Individual Patient Compassionate Use of Mirdametinib
Primary Purpose
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Mirdametinib (MEK Inhibitor)
Sponsored by
About this trial
This is an expanded access trial for Neurofibromatosis Type 1-Associated Plexiform Neurofibromas focused on measuring RASopathies, NF1, Neurofibromatosis, Plexiform Neurofibromas, Langerhans
Eligibility Criteria
Inclusion Criteria:
- Patient has a serious, debilitating or life-threatening medical condition with no suitable treatment available, has exhausted current standard of care or declined medically appropriate treatment for ethical or medical reasons.
- Patient does not qualify to participate in any ongoing clinical trial sponsored by SpringWorks in an accessible geographical location.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT05028166
First Posted
August 24, 2021
Last Updated
August 30, 2023
Sponsor
SpringWorks Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05028166
Brief Title
Individual Patient Compassionate Use of Mirdametinib
Study Type
Expanded Access
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Available
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
SpringWorks Therapeutics, Inc.
4. Oversight
5. Study Description
Brief Summary
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee and Single Patient IND approval.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
Keywords
RASopathies, NF1, Neurofibromatosis, Plexiform Neurofibromas, Langerhans
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Mirdametinib (MEK Inhibitor)
Intervention Description
Patients will receive oral mirdametinib.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Eligibility Criteria
Inclusion Criteria:
Patient has a serious, debilitating or life-threatening medical condition with no suitable treatment available, has exhausted current standard of care or declined medically appropriate treatment for ethical or medical reasons.
Patient does not qualify to participate in any ongoing clinical trial sponsored by SpringWorks in an accessible geographical location.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
SpringWorks Medical Information
Phone
(888) 400-7989
Email
medinfo@springworkstx.com
12. IPD Sharing Statement
Learn more about this trial
Individual Patient Compassionate Use of Mirdametinib
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