Neurofibromatosis Type 1NCT05388370
PASS of Paediatric Patients Initiating Selumetinib
An observational study of Neurofibromatosis Type 1, run by AstraZeneca at 46 sites in 10 countries.
Trial summary
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Neurofibromatosis Type 1
- How many people
- 124 participants
- Where
- 46 sitesin 10 countries
- Study dates
- 23 May 2022to 23 May 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 17 years
- All sexes
- No healthy volunteers
- Child
Study population: The target population for this study are patients with NF1 with symptomatic, inoperable PN who have been prescribed at least 1 dose of selumetinib and who are aged 3 to < 18 years at the start of selumetinib treatment, except for those patients receiving treatment with a mitogen-activated protein kinase inhibitor before the index date.
Who may be able to join
- Have been diagnosed with NF1 with symptomatic, inoperable PN
- Have initial treatment with selumetinib up to 6 months (i.e.182 days)prior to enrolment into the study (i.e. signature of the ICF)
- Are aged 3 years and above, and are < 18 years of age on the index date
- Parent or legal guardian, as required by country-specific regulation, have provided informed consent (unless a country-specific waiver is obtained) Additional Criteria for Nested Prospective Cohort
- Are at least 8 years old and
- Are prior to attainment of Tanner Stage V on the index date
Who cannot join
- Have received treatment with a mitogen-activated protein kinase inhibitor before the index date
- Are participating in an interventional study at index date
Trial locations and contacts
46 sites in 10 countries.
46 sites in 10 countries
France13 sites
Research Site
Amiens
Research Site
Angers
Research Site
Bordeaux
Research Site
Lille
Research Site
Lyon
Research Site
Marseille
Research Site
Paris
Research Site
Rennes
Research Site
Strasbourg
Research Site
Toulouse
Research Site
Tours
Research Site
Vandœuvre-lès-Nancy
Research Site
Villejuif
Italy8 sites
Research Site
Florence
Research Site
Genova
Research Site
Milan
Research Site
Padova
Research Site
Pavia
Research Site
Roma
Research Site
Torino
Research Site
Trieste
Germany6 sites
Research Site
Dresden
Research Site
Duisburg
Research Site
Hamburg
Research Site
Hanover
Research Site
München
Research Site
Tübingen
Spain5 sites
Research Site
Barcelona
Research Site
Madrid
Research Site
Málaga
Research Site
Santiago de Compostela
Research Site
Seville
Switzerland4 sites
Research Site
Basel
Research Site
Bern
Research Site
Lausanne
Research Site
Sankt Gallen
Israel3 sites
Research Site
Petah Tikva
Research Site
Ramat Gan
Research Site
Tel Aviv
United Kingdom3 sites
Research Site
London
Research Site
Manchester
Research Site
Newcastle upon Tyne
About this study
Official title: Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study.
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1. Plexiform neurofibromas (PN) are histologically benign nerve sheath tumours, which typically grow along large nerves and plexi.
On 5 March 2020, a centralised Marketing Authorisation Application was submitted to the European Medicines Agency (EMA), Marketing Authorisation in EU was granted on 17 Jun 2021.
Read more
As part of the approval process, a Risk Management Plan (RMP) was developed and submitted to the EMA to summarise the safety concerns emerging from the clinical development program. The RMP included additional pharmacovigilance plans for a noninterventional Post-authorisation Safety Study (PASS) to further characterise the safety of selumetinib in paediatric patients with NF1-related PN in routine clinical practice.
The planned non-interventional PASS will address gaps in knowledge identified by the RMP, including the important identified risk and some of the potential risks and missing information on long-term developmental toxicity in children, by characterising the safety profile associated with selumetinib use among paediatric patients (age d 8 to < 18 years old) with a diagnosis of NF1 with symptomatic, inoperable PN.
This study is a specific obligation in the context of a conditional marketing authorisation for selumetinib (ie, Category 2 PASS). Study results will contribute to updating the safety profile of selumetinib in a relatively large population of patients with different personal characteristics across multiple health care systems and patterns of real-world clinical practice in European countries and Israel.
The study will enrol 2 cohorts:
- The Base Cohort includes all enrolled patients aged 3 to < 18 years.
- The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date.
Read the detailed description
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1. Plexiform neurofibromas (PN) are histologically benign nerve sheath tumours, which typically grow along large nerves and plexi.
On 5 March 2020, a centralized Marketing Authorisation Application was submitted to the European Medicines Agency (EMA), Marketing Authorization in EU was granted on 17 Jun 2021.
