Managed Access Programs for PKC412, Midostaurin

• Conditions: FMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid Leukemia; Acute Myeloid Leukemia; Aggressive Systemic Mastocytosis; Mast Cell Leukemia; Systemic Mastocytosis With an Associated Hematologic Neoplasm

• Registration Number: NCT05219266
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The purpose of this registration is to list Managed Access Programs (MAPs) related to PKC4, Midostaurin.

Detailed Description

* CPKC412A2001X - No longer available- An open-labeled, multi-center, Expanded Treatment Protocol (ETP) of midostaurin (PKC412) in patients 18 years of age or older with newly-diagnosed FLT3-mutated acute myeloid leukemia (AML) who are eligible for standard induction and consolidation chemotherapy

* CPKC412AUS56X - No longer available - An open-label, multi-center, Expanded Treatment Protocol of Midostaurin (PKC412) in adult patients with newly diagnosed Fms-like tyrosine kinase receptor (FLT3) mutated Acute Myeloid Leukemia (AML) who are eligible for standard induction and consolidation chemotherapy.

* CPKC412A2407I - No longer available- Managed Access Program (MAP) Cohort Treatment Plan CPKC412A2407I to provide access to midostaurin (PKC412) for patients 18 years of age and older with newly-diagnosed FLT3-mutated AML and eligible for induction and consolidation chemotherapy

* CPKC412D2001M - No longer available- Managed Access Program (MAP) to provide access to Midostaurin (PKC412), for an individual patient with aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL) or mast cell sarcoma (MCS)

Study Timeline

• Study First Submitted: 2021-10-05
• Study First Submitted QC: 2022-02-01
• Study First Posted: 2022-02-02
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-27
• Last Update Posted: 2024-06-28

Recruitment & Eligibility

• Status: NO_LONGER_AVAILABLE
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• An independent request should be received from a licensed physician (in some instances from Health Authorities, Institutions or Governments).
• The patient has a serious or life-threatening disease or condition and no comparable or satisfactory alternative therapy is available for diagnosis, monitoring or treatment; patient is not medically eligible for available treatment alternatives or has exhausted all available treatment options.
• The patient is not eligible or able to enroll in a Novartis clinical trial or continue participation in a Novartis clinical trial.
• There is a potential patient benefit to justify the potential risk of the treatment use, and the potential risk is not unreasonable in the context of the disease or condition to be treated.
• The patient must meet any other medical criteria established by the medical experts responsible for the product or by the Health Authority in a country (as applicable).
• Provision of the product will not interfere with the initiation, conduct or completion of a Novartis clinical trial or overall development program.
• Managed access provision is allowed per local laws/regulations.

Exclusion Criteria

Not provided

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified