Neurofibromatosis Type 1Phase 2NFitorNCT01412892
Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas
A Phase 2 study testing RAD001: Everolimus in people with Neurofibromatosis Type 1 and Plexiform Neurofibroma, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Background: Patients with the genetic disorder neurofibromatosis Type 1 (NF1) are at increased risk of developing tumors of the central and peripheral nervous system. These include plexiform neurofibromas. The conventional treatment of these internal plexiform neurofibromas is surgery. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Neurofibromatosis Type 1, Plexiform Neurofibroma, Neurofibromatoses
- What is being tested?
- RAD001: Everolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- Apr 2011to Oct 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Clinical diagnosis of NF1, according to NIH criteria, with internal plexiform neurofibroma (PN) and at least 1 of criteria for NF1:
- 6 or more café-au-lait spots Freckling in the axilla or groin Optic glioma 2 or more Lisch nodules Distinctive bony lesion
- 1-degree relative with NF1
- At least 1 inoperable PN(s) that has/have the potential to cause significant morbidity: Paravertebral lesions that could compromise the spinal cord Head and neck lesions that could compromise the airway or great vessels Brachial or lumbar plexus lesions that could cause nerve compression and loss of function Lesions that could result in major deformity (e.g., orbital lesions) or significant cosmetic problems Lesions of the extremity that cause limb hypertrophy or loss of function Painful lesions
- Complete resection of a PN with acceptable morbidity is not feasible OR patient refuses surgery OR the number of PNs leads to not feasible surgery according to the steering committee's site
- Measurable PN amenable to volumetric MRI analysis using fusion of images
Show all 17Show fewer
- Measurable lesion (at least 3 cm in one dimension)
- Karnofsky >70%
- 18≤ Age ≤60
- absolute neutrophil count (ANC) ≥1.5x109/L, Platelets ≥100x109/L, Hb >9g/dL
- bilirubin: ≤1.5xULN, ALT and AST ≤2.5xULN unless evident Gilbert disease (amendment n°2). For patients with known liver metastases: AST and ALT ≤ 5xULN
- Creatinine ≤ 1.5xULN
- Life expectancy ≥ 2 years
- Cholesterol ≤300 mg/dL or ≤7.75 mmol/L and triglycerides ≤ 2.5x ULN
- Women of childbearing potential must have had a negative serum pregnancy test within 7 days and a negative urine pregnancy test within 72 hours prior to the administration of RAD001 start and must use an effective birth control method.
- Men should use condoms and their partner(s) use an effective birth control method
- A written informed consent obtained
Who cannot join
- Patients who/with:
- have previously received mTOR inhibitors
- a known hypersensitivity to RAD001 or other rapamycin or to its excipients
- receiving chronic systemic treatment with corticosteroids or another immunosuppressive agent. (Dose equivalent to 10 mg/day of methylprednisone), topical steroids or organotherapy for bilateral adrenalectomy are acceptable
- a known history of HIV seropositivity
- acute viral hepatitis
Show all 15Show fewer
- autoimmune hepatitis
- with an active, bleeding diathesis. Patients may use coumadin or heparin preparations
- have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation
- have a history of another primary malignancy ≤3 years, with the exception of non-melanoma skin cancer and carcinoma in situ of the uterine cervix
- Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are being used, these must be continued throughout the trial by both sexes. Oral contraceptives are not acceptable alone
- a contraindication to MRI
- are using other investigational agents or who had received investigational drugs ≤ 4 weeks prior to study treatment start
- unwilling or unable to comply with the protocol
- not affiliated to health system
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single Arm, Multicenter Phase II a Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas That Cannot be Removed by Surgery
Background:
Patients with the genetic disorder neurofibromatosis Type 1 (NF1) are at increased risk of developing tumors of the central and peripheral nervous system. These include plexiform neurofibromas. The conventional treatment of these internal plexiform neurofibromas is surgery. This surgery can be possible on a single and limited tumor. On the other hand these tumors are often surgically intractable due to their multiplicity and their infiltrating characteristics Increased activity of mammalian target of rapamycin(mTOR) protein is seen in neurofibromas. mTOR inhibitor rapamycin , or its derivatives such as everolimus may slow or stop tumor growth in patients with NF1.
