Neurofibromatosis Type 1NCT05683678
US Selumetinib Registry
An observational study of Neurofibromatosis Type 1 and Plexiform Neurofibromas, run by Alexion Pharmaceuticals, Inc. at 10 sites in United States.
Trial summary
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs)… Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Neurofibromatosis Type 1, Plexiform Neurofibromas
- How many people
- 37 participants
- Where
- 10 sitesUnited States
- Study dates
- 4 Jan 2019to 13 Nov 2024
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.
Who may be able to join
- Participants diagnosed with NF1 and PN
- Started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020, including patients who:
- Discontinued selumetinib before enrollment; or
- Initiated treatment before enrollment and are currently on selumetinib; or
- Initiated selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment
- Able to give written informed consent. Participant or participant's parent/legal guardian must be willing and able to give written informed consent and the participant (if minor) must be willing to give written informed assent.
Who cannot join
- Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Albany Medical Center
Albany, New York, 12208
Cook Children's Medical Center
Fort Worth, Texas, 76104
Children's Hospital Los Angeles
Los Angeles, California, 90027-6062
Nicklaus Children's Hospital
Miami, Florida, 33155
Ochsner Medical Center
New Orleans, Louisiana, 70121
Arnold Palmer Hospital For Children
Orlando, Florida, 32806
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Mayo Clinic
Rochester, Minnesota, 55905
Waters Center for Childrens Cancer and Blood Disorders
Syracuse, New York, 13210
The Children's Hospital at Montefiore (CHAM)
The Bronx, New York, 10467
About this study
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020 in a real-world setting.
Read the detailed description
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibromas
- Keywords
- neurofibromatosis type 1, plexiform neurofibromas, NF1, PNs
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform
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 |
|---|---|---|
| Cohort 1 | Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort). | |
| Cohort 2 | Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort). | |
| Cohort 3 | Participants who started selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment (the "initiating" cohort). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
Measured: Up to Month 60
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
Measured: Up to Month 60
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
Skeletal manifestations may include changes in the angle of spine curvature.
Measured: Up to Month 60
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
Measured: Up to Month 60
Secondary outcomes (4)
Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60
Measured: Baseline, Month 60
Change From Baseline in the Pain Interference Index (PII) Score at Month 60
Measured: Baseline, Month 60
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60
Measured: Baseline, Month 60
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60
Measured: Baseline, Month 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2023
- Study start4 Jan 2019
- Primary completion13 Nov 2024
- Study completion13 Nov 2024
Why it stopped: Sponsor decision.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The 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 only
- Time perspective
- Prospective
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05683678
- Sponsor's protocol ID
- ALX-NF1-501
- Status last verified
- Mar 2025
- First submitted
- 5 Jan 2023
- Last updated
- 4 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- US Selumetinib Registry
- ClinicalTrials.gov ID
- NCT05683678
- Acronym / sponsor ID
- ALX-NF1-501
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibromas
- Keywords
- neurofibromatosis type 1, plexiform neurofibromas, NF1, PNs
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor decision.
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Jan 2019 (actual)
- Primary completion
- 13 Nov 2024 (actual)
- Study completion
- 13 Nov 2024 (actual)
- First posted
- 13 Jan 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 understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020 in a real-world setting.
Detailed description
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | –Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort). |
| Cohort 2 | – | –Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort). |
| Cohort 3 | – | –Participants who started selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment (the "initiating" cohort). |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.
Key inclusion criteria (6 listed)
- Participants diagnosed with NF1 and PN
- Started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020, including patients who:
- Discontinued selumetinib before enrollment; or
- Initiated treatment before enrollment and are currently on selumetinib; or
- Initiated selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment
Key exclusion criteria (1 listed)
- Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.
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
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
Time frame: Up to Month 60
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
Time frame: Up to Month 60
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
Time frame: Up to Month 60
Skeletal manifestations may include changes in the angle of spine curvature.
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
Time frame: Up to Month 60
4 secondary outcomes
Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Pain Interference Index (PII) Score at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60
Time frame: Baseline, Month 60
Results and safety
The study was stopped early (reason given: Sponsor decision.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2025.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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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.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibromatosis Type 1 and Plexiform Neurofibromas, run by Alexion Pharmaceuticals, Inc. at 10 sites 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 6 inclusion and 1 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 4 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites 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?
The study was stopped early (reason given: Sponsor decision.). 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?
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician (time frame: Up to Month 60). The study lists 4 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 4 Apr 2025.