Advanced Malignancies That Harbor IDHR132 MutationsPhase 1NCT02381886
A Study of IDH305 in Patients With Advanced Malignancies That Harbor IDH1R132 Mutations
A Phase 1 study testing IDH305 in people with Advanced Malignancies That Harbor IDHR132 Mutations, run by Novartis Pharmaceuticals at 16 sites in 8 countries.
Trial summary
A Phase I study of IDH305 in patients with advanced malignancies that harbor IDH1R132 mutations. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Advanced Malignancies That Harbor IDHR132 Mutations
- What is being tested?
- IDH305
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 166 participants
- Where
- 16 sitesin 8 countries
- Study dates
- 6 Mar 2015to 31 Oct 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documented IDH1R132-mutant tumors
- ECOG performance status ≤ 2
Who cannot join
- 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
Trial locations and contacts
16 sites in 8 countries.
16 sites in 8 countries
United States5 sites
Dana Farber Cancer Institute SC (1)
Boston, Massachusetts, 02115
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Uni Of TX MD Anderson Cancer Cntr
Houston, Texas, 77030
Columbia University Medical Center- New York Presbyterian Onc Dept.
New York, New York, 10032
Columbia University Medical Center
New York, New York, 10032
Germany3 sites
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Heidelberg, 69120
Novartis Investigative Site
Jena, Thuringia, 07740
Australia2 sites
Novartis Investigative Site
Melbourne, Victoria, 3004
Novartis Investigative Site
Parkville, Victoria, 3050
Netherlands2 sites
Novartis Investigative Site
Rotterdam, South Holland, 3015 GD
ErasmusMC Cancer lnstitute, Neuro-Oncology, Rm G3-55
Rotterdam, 3075 EA
About this study
Official title: 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.
- Being tested
- IDH305
- Keywords
- IDHR132 mutations, IDH305, IDH, IDH1, AML, Acute Myeloid Leukemia, Leukemia, Myeloid, Acute, MDS, Myelodysplastic Syndrome, Glioma, Oligodendroglioma, Astrocytoma, Glioblastoma, Cholangiocarcinoma, Solid Tumors
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelodysplastic Syndromes, Glioma, Oligodendroglioma, Astrocytoma, Glioblastoma, Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IDH305Drug
| Study group | Role | What they receive |
|---|---|---|
| IDH305 | Experimental | Not described Receives: Drug: IDH305 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incident rate of dose limiting toxicities (DLTs)
To estimate the maximum tolerated dose/recommended dose for expansion (MTD/RDE)
Measured: 21 days
Secondary outcomes (5)
Incidence of adverse events (AEs)
To characterize the safety and tolerability of IDH305
Measured: 30 months
Plasma PK parameters (AUC, Cmax, Tmax)
To characterize the PK profile of IDH305
Measured: 30 months
Changes of 2-hydroxyglutarate concentration in patient specimens
To characterize the PD profile of IDH305
Measured: 30 months
Overall response rate (ORR)
To assess any preliminary anti-tumor activity of IDH305
Measured: 30 months
Incidence of serious adverse events (SAE)
To characterize the safety and tolerability of IDH305
Measured: 30 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2015 (estimated)
- Study start6 Mar 2015
- Primary completion7 Dec 2016
- Study completion31 Oct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Advanced Malignancies That Harbor IDHR132 MutationsStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 166 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02381886
- Sponsor's protocol ID
- CIDH305X2101
- Status last verified
- Apr 2026
- First submitted
- 2 Mar 2015
- Last updated
- 13 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 35353219
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of IDH305 in Patients With Advanced Malignancies That Harbor IDH1R132 Mutations
- ClinicalTrials.gov ID
- NCT02381886
- Acronym / sponsor ID
- CIDH305X2101
- Keywords
- IDHR132 mutations, IDH305, IDH, IDH1, AML, Acute Myeloid Leukemia, Leukemia, Myeloid
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 166 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 6 Mar 2015 (actual)
- Primary completion
- 7 Dec 2016 (actual)
- Study completion
- 31 Oct 2030 (estimated)
- First posted
- 6 Mar 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase I study of IDH305 in patients with advanced malignancies that harbor IDH1R132 mutations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IDH305 | Experimental | Drug: IDH305 |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Documented IDH1R132-mutant tumors
- ECOG performance status ≤ 2
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IDH305 | Drug | IDH305 |
Primary outcome
Incident rate of dose limiting toxicities (DLTs)
Time frame: 21 days
To estimate the maximum tolerated dose/recommended dose for expansion (MTD/RDE)
5 secondary outcomes
Incidence of adverse events (AEs)
Time frame: 30 months
Plasma PK parameters (AUC, Cmax, Tmax)
Time frame: 30 months
Changes of 2-hydroxyglutarate concentration in patient specimens
Time frame: 30 months
Overall response rate (ORR)
Time frame: 30 months
Incidence of serious adverse events (SAE)
Time frame: 30 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Derived from this study: PubMed 35353219DiNardo 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.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
16 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IDH305 in people with Advanced Malignancies That Harbor IDHR132 Mutations, run by Novartis Pharmaceuticals at 16 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Germany, Australia, Netherlands, Belgium and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incident rate of dose limiting toxicities (DLTs) (time frame: 21 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02381886, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.