Read more
Objectives:
Primary objectives To determine whether everolimus has an effect on the volume of surgically intractable and life-threatening internal plexiform neurofibromas in patients with neurofibromatosis 1.
Secondary objectives To determine whether everolimus has an effect on the number and the volume of cutaneous neurofibromas; to determine whether everolimus modify the signaling pathways in cutaneous neurofibromas.
Eligibility:
- Adults with neurofibromatosis type 1 with at least one internal plexiform neurofibroma, life-threatening or causing significant morbidity through compression of organs. This or these internal plexiform neurofibroma(s) should be intractable by surgery.
Design:
An open-label, single arm, non-randomized, single stage phase IIa study. Baseline phase: Baseline evaluations will be performed within 2 weeks, and up to a maximum of 4 weeks for specific exams, before the first dose of study drug.
Treatment phase/duration of treatment: All patients will be treated with RAD001 10 mg p.o daily dose for one year except in case of unacceptable toxicity, death, or discontinuation from the study for any other reason.
Follow-up phase: All patients will have two follow-up visits scheduled at 18 and 24 months after the first dose of the study drug to follow for adverse events (AEs) and serious adverse events (SAEs) that may have occurred after discontinuation from the study and for internal plexiform neurofibromas assessment.
Radiological review: All Magnetic Resonance Imaging (MRIs) obtained at baseline, during the treatment period and the follow-up period will be reviewed by the Neuroradiologist of the study.
Read the detailed description
Neurofibromatosis 1 (NF1) is an autosomal dominant disease affecting 1 in 3000 to 1 in 4000 people. NF1 is characterized by multiple dermal neurofibromas, plexiform neurofibromas, malignant peripheral nerve sheath tumors (MPNST), and optic pathway gliomas, as well as by café-au-lait spots and abnormalities of the skeletal, cardiovascular and central nervous systems. The NF1 gene is located on chromosome 17q11.2, and its protein, neurofibromin, functions as a tumor suppressor.
People with NF1 have a decrease in life expectancy of 15 years, with MPNST as a leading cause of death in young adults. A specific phenotype at risk of mortality has been identified, patients with subcutaneous neurofibromas. Individuals with subcutaneous neurofibromas are more than 3 times as likely to have internal plexiform neurofibromas as others. Individuals with internal plexiform neurofibromas are 18 times more likely to develop MPNST than patients without internal plexiform neurofibromas. Beside MPNST, internal plexiform neurofibromas can be life-threatening or cause of significant morbidity through compression of organs mainly spine or nerve roots.
The conventional treatment of these internal plexiform neurofibromas is surgery. This surgery can be possible on a single and limited tumor. On the other hand these tumors are often surgically intractable due to their multiplicity and their infiltrating characteristics. In this context a medical treatment decreasing the size of these tumors would have its place with as short term aim to lower the consequence of compression and long-term aim to reduce the risk of malignant transformation.
NF1 is a consequence of the loss-of-function mutations in the NF1 tumor suppressor gene. The NF1-encoded protein, neurofibromin, functions as a Ras-GTPase activating protein (RasGAP). Accordingly, deregulation of Ras is thought to contribute to NF1 development. The mTOR pathway is tightly regulated by neurofibromin. mTOR is constitutively activated in both NF1-deficient primary cells and human tumors in the absence of growth factors. This aberrant activation depends on Ras and PI3 kinase, and is mediated by the phosphorylation and inactivation of the TSC2-encoded protein tuberin by AKT. Importantly, tumor cell lines derived from NF1 patients, and a genetically engineered cell system that requires Nf1-deficiency for transformation, are highly sensitive to the mTOR inhibitor rapamycin. Furthermore, the activation of endogenous Ras leads to constitutive mTOR signaling in this disease state, and in normal cells Ras is differentially required for mTOR signaling in response to various growth factors. Thus, the NF1 tumor suppressor is an indispensable regulator of TSC2 and mTOR. Ras plays a critical role in the activation of mTOR in both normal and tumorigenic settings.
mTOR inhibitor rapamycin potently suppresses the growth of aggressive NF1-associated malignancies in a genetically engineered murine model. In these tumors rapamycin does not function via mechanisms generally assumed to mediate tumor suppression, including inhibition of HIF-1 alpha and indirect suppression of AKT, but does suppress the mTOR target Cyclin D1. The mammalian target of rapamycin complex 1 inhibitor RAD001 (Everolimus) decreased growth 19% to 60% after 4 days of treatment in NF1 MPNST cell lines.
