NeurofibromaNCT05891847
Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
An observational study of Neurofibroma, run by AstraZeneca at 5 sites in Russia.
Trial summary
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Neurofibroma
- How many people
- 150 participants
- Where
- 5 sitesRussia
- Study dates
- 21 Mar 2023to 11 Jul 2025
- Phase
- Observational
- 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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
Who may be able to join
- NF1 diagnosed (according to the criteria for evaluating NF1),
- Symptomatic inoperable PN,
- Patients receiving therapy with selumetinib. For newly treated patients - registration on the first day of starting treatment with selumetinib,
- Age at starting of selumetinib treatment ≥ 3 to ≤ 18 years old (included in the study or started the therapy at the age of 18 years old),
- Patients whose parents or guardians have signed a consent form.
Who cannot join
- Patients currently participating in an interventional study/clinical trial,
- Patients are excluded if there is evidence of an optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy,
- Patients who receive other antitumor treatment options on optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer. If the patient on selumetinib treatment develop optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer, he can continue selumetinib therapy +- other treatment options on physician decision,
- Female patients above 12 age who are pregnant /plan to become pregnant or at breast feeding.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Russia5 sites
Research Site
Moscow, Moscow, 123112
Research Site
Moscow
Research Site
Novosibirsk
Research Site
Saint Petersburg
Research Site
Yekaterinburg
About this study
Official title: Non-interventional Study of Patients With Plexiform Neurofibromas and Neurofibromatosis Type I Starting Selumetinib in Russia
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
Read the detailed description
This study will be non-interventional retrospective and prospective study. Assessment of parameters will be carried out as if a patient is treated in real-life clinical setting. The patients should be enrolled into this project after evaluation of eligibility criteria by an investigator 10 clinical sites. No additional procedures besides those already used in the routine clinical practice will be applied to the patients. Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
- Conditions
- Neurofibroma
- Keywords
- NON-INTERVENTIONAL, Selumetinib
- Medical subject headings
- Neurofibroma
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR in patients receiving selumetinib according to REiNS criteria
ORR (Overall response rate) in patients receiving selumetinib according to REiNS criteria:
ORR is defined as the number of patients with CR (complete response) or cPR (confirmed partial response). CR is defined as disappearance of the target lesion. PR is defined as decrease in the volume of the target PN by 20% or more compared to the baseline and is considered unconfirmed at the first detection, confirmed when observed again within 3-6 months, and durable when the response is maintained for 1 year or longer
In this trial ORR could be taken from the source records of the patient retrospectively. In this case CR or cPR counted from the therapy start date of a patient, which could have been before ICF signing.
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Best objective response on selumetinib treatment
a percentage of subjects who achieved a best response of CR, PR or stable disease during treatment
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Time to discontinuation of selumetinib
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Description of diagnostic algorithms for PN NF1 in Russia
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Reasons for discontinuation of selumetinib
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Secondary outcomes (4)
Safety: adverse events (date, type, severe grade, seriousness criteria, outcome);
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Clinical profile of patients with PN NF1 in Russia
features of molecular diagnostics, lesion volume and localization of PN (plexiform neurofibroma)
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Demographic profile of patients with PN NF1 in Russia
age, gender of patients
Measured: from the FSI date (21 Mar 2023) till 36 months from FSI
Clinical outcomes of selumetinib treatment
changes in PN-associated symptoms
Measured: From FSI date (21 Mar 2023) till 36 months from FSI
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2023
- Study start21 Mar 2023
- Primary completion11 Jul 2025
- Study completion11 Jul 2025
Why it stopped: Statistically significant failure to achieve key secondary endpoints. The current data do not demonstrate sufficient potential to achieve the planned scientific objectives. The need to optimize the allocation of company resources.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeurofibromaStudies 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
- Observational
- Observational model
- Case-control
- Time perspective
- Other
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05891847
- Sponsor's protocol ID
- D1340R00002
- Status last verified
- Jun 2026
- First submitted
- 21 Mar 2023
- Last updated
- 8 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-interventional Study of Patients With Plexiform Neurofibromas and Neurofibromatosis Type I Starting Selumetinib in Russia
- ClinicalTrials.gov ID
- NCT05891847
- Acronym / sponsor ID
- D1340R00002
- Conditions
- Neurofibroma
- Keywords
- NON-INTERVENTIONAL, Selumetinib
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Statistically significant failure to achieve key secondary endpoints. The current data do not demonstrate sufficient potential to achieve the planned scientific objectives. The need to optimize the allocation of company resources.
