Clinical trial · Observational
Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
Non-interventional Study of Patients With Plexiform Neurofibromas and Neurofibromatosis Type I Starting Selumetinib in Russia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): 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.
Summary
Brief summary (as posted)
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
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neurofibroma | Neurofibroma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (0)
Data not yet available
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (5)
- measure
- ORR in patients receiving selumetinib according to REiNS criteria
- timeFrame
- from the FSI date (21 Mar 2023) till 36 months from FSI
- description
- 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.
- measure
- Best objective response on selumetinib treatment
- timeFrame
- from the FSI date (21 Mar 2023) till 36 months from FSI
- description
- a percentage of subjects who achieved a best response of CR, PR or stable disease during treatment
- measure
- Time to discontinuation of selumetinib
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Years
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * 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. Exclusion Criteria: * 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.
References
Publications (0)
Data not yet available