RAD001 Drug: Everolimus
Other Name: RAD001

  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Clinically definite diagnosis of NF1 according to the NIH consensus conference criteria.
  2. Patients must have PN that have the potential to cause significant morbidity, such as lesions that could compromise the airway or the great vessels, lesions that could cause nerve compression, lesions that could result in major deformity or significant cosmetic problems
  3. Measurable disease: patient must have at least one measurable PN amenable to volumetric MRI analysis.

Exclusion Criteria:

  1. Chronic treatment with systemic steroids or another immunosuppressive agent.
  2. Evidence of an active optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer requiring treatment with chemotherapy or radiation therapy.
  3. Clinical evidence of significantly impaired lung function
  4. Pregnancy or breast feeding.
  5. Prior therapy with mTOR inhibitors (e.g.sirolimus, temsirolimus, everolimus).
  6. No contraindications for MRI assessments

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01365468

Contacts
Contact: Novartis Pharmaceuticals +41613241111
Contact: Novartis Pharmaceuticals

Locations
Israel
Novartis Investigative Site Recruiting
Tel-Aviv, Israel, 64239
Novartis Investigative Site Recruiting
Tel-Hashomer, Israel, 52621
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01365468     History of Changes
Other Study ID Numbers: CRAD001MIL04T
Study First Received: May 27, 2011
Last Updated: January 6, 2014
Health Authority: Israel: Ministry of Health

Keywords provided by Novartis:
RAD001,
Everolimus,
Plexiform Neurofibroma,
Neurofibromatosis Type 1

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Neoplastic Syndromes, Hereditary
Nerve Sheath Neoplasms
Nervous System Diseases
Nervous System Neoplasms
Neurocutaneous Syndromes
Neurodegenerative Diseases
Neuromuscular Diseases
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Everolimus
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 19, 2014