IDH305 for Advanced Cancer

Not currently recruiting at 18 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Cardiac, Respiratory, Gastrointestinal, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 21 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called IDH305 for individuals with advanced cancers that have a specific genetic change known as the IDH1R132 mutation. The main goal is to determine if IDH305 can safely assist these patients in fighting their cancer. Suitable candidates for this trial have tumors with the IDH1R132 mutation and can manage daily activities independently. Those who have previously received a similar treatment, except for those with glioma (a type of brain tumor), may not qualify. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that IDH305 is likely to be safe for humans?

Research has shown that IDH305 has been safe for most people in early tests, indicating that participants generally handled the drug well. Some studies also found that IDH305 may help fight tumors in patients with IDH1 mutations, particularly in blood cancers like AML, a type of leukemia.

Although researchers are still testing the treatment, early results are encouraging. No serious safety issues have been reported so far. However, like any new treatment, there might be some side effects. Discuss potential risks with the trial team before deciding to join. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about IDH305 because it targets a specific mutation in the IDH1 gene, which is present in some advanced cancers. This is different from standard treatments like chemotherapy, which attack rapidly dividing cells more broadly. IDH305 works by inhibiting the mutated enzyme, potentially stopping cancer growth more precisely and with fewer side effects. This targeted approach could offer a new hope for patients with cancers harboring this mutation, leading to more personalized and effective treatment options.

What evidence suggests that IDH305 might be an effective treatment for advanced cancer?

Research has shown that IDH305, the investigational treatment in this trial, could be promising for treating advanced cancers with specific IDH1 mutations. In earlier studies, this treatment helped shrink or halt tumor growth in patients with IDH1-mutated acute myeloid leukemia (AML). Specifically, about 33% of these patients showed measurable improvements in tumor size or disease symptoms. Additionally, 9.5% of patients experienced complete remission, meaning their cancer disappeared. These findings suggest that IDH305 could significantly impact cancers with these mutations. ^{1 2 5 6 7}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for patients with advanced cancers that have a specific genetic change called IDH1R132 mutation. They should be relatively active and able to care for themselves (ECOG ≤ 2). It's not open to those who've tried similar IDH1 inhibitors, except if they have glioma, or those with serious heart, lung, stomach, kidney, liver or nerve diseases. Pregnant or breastfeeding women can't join.

Inclusion Criteria

I can take care of myself but might not be able to do heavy physical work.

My tumor has an IDH1R132 mutation.

Exclusion Criteria

I have been diagnosed with Acute Promyelocytic Leukemia.

I am not pregnant or breastfeeding.

I have been treated with an IDH1 inhibitor before, except if I had a glioma.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive IDH305 to determine the maximum tolerated dose and assess safety and tolerability

21 days

Follow-up

Participants are monitored for safety, tolerability, and preliminary anti-tumor activity

30 months

What Are the Treatments Tested in This Trial?

Interventions

• IDH305

Trial Overview The study is testing the safety and effectiveness of a drug named IDH305 on patients whose tumors have the IDH1R132 mutation. This early-phase trial will help determine how well this treatment works and what doses are safe.

How Is the Trial Designed?

Group I: IDH305 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S0006497119310742

A Phase I Study of IDH305 in Patients with Advanced ... Conclusion: Preliminary clinical data suggest that IDH305 has a favorable safety profile and promising antitumor activity in IDH1-mutated AML.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5641959/

Discovery and Evaluation of Clinical Candidate IDH305, a ... Removal of PMB and subsequent SNAr reaction with 2,4-difluoropyrimidine yielded 18 in 59% overall yield.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02381886

NCT02381886 | A Study of IDH305 in Patients With ... It identifies the role of the intervention that participants receive. Types of arms include experimental arm, active comparator arm, placebo comparator arm, ...

4. researchgate.net researchgate.net/publication/337320952_A_Phase_I_Study_of_IDH305_in_Patients_with_Advanced_Malignancies_Including_RelapsedRefractory_AML_and_MDS_That_Harbor_IDH1R132_Mutations

A Phase I Study of IDH305 in Patients with Advanced ... Objective responses were reported in 7 (33%) AML patients: complete remission in 2 (9.5%), complete remission with incomplete recovery in 1 (4.8 ...

5. trial.medpath.com trial.medpath.com/clinical-trial/b72a0771646313fa/nl-omon43902-phase-1-study-idh305-advanced-malignancies

A Phase I study of IDH305 in patients with advanced ... * Patients with advanced malignancies whose tumors harbor IDHR132 mutations and for whom there are no curative therapy options who have progressed despite ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11546319/

Efficacy and safety of IDH inhibitors in IDH-mutated cancers The overall efficacy of IDH inhibitors in treating cancers with IDH mutations is superior to that of conventional medical therapy, potentially providing more ...

7. trial.medpath.com trial.medpath.com/clinical-trial/bffa1b4e49b1e503

A Study of IDH305 in Patients With Advanced Malignancies ... To characterize the safety and tolerability of IDH305. Incidence of serious adverse events (SAE), 30 months. To characterize the safety and tolerability of ...