Finally, these data suggest that rapamycin, or its derivatives such as everolimus may represent a viable therapy for NF1. This proof of concept has been done in tuberous sclerosis where rapamycin was efficient to treat angiomyolipomas11.
The management of neurofibromatoses in France is coordinated by Pr. Pierre WOLKENSTEIN through the French National Referral Centre for Neurofibromatoses and a network, NF-France. The cohort followed up by the centre and its network is constituted of about 3000 patients and among them between 80 and 100 have life-threatening internal plexiform neurofibromas.
Therefore the investigators propose a trial to evaluate the efficacy of everolimus in surgically intractable and life-threatening internal neurofibromas in neurofibromatosis 1 based on the data of the literature and on our cohort.
- Being tested
- RAD001: Everolimus
- Keywords
- Neurofibromatosis type 1, Everolimus, Plexiform neurofibromas, Rapamycin
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform, Neurofibromatoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAD001: EverolimusDrug
10 mg of RAD001 will be self-administered orally once daily continuously for one year or until unacceptable toxicity or discontinuation from the study from any other reason.
| Study group | Role | What they receive |
|---|---|---|
| Everolimus | Experimental | RAD001: Everolimus Receives: Drug: RAD001: Everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Radiographic response assessed by MRI analysis
Measured: after 1 year of treatment
Secondary outcomes (10)
Radiographic response assessed by MRI analysis
Measured: At 2 years
Overall survival
Measured: At 2 years
Pain
Measured: At 2 years
Deficiency
Measured: At 2 years
Quality of life
Measured: At 2 years
adverse events
Measured: At 2 years
laboratory evaluations
Measured: At 2 years
measurement of vital signs
Measured: At 2 years
performance of physical examinations
Measured: At 2 years
all concomitant medications and therapies
Measured: At 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2011 (estimated)
- Study startApr 2011
- Primary completionOct 2013
- Study completionOct 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Sponsor
- Investigators
- Pierre Wolkenstein, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01412892
- Sponsor's protocol ID
- P090502
- Other IDs
- 2010-023137-34
- Acronym
- NFitor
- Status last verified
- Nov 2014
- First submitted
- 8 Aug 2011
- Last updated
- 14 Nov 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.
PubMed 18184959Zehou O, Ferkal S, Brugieres P, Barbarot S, Bastuji-Garin S, Combemale P, Valeyrie-Allanore L, Sbidian E, Wolkenstein P. Absence of Efficacy of Everolimus in Neurofibromatosis 1-Related Plexiform Neurofibromas: Results from a Phase 2a Trial. J Invest Dermatol. 2019 Mar;139(3):718-720. doi: 10.1016/j.jid.2018.09.016. Epub 2018 Oct 16. No abstract available.
PubMed 30339775
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm, Multicenter Phase II a Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas That Cannot be Removed by Surgery
- ClinicalTrials.gov ID
- NCT01412892
- Acronym / sponsor ID
- NFitor / P090502
- Keywords
- Neurofibromatosis type 1, Everolimus, Plexiform neurofibromas, Rapamycin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Apr 2011
- Primary completion
- Oct 2013 (actual)
- Study completion
- Oct 2014 (actual)
- First posted
- 9 Aug 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
Patients with the genetic disorder neurofibromatosis Type 1 (NF1) are at increased risk of developing tumors of the central and peripheral nervous system. These include plexiform neurofibromas. The conventional treatment of these internal plexiform neurofibromas is surgery. This surgery can be possible on a single and limited tumor. On the other hand these tumors are often surgically intractable due to their multiplicity and their infiltrating characteristics Increased activity of mammalian target of rapamycin(mTOR) protein is seen in neurofibromas. mTOR inhibitor rapamycin , or its derivatives such as everolimus may slow or stop tumor growth in patients with NF1.