As part of the approval process, a Risk Management Plan (RMP) was developed and submitted to the EMA to summarise the safety concerns emerging from the clinical development program. The RMP included additional pharmacovigilance plans for a non-interventional Post-authorisation Safety Study (PASS) to further characterise the safety of selumetinib in paediatric patients with NF1-related PN in routine clinical practice.
The RMP version 1.0 (succession 4) approved by EMA on 22 April 2021 had 1 important identified risk with selumetinib treatment:
-LVEF reduction
The RMP also identified 5 important potential risks with selumetinib treatment:
- Physeal dysplasia
- Ocular toxicity
- Myopathy
- Hepatotoxicity
- Choking on the capsule Long-term exposure (including long-term safety data on developmental toxicity in children) was identified in the RMP as an area of missing information.
The planned non-interventional PASS will address gaps in knowledge identified by the RMP, including the important identified risk and some of the potential risks and missing information on long-term developmental toxicity in children, by characterising the safety profile associated with selumetinib use among paediatric patients (aged d 8 to < 18 years old) with a diagnosis of NF1 with symptomatic, inoperable PN.
This study is a specific obligation in the context of a conditional marketing authorisation for selumetinib (ie, Category 2 PASS). Study results will contribute to updating the safety profile of selumetinib in a relatively large population of patients with different personal characteristics across multiple health care systems and patterns of real-world clinical practice in up to 52 specialist clinics for the treatment of pediatric patients with NF1 across up to 12 European countries and in Israel.
The primary objective of this study is:
- To characterise the safety of selumetinib, including up to 6 years of long-term safety, in paediatric patients with NF1-related symptomatic, inoperable PN, 8 to < 18 years old who have not reached Tanner Stage V at the start of selumetinib treatment (Nested Prospective Cohort).
The secondary objective of this study is:
- To describe the demographic and clinical profile of the paediatric population 3 to < 18 years old with NF1-related symptomatic inoperable PN who start selumetinib in routine clinical practice (Base Cohort).
The study observation period was anticipated to begin in Q2 of 2022, with some variation by country (actual start date was 23 May 2022). Patients will be enrolled after selumetinib access is commercially available and patients are able to receive the medicine as part of local clinical practice.
The target population for this study are patients with NF1 in the EU with symptomatic, inoperable PN who have been prescribed at least 1 dose of selumetinib and who are aged 3 to < 18 years at the start of selumetinib treatment, except for those patients receiving treatment with a mitogen-activated protein kinase inhibitor before the index date.
The study will enrol 2 cohorts:
- The Base Cohort includes all enrolled patients aged 3 to < 18 years.
- The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date.
Patient screening will be conducted throughout the enrolment period and baseline data for all patients will be abstracted from medical records. Those meeting the criteria for enrolment in the Nested Prospective Cohort will be followed up during their routine standard of care visits with the treating clinician (expected to occur every 6 to 12 months) for up to 6 years.
- Conditions
- Neurofibromatosis Type 1
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Base Cohort | The Base Cohort includes all enrolled patients aged 3 to < 18 years. | |
| Nested Prospective Cohort | The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date |
What is being measured
The primary outcome is the main question the study is designed to answer.
LVEF reduction
LVEF reduction will be detected as present or absent and when present if symptomatic or asymptomatic. All cardiac tests conducted will be collected Measured on routine echocardiogram or a cardiac MRI (CT, angiography, etc.) and then collected into the study from the medical records.
