MedPath

Study of Safety and Efficacy of HDM201 in Combination With LEE011 in Patients With Liposarcoma

Phase 1
Completed
Conditions
Liposarcoma
Interventions
Registration Number
NCT02343172
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

To determine the MTD/RP2D of the HDM201 and LEE011 combination and evaluate whether the combination is safe and has beneficial effects in patients with liposarcoma.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
74
Inclusion Criteria
  • Patients with histologically documented, locally advanced or metastatic WD/DD liposarcoma who have received at least one prior systemic therapy
  • Patients with radiologic progression, defined by RECIST v.1.1, occurring while on/or within 6 months after last systemic treatment, prior to enrollment
  • ECOG performance status of 0-1
Exclusion Criteria
  • Prior treatment with compounds with the same mode of action
  • Patients with TP53 mutated tumors, if the molecular status is known
  • Symptomatic central nervous system metastases
  • Inadequate organ function
  • Previous and concomitant therapy that precludes enrollment, as defined by protocol

Other protocol-defined inclusion/exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
HDM201+LEE011HDM201-
HDM201+LEE011LEE011-
Primary Outcome Measures
NameTimeMethod
Phase Ib: Incidence of Dose Limiting Toxicities (DLTs) during the first cycle of treatment.5 years

DLTs in the first cycle of treatment.

Phase Ib: Exposure to HDM201 and LEE011 as measured by AUC 0-24h5 years

as measured by AUC0-24h

Phase II: Progression free survival (PFS)5 years

To assess the preliminary anti-tumor activity of HDM201 in combination with LEE011 in liposarcoma

Secondary Outcome Measures
NameTimeMethod
Phase Ib/II: Incidence and severity of AEs and SAEs5 years

Run-in part to assess safety of HDM201 in combination with LEE011

Phase Ib/II: Pharmacokinetics (PK) parameters of HDM201 and LEE011: AUCtau5 years

Run-in part to evaluate the PK parameters of HDM201 and LEE011

Phase Ib: BOR, ORR and PFS as per RECIST v1.1, assessed by investigator5 years

Run-in part to assess the preliminary anti-tumor activity of HDM201 in combination with LEE011 in liposarcoma

Phase Ib/II: number of patients with dose interruptions and reduction5 years

Run-in part To assess tolerability of HDM201 in combination with LEE011

Phase II: BOR, ORR and PFS as per RECIST v1.1, assessed by investigator5 years

Run-in part to further assess the anti-tumor activity of HDM201 in combination with LEE011 in liposarcoma

Phase Ib/II: Pharmacokinetics (PK) parameters of HDM201 and LEE011: Tmax5 years

Run-in part to evaluate the PK parameters of HDM201 and LEE011

Phase Ib/II: Pharmacokinetics (PK) parameters of HDM201 and LEE011: AUClast5 years

Run-in part to evaluate the PK parameters of HDM201 and LEE011

Phase Ib/II: Changes from baseline of Pharmacodynamics (PD) markers in tumor tissue (p21, MDM2)5 years

Run-in part measure of p21 and MDM2 protein levels by IHC (H-Score) (p53 and CDK4 pathways) to assess changes from baseline of PD markers in tumor tissue and a potential relationship with clinical outcome.

Phase Ib/II: anti-tumor activity endpoint (BOR, PFS)5 years

Run-in part to assess PD effect of HDM201 and LEE011 and a potential relationship with clinical outcome

Phase Ib/II: dose intensity5 years

Run-in part To assess tolerability of HDM201 in combination with LEE011

Phase Ib/II: Changes from baseline of Pharmacodynamics (PD) markers in blood (GDF-15)5 years

Run-in part measure of GDF-15 fold-change in protein levels (PD direct targets of p53) to assess PD changes from baseline in blood and a potential relationship with clinical outcome.

Phase Ib/II: Pharmacokinetics (PK) parameters of HDM201 and LEE011: Cmax5 years

Run-in part to evaluate PK parameters of HDM201 and LEE011

Trial Locations

Locations (1)

Novartis Investigative Site

🇨🇳

Taipei, Taiwan

© Copyright 2025. All Rights Reserved by MedPath