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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.
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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
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
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 | from the FSI date (21 Mar 2023) till 36 months from FSI |
| Time to discontinuation of selumetinib | from the FSI date (21 Mar 2023) till 36 months from FSI | |
| Description of diagnostic algorithms for PN NF1 in Russia | from the FSI date (21 Mar 2023) till 36 months from FSI | |
| Reasons for discontinuation of selumetinib | from the FSI date (21 Mar 2023) till 36 months from FSI |
| Measure | Description | Time Frame |
|---|---|---|
| Safety: adverse events (date, type, severe grade, seriousness criteria, outcome); | 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) |
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Inclusion Criteria:
Exclusion Criteria:
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Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Moscow | Moscow | 123112 | Russia | ||
| Research Site |
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| Label | URL |
|---|---|
| Redacted CSR Synopsis | View source |
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| ID | Term |
|---|---|
| D009455 | Neurofibroma |
| ID | Term |
|---|---|
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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n\a, as all procedures will be performed as routine clinical practice
| 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 | from the FSI date (21 Mar 2023) till 36 months from FSI |
| Clinical outcomes of selumetinib treatment | changes in PN-associated symptoms | From FSI date (21 Mar 2023) till 36 months from FSI |
| Moscow |
| Russia |
| Research Site | Novosibirsk | Russia |
| Research Site | Saint Petersburg | Russia |
| Research Site | Yekaterinburg | Russia |
| D010524 |
| Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009422 | Nervous System Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |