US Selumetinib Registry

Neurofibromatosis Type 1 Clinical Trial

Official title:

US Selumetinib Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05683678
• Other study ID #: ALX-NF1-501
• Status: Recruiting
• Start date: July 18, 2023
• Est. completion date: June 21, 2028

Study information

• Verified date: July 2023
• Source: Alexion
• Contact: Alexion Pharmaceuticals, Inc.
• Phone: 1-855-752-2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Observational

Clinical 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 participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.

Description:

This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: June 21, 2028
• Est. primary completion date: February 6, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 18 Years

Eligibility

Inclusion Criteria:

• Participants diagnosed with NF1 and PN who were or will be aged 2 to 18 years at the time of selumetinib initiation on or after 10 April 2020 including participants who:

• Discontinued selumetinib before enrollment; or

• Initiated treatment before enrollment and are currently on selumetinib; or

• Intend to initiate selumetinib within approximately 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.

Exclusion Criteria:

• Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibroma, Plexiform
• Neurofibromatoses
• Neurofibromatosis 1
• Neurofibromatosis Type 1
• Plexiform Neurofibromas

Locations

Country	Name	City	State
United States	Clinical Trial Site	Albany	New York

Sponsors (1)

Lead Sponsor	Collaborator
Alexion

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician		Up to Month 60
Primary	Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician		Up to Month 60
Primary	Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations	Skeletal manifestations may include changes in the angle of spine curvature.	Up to Month 60
Primary	Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)		Up to Month 60
Secondary	Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60		Baseline, Month 60
Secondary	Change From Baseline in the Pain Interference Index (PII) Score at Month 60		Baseline, Month 60
Secondary	Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60		Baseline, Month 60
Secondary	Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60		Baseline, Month 60