Individual Patient Compassionate Use of Nirogacestat | NCT05041036

Studies
Individual Patient Compassionate Use of Nirogacestat

NCT05041036

Sponsor

SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany

Status

Available

Last Update Posted

May 8, 2026Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Desmoid Tumor
NOTCH Gene Mutation Positive Tumors

Interventions

Nirogacestat (Gamma Secretase Inhibitor)

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2026

Overall Recruitment Status or Expanded Access Status

Available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Sep 7, 2021

First Submission Date that Met QC Criteria

Sep 7, 2021

First Posted Date

Sep 10, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 4, 2026

Last Update Posted Date

May 8, 2026Actual

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Individual Patients

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Arms/Interventions Module

Arm Groups

Not provided

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Nirogacestat (Gamma Secretase Inhibitor)	Drug	Patients will receive nirogacestat tablets to be taken orally.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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.

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Role	Phone	Extension	Email
US Medical Information	Contact	888-275-7376		eMediUSA@emdserono.com

Condition Browse Module

MeSH Terms

ID	Term
D018222	Desmoid Tumors

Ancestor Terms

ID	Term
D005350	Fibroma
D018218	Neoplasms, Fibrous Tissue
D009372	Neoplasms, Connective Tissue
D018204	Neoplasms, Connective and Soft Tissue

Browse Leaves

Not provided

Browse Branches

Not provided