Objectives:
Primary objectives To determine whether everolimus has an effect on the volume of surgically intractable and life-threatening internal plexiform neurofibromas in patients with neurofibromatosis 1.
Secondary objectives To determine whether everolimus has an effect on the number and the volume of cutaneous neurofibromas; to determine whether everolimus modify the signaling pathways in cutaneous neurofibromas.
Eligibility:
- Adults with neurofibromatosis type 1 with at least one internal plexiform neurofibroma, life-threatening or causing significant morbidity through compression of organs. This or these internal plexiform neurofibroma(s) should be intractable by surgery.
Design:
An open-label, single arm, non-randomized, single stage phase IIa study. Baseline phase: Baseline evaluations will be performed within 2 weeks, and up to a maximum of 4 weeks for specific exams, before the first dose of study drug.
Treatment phase/duration of treatment: All patients will be treated with RAD001 10 mg p.o daily dose for one year except in case of unacceptable toxicity, death, or discontinuation from the study for any other reason.
Follow-up phase: All patients will have two follow-up visits scheduled at 18 and 24 months after the first dose of the study drug to follow for adverse events (AEs) and serious adverse events (SAEs) that may have occurred after discontinuation from the study and for internal plexiform neurofibromas assessment.
Radiological review: All Magnetic Resonance Imaging (MRIs) obtained at baseline, during the treatment period and the follow-up period will be reviewed by the Neuroradiologist of the study.
Detailed description
Neurofibromatosis 1 (NF1) is an autosomal dominant disease affecting 1 in 3000 to 1 in 4000 people. NF1 is characterized by multiple dermal neurofibromas, plexiform neurofibromas, malignant peripheral nerve sheath tumors (MPNST), and optic pathway gliomas, as well as by café-au-lait spots and abnormalities of the skeletal, cardiovascular and central nervous systems. The NF1 gene is located on chromosome 17q11.2, and its protein, neurofibromin, functions as a tumor suppressor.
People with NF1 have a decrease in life expectancy of 15 years, with MPNST as a leading cause of death in young adults. A specific phenotype at risk of mortality has been identified, patients with subcutaneous neurofibromas. Individuals with subcutaneous neurofibromas are more than 3 times as likely to have internal plexiform neurofibromas as others. Individuals with internal plexiform neurofibromas are 18 times more likely to develop MPNST than patients without internal plexiform neurofibromas. Beside MPNST, internal plexiform neurofibromas can be life-threatening or cause of significant morbidity through compression of organs mainly spine or nerve roots.
The conventional treatment of these internal plexiform neurofibromas is surgery. This surgery can be possible on a single and limited tumor. On the other hand these tumors are often surgically intractable due to their multiplicity and their infiltrating characteristics. In this context a medical treatment decreasing the size of these tumors would have its place with as short term aim to lower the consequence of compression and long-term aim to reduce the risk of malignant transformation.
NF1 is a consequence of the loss-of-function mutations in the NF1 tumor suppressor gene. The NF1-encoded protein, neurofibromin, functions as a Ras-GTPase activating protein (RasGAP). Accordingly, deregulation of Ras is thought to contribute to NF1 development. The mTOR pathway is tightly regulated by neurofibromin. mTOR is constitutively activated in both NF1-deficient primary cells and human tumors in the absence of growth factors. This aberrant activation depends on Ras and PI3 kinase, and is mediated by the phosphorylation and inactivation of the TSC2-encoded protein tuberin by AKT. Importantly, tumor cell lines derived from NF1 patients, and a genetically engineered cell system that requires Nf1-deficiency for transformation, are highly sensitive to the mTOR inhibitor rapamycin. Furthermore, the activation of endogenous Ras leads to constitutive mTOR signaling in this disease state, and in normal cells Ras is differentially required for mTOR signaling in response to various growth factors. Thus, the NF1 tumor suppressor is an indispensable regulator of TSC2 and mTOR. Ras plays a critical role in the activation of mTOR in both normal and tumorigenic settings.
mTOR inhibitor rapamycin potently suppresses the growth of aggressive NF1-associated malignancies in a genetically engineered murine model. In these tumors rapamycin does not function via mechanisms generally assumed to mediate tumor suppression, including inhibition of HIF-1 alpha and indirect suppression of AKT, but does suppress the mTOR target Cyclin D1. The mammalian target of rapamycin complex 1 inhibitor RAD001 (Everolimus) decreased growth 19% to 60% after 4 days of treatment in NF1 MPNST cell lines.