- Enrolment
- 150 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 21 Mar 2023 (actual)
- Primary completion
- 11 Jul 2025 (actual)
- Study completion
- 11 Jul 2025 (actual)
- First posted
- 7 Jun 2023 (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.
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
Detailed description
This study will be non-interventional retrospective and prospective study. Assessment of parameters will be carried out as if a patient is treated in real-life clinical setting. The patients should be enrolled into this project after evaluation of eligibility criteria by an investigator 10 clinical sites. No additional procedures besides those already used in the routine clinical practice will be applied to the patients. Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
Study design
- Observational model
- Case-control
- Time perspective
- Other
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
Key inclusion criteria (5 listed)
- NF1 diagnosed (according to the criteria for evaluating NF1),
- Symptomatic inoperable PN,
- Patients receiving therapy with selumetinib. For newly treated patients - registration on the first day of starting treatment with selumetinib,
- Age at starting of selumetinib treatment ≥ 3 to ≤ 18 years old (included in the study or started the therapy at the age of 18 years old),
- Patients whose parents or guardians have signed a consent form.
Key exclusion criteria (4 listed)
- Patients currently participating in an interventional study/clinical trial,
- Patients are excluded if there is evidence of an optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy,
- Patients who receive other antitumor treatment options on optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer. If the patient on selumetinib treatment develop optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer, he can continue selumetinib therapy +- other treatment options on physician decision,
- Female patients above 12 age who are pregnant /plan to become pregnant or at 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.
Primary outcome
ORR in patients receiving selumetinib according to REiNS criteria
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
ORR (Overall response rate) in patients receiving selumetinib according to REiNS criteria: ORR is defined as the number of patients with CR (complete response) or cPR (confirmed partial response). CR is defined as disappearance of the target lesion. PR is defined as decrease in the volume of the target PN by 20% or more compared to the baseline and is considered unconfirmed at the first detection, confirmed when observed again within 3-6 months, and durable when the response is maintained for 1 year or longer In this trial ORR could be taken from the source records of the patient retrospectively. In this case CR or cPR counted from the therapy start date of a patient, which could have been before ICF signing.
Best objective response on selumetinib treatment
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
a percentage of subjects who achieved a best response of CR, PR or stable disease during treatment
Time to discontinuation of selumetinib
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
Description of diagnostic algorithms for PN NF1 in Russia
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
Reasons for discontinuation of selumetinib
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
4 secondary outcomes
Safety: adverse events (date, type, severe grade, seriousness criteria, outcome);
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
Clinical profile of patients with PN NF1 in Russia
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
Demographic profile of patients with PN NF1 in Russia
Time frame: from the FSI date (21 Mar 2023) till 36 months from FSI
Clinical outcomes of selumetinib treatment
Time frame: From FSI date (21 Mar 2023) till 36 months from FSI
Results and safety
The study was stopped early (reason given: Statistically significant failure to achieve key secondary endpoints. The current data do not demonstrate sufficient potential to achieve the planned scientific objectives. The need to optimize the allocation of company resources.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2026.
- Redacted CSR SynopsisLink provided by the sponsor.
Related trials for Neurofibroma
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms
RecruitingPhase 1
Metarrestin (ML-246) in Subjects With Metastatic Solid Tumors
Advanced Solid Tumors, Metastatic Pancreatic Cancer
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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibroma, run by AstraZeneca at 5 sites in Russia.
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 5 inclusion and 4 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: stopped early, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Russia. 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?
The study was stopped early (reason given: Statistically significant failure to achieve key secondary endpoints. The current data do not demonstrate sufficient potential to achieve the planned scientific objectives. The need to optimize the allocation of company resources.). 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.
What does the primary outcome measure?
ORR in patients receiving selumetinib according to REiNS criteria (time frame: from the FSI date (21 Mar 2023) till 36 months from FSI). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 8 Jul 2026.