Plexiform Neurofibroma Associated With Neurofibromatosis Type 1 Clinical Trial
Official title:
A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)
| Verified date | June 2015 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Israel: Ministry of Health |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 9 |
| Est. completion date | April 2015 |
| Est. primary completion date | April 2015 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 10 Years and older |
| Eligibility |
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 |
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Israel | Novartis Investigative Site | Tel-Aviv | |
| Israel | Novartis Investigative Site | Tel-Hashomer |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
Israel,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | time to disease progression (TTP) based on change in volumetric MRI measurements in children and adults | Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days) | No | |
| Secondary | objective radiographic responses based on volumetric MRI measurements in children and adults with NF1 and inoperable PN | Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days) | No | |
| Secondary | feasibility and toxicity of chronic RAD001 administration in this patient population as assessed by the NCI Common Toxicity Criteria, version 4.0 | From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months) | Yes | |
| Secondary | evaluate the clinical effect of RAD001 on clinical response by quality of life questionnaire and improvement in function or performance scale | Screening, Day 1, every 3 courses, End of Study | Yes | |
| Secondary | evaluate the effect of RAD001 on skin lesions as assessed by Digital Photographs | Screening, after course #3, #6, #12, #18, #24, End of Treatment | No |