Finally, these data suggest that rapamycin, or its derivatives such as everolimus may represent a viable therapy for NF1. This proof of concept has been done in tuberous sclerosis where rapamycin was efficient to treat angiomyolipomas11.
The management of neurofibromatoses in France is coordinated by Pr. Pierre WOLKENSTEIN through the French National Referral Centre for Neurofibromatoses and a network, NF-France. The cohort followed up by the centre and its network is constituted of about 3000 patients and among them between 80 and 100 have life-threatening internal plexiform neurofibromas.
Therefore the investigators propose a trial to evaluate the efficacy of everolimus in surgically intractable and life-threatening internal neurofibromas in neurofibromatosis 1 based on the data of the literature and on our cohort.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Everolimus | Experimental | Drug: RAD001: EverolimusRAD001: Everolimus |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (17 listed)
- Clinical diagnosis of NF1, according to NIH criteria, with internal plexiform neurofibroma (PN) and at least 1 of criteria for NF1:
- 6 or more café-au-lait spots Freckling in the axilla or groin Optic glioma 2 or more Lisch nodules Distinctive bony lesion
- 1-degree relative with NF1
- At least 1 inoperable PN(s) that has/have the potential to cause significant morbidity: Paravertebral lesions that could compromise the spinal cord Head and neck lesions that could compromise the airway or great vessels Brachial or lumbar plexus lesions that could cause nerve compression and loss of function Lesions that could result in major deformity (e.g., orbital lesions) or significant cosmetic problems Lesions of the extremity that cause limb hypertrophy or loss of function Painful lesions
- Complete resection of a PN with acceptable morbidity is not feasible OR patient refuses surgery OR the number of PNs leads to not feasible surgery according to the steering committee's site
Key exclusion criteria (15 listed)
- have previously received mTOR inhibitors
- a known hypersensitivity to RAD001 or other rapamycin or to its excipients
- receiving chronic systemic treatment with corticosteroids or another immunosuppressive agent. (Dose equivalent to 10 mg/day of methylprednisone), topical steroids or organotherapy for bilateral adrenalectomy are acceptable
- a known history of HIV seropositivity
- acute viral hepatitis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RAD001: Everolimus10 mg of RAD001 will be self-administered orally once daily continuously for one year or until unacceptable toxicity or discontinuation from the study from any other reason. | Drug | Everolimus |
Primary outcome
Radiographic response assessed by MRI analysis
Time frame: after 1 year of treatment
10 secondary outcomes
Radiographic response assessed by MRI analysis
Time frame: At 2 years
Overall survival
Time frame: At 2 years
Pain
Time frame: At 2 years
Deficiency
Time frame: At 2 years
Quality of life
Time frame: At 2 years
adverse events
Time frame: At 2 years
laboratory evaluations
Time frame: At 2 years
measurement of vital signs
Time frame: At 2 years
performance of physical examinations
Time frame: At 2 years
all concomitant medications and therapies
Time frame: At 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2014.
- Background: PubMed 18184959Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.
- Derived from this study: PubMed 30339775Zehou O, Ferkal S, Brugieres P, Barbarot S, Bastuji-Garin S, Combemale P, Valeyrie-Allanore L, Sbidian E, Wolkenstein P. Absence of Efficacy of Everolimus in Neurofibromatosis 1-Related Plexiform Neurofibromas: Results from a Phase 2a Trial. J Invest Dermatol. 2019 Mar;139(3):718-720. doi: 10.1016/j.jid.2018.09.016. Epub 2018 Oct 16. No abstract available.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pierre Wolkenstein, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RAD001: Everolimus in people with Neurofibromatosis Type 1 and Plexiform Neurofibroma, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Radiographic response assessed by MRI analysis (time frame: after 1 year of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01412892, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Nov 2014.