Measured: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
Occurrence of Physeal dysplasia after treatment start
Physeal dysplasia will be detected as present or absent based on the physician reading of: MRI: Knee (preferred) or wrist X-ray: Knee (preferred) and/or wrist to assess growth plate Height and weight records
Measured: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
Rise of serum creatine phosphokinase levels AND concurrent musculoskeletal symptoms
A clinically meaningful rise in serum creatine phosphokinase (eg, above the normal limit or increase by 1 or more CTCAE grade shift) combined with musculoskeletal symptoms will be detected as present or absent based on the physician's reading, as a marker of potential myopathy
Measured: at routine clinical care throughout the follow up, with frequency of 6 to 12 months, up to 6 years
Rise in transaminase (ALT and AST) and concurrent rise in bilirubin
A clinically meaningful rise in the measured levels (eg, above the normal limit or increase by 1 or more CTCAE grade shift) will be detected as present or absent, and when present if symptomatic or asymptomatic, as a marker of potential hepatotoxicity
Measured: at routine clinical care throughout the follow up, with frequency of 6 to 12 months, up to 6 years
Cumulative incidence of ocular toxicity
An abnormal ocular examination will be detected as present or absent based on the physician's reading, as a marker of potential ocular toxicity
Measured: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
Cumulative incidence of Abnormal pubertal development
Tanner stage criteria (Stages I-V). Abnormal pubertal development will require interpretation by the Investigator with respect to Tanner Stage in the context of the patient's age; recorded as normal or abnormal (if abnormal, further specified as delayed puberty or precocious puberty)
Measured: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
Secondary outcomes (9)
baseline data - demographics
Demographics: Age
Measured: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
sex
Measured: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
height (cm)
Measured: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
weight (kg)
Measured: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Tanner staging level
Measured: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Ethnicity (where allowed by GDPR/privacy laws)
Measured: At baseline - most recent assessments made within 365 days before the index date
Baseline data - Clinical characteristics
PN(s) (number, location, classification and morbidities)
Measured: At baseline - most recent assessments made within 365 days before the index date
Baseline data - Clinical characteristics
prior medication and relevant procedures, concomitant medications
Measured: At baseline - most recent assessments made within 365 days before the index date
Baseline data - Clinical characteristics
date of initial NF1 and PN diagnosis, NF1 origin (familial or spontaneous), and any genetic testing results
Measured: At baseline - most recent assessments made within 365 days before the index date
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2022
- Study start23 May 2022
- Primary completion23 May 2028 (estimated)
- Study completion23 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)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 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 124 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05388370
- Sponsor's protocol ID
- D1346R00004
- Status last verified
- Aug 2026
- First submitted
- 12 Apr 2022
- Last updated
- 24 Aug 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The study protocol, study progress reports, and final study report will be included in regulatory communications in line with the RMP, PSUR, and other regulatory reporting requirements. Study reports will be prepared using a template following the GVP Module VIII Section B.6.3.
Publications
EMA2017b EMA. Guideline on good pharmacovigilance practices (GVP). Module VIII - Post-authorisation safety studies (EMA/813938/2011 Rev 3). European Medicines Agency; 09 October 2017b. Available at: https://www.ema.europa.eu/documents/scientificguideline/ guideline-good-pharmacovigilance-practices-gvp-
International Committee Of Medical Journal Editors. [Recommendations for the conduct, reporting, editing and publication of scholarly work in medical journals (revised in January 2024): a Korean translation]. Ewha Med J. 2024 Oct;47(4):e48. doi: 10.12771/emj.2024.e48. Epub 2024 Oct 31. No abstract available. Korean.
PubMed 40704003ISPE. Guidelines for good pharmacoepidemiology practices (GPP). Pharmacoepidemiol Drug Saf. 2008 Feb;17(2):200-8. doi: 10.1002/pds.1471. No abstract available.
PubMed 17868186KOSELUGO (selumetinib) KOSELUGO (selumetinib) capsules, for oral use, initial US Approval: 2020. Distributed by: AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850. USPI revised April 2020, Reference ID 4590044.
von Elm E, Altman DG, Egger M, Pocock SJ, Gotzsche PC, Vandenbroucke JP; STROBE Initiative. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies. Lancet. 2007 Oct 20;370(9596):1453-7. doi: 10.1016/S0140-6736(07)61602-X.
PubMed 18064739
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study.
- ClinicalTrials.gov ID
- NCT05388370
- Acronym / sponsor ID
- D1346R00004
- Conditions
- Neurofibromatosis Type 1
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 124 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 23 May 2022 (actual)
- Primary completion
- 23 May 2028 (estimated)
- Study completion
- 23 May 2028 (estimated)
- First posted
- 24 May 2022 (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.
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1. Plexiform neurofibromas (PN) are histologically benign nerve sheath tumours, which typically grow along large nerves and plexi.
On 5 March 2020, a centralised Marketing Authorisation Application was submitted to the European Medicines Agency (EMA), Marketing Authorisation in EU was granted on 17 Jun 2021.
As part of the approval process, a Risk Management Plan (RMP) was developed and submitted to the EMA to summarise the safety concerns emerging from the clinical development program. The RMP included additional pharmacovigilance plans for a noninterventional Post-authorisation Safety Study (PASS) to further characterise the safety of selumetinib in paediatric patients with NF1-related PN in routine clinical practice.
The planned non-interventional PASS will address gaps in knowledge identified by the RMP, including the important identified risk and some of the potential risks and missing information on long-term developmental toxicity in children, by characterising the safety profile associated with selumetinib use among paediatric patients (age d 8 to < 18 years old) with a diagnosis of NF1 with symptomatic, inoperable PN.
