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Glasdegib

Phase 2

Acute Myelogenous Leukemia (AML) Due to Therapy | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Mar 6, 2026

Target and mechanism

Molecular targetSMO
Target classAntagonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment30

FDA Designations

No designations recorded

Clinical trial landscape

Glasdegib · 1 trial · 2 indications

Phase 2 1
NCT04231851CPX-351 and Glasdegib for Newly Diagnosed Acute Myelogenous Leukemia With MDS Related Changes or Therapy-related Acute Myeloid LeukemiaAcute Myelogenous Leukemia (AML) Due to Therapy
ACTIVE NOT_RECRUITING30 Analytics
PHASE2ACTIVE NOT_RECRUITING
CPX-351 and Glasdegib for Newly Diagnosed Acute Myelogenous Leukemia With MDS Related Changes or Therapy-related Acute Myeloid Leukemia
Acute Myelogenous Leukemia (AML) Due to TherapyUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants With Event-Free Survival at 6 Months
6 months

This is defined as the percentage of subjects with event-free survival (EFS) at 6 months. EFS is defined as the number of months where patients are in a remission state.

Secondary Endpoints

Percentage of Grade 3-5 Adverse Events
From the start date of treatment until 4 weeks after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 1 year.
Overall Response Rate
From the start date of treatment until first date of CR/CRi or an average of 1 year.
Durability of Response
From the start date of treatment until first date of CR/CRi or an average of 1 year.
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
CPX-351 and GlasdegibEXPERIMENTALIn Induction, subjects receive 44mg/m2/100mg/m2 IV on days 1, 3 and 5 and Glasdegib 100mg PO daily on days 6 to 28. If re-induction is needed: Subjects receive 44mg/m2/100mg/m2 IV on days 1 and 3 and Glasdegib 100mg PO daily on days 4 to 28. In consolidation: Subjects receive 29mg/m2/65mg/m2 IV on days 1 and 3 and Glasdegib 100mg PO daily on days 4 to 28. If maintenance is required, Subjects receive Glasdegib 100mg PO daily for up to one year

Interventions

NameTypeDescription
GlasdegibDRUGGiven PO
CPX-351DRUGGiven IV
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Previously untreated therapy-related AML or AML with myelodysplastic related changes as described by World Health Organization (WHO) 2016 1. AML arising in MDS (including CMML) or MDS/MPN syndrome 2. AML with MDS-related cytogenetic abnormalities (Appendix A, metaphase FI...

Countries:United States
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Frequently asked questions about Glasdegib

What is Glasdegib used for?

Glasdegib is an investigational small molecule being studied for Acute Myelogenous Leukemia (AML) Due to Therapy. It is also being evaluated in Acute Myeloid Leukemia With Myelodysplasia-Related Changes. The drug is currently in Phase 2 clinical development and has not been approved by the FDA.

What does Glasdegib target?

Glasdegib targets the Hedgehog signaling pathway, as indicated by its -degib (Hedgehog) target class. It is a small molecule designed to interfere with this pathway, which is implicated in certain cancers. The drug is being studied in combination with CPX-351 for specific types of acute myeloid leukemia.

Who makes Glasdegib?

Glasdegib is being developed by Jazz Pharmaceuticals plc, a biopharmaceutical company traded on the NASDAQ under the ticker JAZZ. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with acute myeloid leukemia.

What phase is Glasdegib in?

Glasdegib is in Phase 2 clinical development. It is currently being studied in an active, not recruiting clinical trial for Acute Myelogenous Leukemia (AML) Due to Therapy and Acute Myeloid Leukemia With Myelodysplasia-Related Changes. The drug is investigational and not yet approved.

What clinical trials is Glasdegib in?

Glasdegib is being evaluated in clinical trial NCT04231851, titled 'CPX-351 and Glasdegib for Newly Diagnosed Acute Myelogenous Leukemia With MDS Related Changes or Therapy-related Acute Myeloid Leukemia'. This Phase 2 trial is active but not recruiting, with an enrollment of 30 participants in the United States.

Is Glasdegib the same as CPX-351?

No, Glasdegib is not the same as CPX-351. Glasdegib is a small molecule targeting the Hedgehog pathway, while CPX-351 is a liposomal formulation of chemotherapy drugs. The two are being studied in combination in clinical trial NCT04231851 for acute myeloid leukemia.