A Dose Finding Study of IDH305 With Standard of Care in IDH1 Mutant Acute Myeloid Leukemia

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to evaluate the safety, tolerability and potential efficacy of IDH305 with standard treatments for newly diagnosed IDH1R132 mutant acute myeloid leukemia (AML).

Official Title
-----------------
A Phase I, Open Label, Multicenter, Dose Finding Study of IDH305 With Standard of Care in IDH1 Mutant Acute Myeloid Leukemia

Conditions
-----------------
Acute Myeloid Leukemia (AML)

Intervention / Treatment
-----------------
* Drug: IDH305


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: -- Previously untreated AML. Patients with untreated, high or very high risk MDS (according to rIPSS or equivalent) are also permitted in Arm 2. Documentation of IDH1R132 mutation of tumor ECOG performance status ≤ 2 Clinically fit for standard of care medication per protocol. Exclusion Criteria: Prior treatment for AML or MDS Any severe or uncontrolled 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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1: Medically fit for induction<br>IDH305 + Standard of care for patients that are medically fit for induction. | Drug: IDH305<br> <br> |
| Experimental: Arm 2 Medically unfit for induction<br>IDH305 + Standard of care for patients that are medically unfit for induction. | Drug: IDH305<br> <br> |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dose Limiting toxicities | (escalation only) | 10 months | 
| Number of patients with adverse events (AEs) |  | 36 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Area Under Curve (AUC) | To characterize the PK profile of IDH305 with SOC medications (each Arm) | 36 months | 
| Maximum Plasma Concentration (Cmax) | To characterize the PK profile of IDH305 with SOC medications (each Arm) | 36 months | 
| Time taken to reach maximum plasma concentration (Tmax) | To characterize the PK profile of IDH305 with SOC medications (each Arm) | 36 months | 
| Complete remission rate (CRR) | To characterize preliminary anti-tumor activity for each arm of the study. (Arm 1 and Arm 2) | 36 months | 
| Overall response rate (ORR) | To characterize preliminary anti-tumor activity for each arm of the study. (Arm 1 and Arm 2) | 36 months | 
| Event free survival (EFS) | To characterize preliminary anti-tumor activity for each arm of the study. (Arm 1 and Arm 2) | 36 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
newly diagnosed acute myeloid leukemia, AML, myeloid dysplastic syndrome, MDS, IDH305, standard of care in IDH1 mutant acute myeloid leukemia