This study is a specific obligation in the context of a conditional marketing authorisation for selumetinib (ie, Category 2 PASS). Study results will contribute to updating the safety profile of selumetinib in a relatively large population of patients with different personal characteristics across multiple health care systems and patterns of real-world clinical practice in European countries and Israel.
The study will enrol 2 cohorts:
- The Base Cohort includes all enrolled patients aged 3 to < 18 years.
- The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date.
Detailed description
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1. Plexiform neurofibromas (PN) are histologically benign nerve sheath tumours, which typically grow along large nerves and plexi.
On 5 March 2020, a centralized Marketing Authorisation Application was submitted to the European Medicines Agency (EMA), Marketing Authorization in EU was granted on 17 Jun 2021.
As part of the approval process, a Risk Management Plan (RMP) was developed and submitted to the EMA to summarise the safety concerns emerging from the clinical development program. The RMP included additional pharmacovigilance plans for a non-interventional Post-authorisation Safety Study (PASS) to further characterise the safety of selumetinib in paediatric patients with NF1-related PN in routine clinical practice.
The RMP version 1.0 (succession 4) approved by EMA on 22 April 2021 had 1 important identified risk with selumetinib treatment:
-LVEF reduction
The RMP also identified 5 important potential risks with selumetinib treatment:
- Physeal dysplasia
- Ocular toxicity
- Myopathy
- Hepatotoxicity
- Choking on the capsule Long-term exposure (including long-term safety data on developmental toxicity in children) was identified in the RMP as an area of missing information.
The planned non-interventional PASS will address gaps in knowledge identified by the RMP, including the important identified risk and some of the potential risks and missing information on long-term developmental toxicity in children, by characterising the safety profile associated with selumetinib use among paediatric patients (aged d 8 to < 18 years old) with a diagnosis of NF1 with symptomatic, inoperable PN.
This study is a specific obligation in the context of a conditional marketing authorisation for selumetinib (ie, Category 2 PASS). Study results will contribute to updating the safety profile of selumetinib in a relatively large population of patients with different personal characteristics across multiple health care systems and patterns of real-world clinical practice in up to 52 specialist clinics for the treatment of pediatric patients with NF1 across up to 12 European countries and in Israel.
The primary objective of this study is:
- To characterise the safety of selumetinib, including up to 6 years of long-term safety, in paediatric patients with NF1-related symptomatic, inoperable PN, 8 to < 18 years old who have not reached Tanner Stage V at the start of selumetinib treatment (Nested Prospective Cohort).
The secondary objective of this study is:
- To describe the demographic and clinical profile of the paediatric population 3 to < 18 years old with NF1-related symptomatic inoperable PN who start selumetinib in routine clinical practice (Base Cohort).
The study observation period was anticipated to begin in Q2 of 2022, with some variation by country (actual start date was 23 May 2022). Patients will be enrolled after selumetinib access is commercially available and patients are able to receive the medicine as part of local clinical practice.
The target population for this study are patients with NF1 in the EU with symptomatic, inoperable PN who have been prescribed at least 1 dose of selumetinib and who are aged 3 to < 18 years at the start of selumetinib treatment, except for those patients receiving treatment with a mitogen-activated protein kinase inhibitor before the index date.
The study will enrol 2 cohorts:
- The Base Cohort includes all enrolled patients aged 3 to < 18 years.
- The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date.
Patient screening will be conducted throughout the enrolment period and baseline data for all patients will be abstracted from medical records. Those meeting the criteria for enrolment in the Nested Prospective Cohort will be followed up during their routine standard of care visits with the treating clinician (expected to occur every 6 to 12 months) for up to 6 years.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Base Cohort | – | –The Base Cohort includes all enrolled patients aged 3 to < 18 years. |
| Nested Prospective Cohort | – | –The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- The target population for this study are patients with NF1 with symptomatic, inoperable PN who have been prescribed at least 1 dose of selumetinib and who are aged 3 to < 18 years at the start of selumetinib treatment, except for those patients receiving treatment with a mitogen-activated protein kinase inhibitor before the index date.
Key inclusion criteria (6 listed)
- Have been diagnosed with NF1 with symptomatic, inoperable PN
- Have initial treatment with selumetinib up to 6 months (i.e.182 days)prior to enrolment into the study (i.e. signature of the ICF)
- Are aged 3 years and above, and are < 18 years of age on the index date
- Parent or legal guardian, as required by country-specific regulation, have provided informed consent (unless a country-specific waiver is obtained) Additional Criteria for Nested Prospective Cohort
- Are at least 8 years old and
Key exclusion criteria (2 listed)
- Have received treatment with a mitogen-activated protein kinase inhibitor before the index date
- Are participating in an interventional study at index date
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
LVEF reduction
Time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
LVEF reduction will be detected as present or absent and when present if symptomatic or asymptomatic. All cardiac tests conducted will be collected Measured on routine echocardiogram or a cardiac MRI (CT, angiography, etc.) and then collected into the study from the medical records.
