A Study of IDH305 in Patients With Advanced Malignancies That Harbor IDH1R132 Mutations

A Phase I Study of IDH305 in Patients With Advanced Malignancies That Harbor IDH1R132 Mutations

A Phase I study of IDH305 in patients with advanced malignancies that harbor IDH1R132 mutations.

Study Overview

• Status: Active, not recruiting
• Conditions: Advanced Malignancies That Harbor IDHR132 Mutations
• Intervention / Treatment: Drug: IDH305

• Study Type: Interventional
• Enrollment (Actual): 166
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • Novartis Investigative Site
    • Parkville, Victoria, Australia, 3050
      • Novartis Investigative Site
• Belgium
  • Leuven, Belgium, 3000
    • Novartis Investigative Site
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Novartis Investigative Site
• Germany
  • Heidelberg, Germany, 69120
    • Novartis Investigative Site
  • Jena, Germany, 07740
    • Novartis Investigative Site
  • Koeln, Germany, 50937
    • Novartis Investigative Site
• Netherlands
  • Rotterdam, Netherlands, 3075 EA
    • ErasmusMC Cancer lnstitute, Neuro-Oncology, Rm G3-55
• Singapore
  • Singapore, Singapore, 168583
    • Novartis Investigative Site
• Spain
  • Catalunya
    • Barcelona, Catalunya, Spain, 08035
      • Novartis Investigative Site
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Dana Farber Cancer Institute SC (1)
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center- New York Presbyterian Onc Dept.
  • Texas
    • Houston, Texas, United States, 77030
      • Uni of TX MD Anderson Cancer Cntr .

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Documented IDH1R132-mutant tumors
• ECOG performance status ≤ 2

Exclusion Criteria:

• Patients who have received prior treatment with a mutant-specific IDH1 inhibitor (with the exception of glioma patients)
• Medical conditions that would prevent the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures such as the presence of other clinically significant cardiac, respiratory, gastrointestinal, renal, hepatic or neurological disease.
• Acute Promyelocytic Leukemia
• Women who are pregnant or lactating

Other protocol-defined Inclusion/Exclusion may apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IDH305	Drug: IDH305

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incident rate of dose limiting toxicities (DLTs)	To estimate the maximum tolerated dose/recommended dose for expansion (MTD/RDE)	21 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events (AEs)	To characterize the safety and tolerability of IDH305	30 months
Plasma PK parameters (AUC, Cmax, Tmax)	To characterize the PK profile of IDH305	30 months
Changes of 2-hydroxyglutarate concentration in patient specimens	To characterize the PD profile of IDH305	30 months
Overall response rate (ORR)	To assess any preliminary anti-tumor activity of IDH305	30 months
Incidence of serious adverse events (SAE)	To characterize the safety and tolerability of IDH305	30 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

General Publications

• DiNardo CD, Hochhaus A, Frattini MG, Yee K, Zander T, Kramer A, Chen X, Ji Y, Parikh NS, Choi J, Wei AH. A phase 1 study of IDH305 in patients with IDH1R132-mutant acute myeloid leukemia or myelodysplastic syndrome. J Cancer Res Clin Oncol. 2023 Mar;149(3):1145-1158. doi: 10.1007/s00432-022-03983-6. Epub 2022 Mar 30.

Study record dates

Study Major Dates

• Study Start (Actual): January 6, 2015
• Primary Completion (Actual): December 7, 2016
• Study Completion (Estimated): October 24, 2025

Study Registration Dates

• First Submitted: March 2, 2015
• First Submitted That Met QC Criteria: March 2, 2015
• First Posted (Estimated): March 6, 2015

Study Record Updates

• Last Update Posted (Actual): February 28, 2024
• Last Update Submitted That Met QC Criteria: February 26, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; Glioma; AML; Leukemia; MDS; Glioblastoma; Solid Tumors; Cholangiocarcinoma; Myelodysplastic Syndrome; IDH; IDH1; Acute; Astrocytoma; Oligodendroglioma; Myeloid; IDHR132 mutations; IDH305
• Additional Relevant MeSH Terms: Neoplasms
• Other Study ID Numbers: CIDH305X2101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No