Individual Patient Compassionate Use of Nirogacestat

NCT05041036 | Unknown

• 1 other identifier: NIR-DT-701
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.

Conditions

Desmoid Tumor; NOTCH Gene Mutation Positive Tumors

Keywords

desmoid tumor; aggressive fibromatosis; desmoid fibromatosis; Notch-positive tumors

Interventions

Nirogacestat (Gamma Secretase Inhibitor) DRUG

Patients will receive nirogacestat tablets to be taken orally.

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient has a serious, debilitating or life-threatening medical condition that cannot be treated satisfactorily with an authorized medicinal product or other available standard treatment options, or all other treatment options have been exhausted.
• Patient does not qualify to participate in an ongoing clinical trial.

Sponsors & Collaborators

• SpringWorks Therapeutics, Inc.: lead

MeSH Terms

Conditions

Desmoid Tumors

Interventions

nirogacestat

Condition Hierarchy (Ancestors)

Fibroma; Neoplasms, Fibrous Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Central Study Contacts

US Medical Information

CONTACT

888-275-7376; eMediUSA@emdserono.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2021
• First Posted: September 10, 2021
• Last Updated: April 1, 2026

Record last verified: 2026-03