Occurrence of Physeal dysplasia after treatment start
Time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
Physeal dysplasia will be detected as present or absent based on the physician reading of: MRI: Knee (preferred) or wrist X-ray: Knee (preferred) and/or wrist to assess growth plate Height and weight records
Rise of serum creatine phosphokinase levels AND concurrent musculoskeletal symptoms
Time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months, up to 6 years
A clinically meaningful rise in serum creatine phosphokinase (eg, above the normal limit or increase by 1 or more CTCAE grade shift) combined with musculoskeletal symptoms will be detected as present or absent based on the physician's reading, as a marker of potential myopathy
Rise in transaminase (ALT and AST) and concurrent rise in bilirubin
Time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months, up to 6 years
A clinically meaningful rise in the measured levels (eg, above the normal limit or increase by 1 or more CTCAE grade shift) will be detected as present or absent, and when present if symptomatic or asymptomatic, as a marker of potential hepatotoxicity
Cumulative incidence of ocular toxicity
Time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
An abnormal ocular examination will be detected as present or absent based on the physician's reading, as a marker of potential ocular toxicity
Cumulative incidence of Abnormal pubertal development
Time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months
Tanner stage criteria (Stages I-V). Abnormal pubertal development will require interpretation by the Investigator with respect to Tanner Stage in the context of the patient's age; recorded as normal or abnormal (if abnormal, further specified as delayed puberty or precocious puberty)
9 secondary outcomes
baseline data - demographics
Time frame: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Time frame: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Time frame: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Time frame: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Time frame: At baseline - most recent assessments made within 365 days before the index date
baseline data - demographics
Time frame: At baseline - most recent assessments made within 365 days before the index date
Baseline data - Clinical characteristics
Time frame: At baseline - most recent assessments made within 365 days before the index date
Baseline data - Clinical characteristics
Time frame: At baseline - most recent assessments made within 365 days before the index date
Baseline data - Clinical characteristics
Time frame: At baseline - most recent assessments made within 365 days before the index date
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2026.
- BackgroundEMA2017b EMA. Guideline on good pharmacovigilance practices (GVP). Module VIII - Post-authorisation safety studies (EMA/813938/2011 Rev 3). European Medicines Agency; 09 October 2017b. Available at: https://www.ema.europa.eu/documents/scientificguideline/ guideline-good-pharmacovigilance-practices-gvp-
- Background: PubMed 40704003International Committee Of Medical Journal Editors. [Recommendations for the conduct, reporting, editing and publication of scholarly work in medical journals (revised in January 2024): a Korean translation]. Ewha Med J. 2024 Oct;47(4):e48. doi: 10.12771/emj.2024.e48. Epub 2024 Oct 31. No abstract available. Korean.
- Background: PubMed 17868186ISPE. Guidelines for good pharmacoepidemiology practices (GPP). Pharmacoepidemiol Drug Saf. 2008 Feb;17(2):200-8. doi: 10.1002/pds.1471. No abstract available.
- BackgroundKOSELUGO (selumetinib) KOSELUGO (selumetinib) capsules, for oral use, initial US Approval: 2020. Distributed by: AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850. USPI revised April 2020, Reference ID 4590044.
- Background: PubMed 18064739von Elm E, Altman DG, Egger M, Pocock SJ, Gotzsche PC, Vandenbroucke JP; STROBE Initiative. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies. Lancet. 2007 Oct 20;370(9596):1453-7. doi: 10.1016/S0140-6736(07)61602-X.
- Individual participant dataSharing planned: Yes. The study protocol, study progress reports, and final study report will be included in regulatory communications in line with the RMP, PSUR, and other regulatory reporting requirements. Study reports will be prepared using a template following the GVP Module VIII Section B.6.3.
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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.
The record lists no contact. Use the official record to reach the sponsor.
46 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibromatosis Type 1, run by AstraZeneca at 46 sites in 10 countries.
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 6 inclusion and 2 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: under way, not looking for participants, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in France, Italy, Germany, Spain, Switzerland and 5 more countries. 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?
This study is still under way. Results are not available in the source record yet.
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?
LVEF reduction (time frame: at routine clinical care throughout the follow up, with frequency of 6 to 12 months). The study lists 6 primary outcomes in all. 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 NCT05388370, which also lists 5 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 24 Aug 2026.