NF type1 With Inoperable Plexiform NeurofibromasNCT03259633
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
A study testing Selumetinib in people with NF type1 With Inoperable Plexiform Neurofibromas, run by AstraZeneca at 1 site in United States.
Trial summary
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. Read more about this study
- Who is it for?
- Ages 2 to 130 years
- Conditions the study is for
- NF type1 With Inoperable Plexiform Neurofibromas
- What is being tested?
- Selumetinib
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 2 to 130 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of neurofibromatosis type 1 (NF1) and have inoperable, progressive/symptomatic plexiform neurofibromas (PN)
- Presence of inoperable PN , defined as a PN that cannot be surgically completely removed without risk for substantial morbidity
- Patients aged ≥2 with onset of disease before they were 18 years and a BSA ≥ 0.55 m2 who are able to swallow whole capsules. (approx. length 15.4 mm, diameter 5.4 mm). A swallow test must be performed before requesting drug
- Normal cardiac function defined as normal ejection fraction (ECHO, MUGA or cardiac MRI) as per institutional normal and absence of prior heart disease
- Adequate blood pressure as defined in line with local practice.
- The patient has exhausted all available approved therapies as appropriate for NF1 with inoperable progressive/symptomatic PN
- Provision of a signed informed consent prior to any protocol specific procedures. Patients already receiving selumetinib through single patient access who enroll in this protocol must be reconsented and sign the consent form for this intermediate access protocol.
- For female patients of childbearing potential, have evidence of a post-menopausal status, or a negative urinary or serum pregnancy test.
Who cannot join
- Unresolved chronic toxicity ≥ CTCAE Grade 2 from previous therapy
- Patients eligible for any ongoing clinical trials with selumetinib in the indication in question
- Ophthalmological conditions: Current or past history of retinal pigment epithelial detachment (RPED)/central serous retinopathy (CSR) or retinal vein occlusion Intraocular pressure (IOP) should not be > 21 mmHg for adults or outside the range of normal for children or uncontrolled glaucoma (irrespective of IOP)
- Male or female patients of reproductive potential and, as judged by the investigator, are not employing an effective method of birth control.
- Female patients who are breast-feeding.
- Have evidence of any other significant clinical disorder or laboratory finding that, as judged by the treating physician, makes it undesirable for the patient to participate in the study.
- Have any evidence of a severe or uncontrolled systemic disease (e.g. unstable or uncompensated respiratory, cardiac, hepatic, or renal disease, active infection (including hepatitis B, hepatitis C, HIV), active bleeding diatheses or renal transplant
- Have refractory nausea and vomiting, chronic gastrointestinal diseases (e.g., inflammatory bowel disease), or significant bowel resection that would adversely affect the absorption / bioavailability of the orally administered study medication
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit.
Approximately 100 patients in the US will be treated as part of this protocol
Read the detailed description
Patients must have received a clinical diagnosis of NF1 and have inoperable, progressive/symptomatic PN, where inoperable is defined as PN that cannot be surgically completely removed without risk of substantial morbidity.
The population are patients with NF1 who have inoperable, progressive/symptomatic PN aged ≥ 2years with onset of disease before they were 18 years and who have demonstrated an ability to swallow whole capsules, who have no further treatment options and are not eligible for clinical trials.
There is no maximum duration for selumetinib treatment. Patients may continue to receive selumetinib as long as they continue to show clinical benefit, as judged by the treating physician, and in the absence of unacceptable toxicity.
Once patients have been discontinued from treatment, other available treatment options will be at the discretion of the physician
- Being tested
- Selumetinib
- Keywords
- Neurofibromatosis type1,, Plexiform Neurofibromas, inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options, mutation in the NF1 gene, Selumetinib, Early Access, Intermediate Access Protocol
- Medical subject headings
- Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelumetinibDrug
open-label, single-arm, multicenter intermediate access protocol
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NF type1 With Inoperable Plexiform NeurofibromasStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Miriam Bornhorst, MD, Principal investigator, Investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03259633
- Sponsor's protocol ID
- D1346R00002
- Other IDs
- D1346R00002
- Status last verified
- Apr 2020
- First submitted
- 21 Aug 2017
- Last updated
- 1 May 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)
- ClinicalTrials.gov ID
- NCT03259633
- Acronym / sponsor ID
- D1346R00002
- Keywords
- Neurofibromatosis type1,, Plexiform Neurofibromas, inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options, mutation in the NF1 gene, Selumetinib, Early Access, Intermediate Access Protocol
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- First posted
- 24 Aug 2017 (actual)
- 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.
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit.
Approximately 100 patients in the US will be treated as part of this protocol
Detailed description
Patients must have received a clinical diagnosis of NF1 and have inoperable, progressive/symptomatic PN, where inoperable is defined as PN that cannot be surgically completely removed without risk of substantial morbidity.
The population are patients with NF1 who have inoperable, progressive/symptomatic PN aged ≥ 2years with onset of disease before they were 18 years and who have demonstrated an ability to swallow whole capsules, who have no further treatment options and are not eligible for clinical trials.
There is no maximum duration for selumetinib treatment. Patients may continue to receive selumetinib as long as they continue to show clinical benefit, as judged by the treating physician, and in the absence of unacceptable toxicity.
Once patients have been discontinued from treatment, other available treatment options will be at the discretion of the physician
Study design
Who may qualify
- Ages
- 2 to 130 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Diagnosis of neurofibromatosis type 1 (NF1) and have inoperable, progressive/symptomatic plexiform neurofibromas (PN)
- Presence of inoperable PN , defined as a PN that cannot be surgically completely removed without risk for substantial morbidity
- Patients aged ≥2 with onset of disease before they were 18 years and a BSA ≥ 0.55 m2 who are able to swallow whole capsules. (approx. length 15.4 mm, diameter 5.4 mm). A swallow test must be performed before requesting drug
- Normal cardiac function defined as normal ejection fraction (ECHO, MUGA or cardiac MRI) as per institutional normal and absence of prior heart disease
- Adequate blood pressure as defined in line with local practice.
Key exclusion criteria (8 listed)
- Unresolved chronic toxicity ≥ CTCAE Grade 2 from previous therapy
- Patients eligible for any ongoing clinical trials with selumetinib in the indication in question
- Ophthalmological conditions: Current or past history of retinal pigment epithelial detachment (RPED)/central serous retinopathy (CSR) or retinal vein occlusion Intraocular pressure (IOP) should not be > 21 mmHg for adults or outside the range of normal for children or uncontrolled glaucoma (irrespective of IOP)
- Male or female patients of reproductive potential and, as judged by the investigator, are not employing an effective method of birth control.
- Female patients who are breast-feeding.
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 |
|---|---|---|
| Selumetinibopen-label, single-arm, multicenter intermediate access protocol | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2020.
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.
Miriam Bornhorst, MD principal investigator
Investigator
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Selumetinib in people with NF type1 With Inoperable Plexiform Neurofibromas, run by AstraZeneca at 1 site in United States.
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 8 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 1 May 2020. 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 United States. 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.
Are results available for this study?
Results are not available in the source record.
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.
Where can I read the original study information?
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 1 May 2020.