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

Phase 1

• Conditions: Plexiform Neurofibroma Associated With Neurofibromatosis Type 1; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2016-001563-36-Outside-EU/EEA
• Lead Sponsor: ovartis Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-05-12
• Last Update Posted: 17 May 2016

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 26

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.

Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified