HMPL-306 for Blood Cancers

Recruiting at 16 trial locations

Overseen By Iris Carton, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Hutchmed

Must not be taking: QT-prolonging drugs

Disqualifiers: Pregnancy, Severe infection, Heart conditions, others

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

Compensation: Varies

Number of Visits: Unknown

46 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests HMPL-306, a new oral medication, to determine its safety and effectiveness for individuals with certain advanced blood cancers unresponsive to usual treatments. The focus is on cancers with specific changes in the IDH gene, which complicate treatment. Eligible participants should have a blood cancer that does not respond to standard therapies and have faced these challenges for some time. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new medication.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are taking medications that prolong the QT interval (a heart rhythm measure), you may not be eligible. Also, if you have taken an investigational agent, there must be a 14-day gap before starting the study drug.

Is there any evidence suggesting that HMPL-306 is likely to be safe for humans?

Research has shown that HMPL-306 was safe in earlier studies, with most patients tolerating it well. These studies found promising results with few serious side effects. Although more research is needed to confirm these findings, the early data encourages those considering joining a trial with HMPL-306. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for blood cancers, which often rely on chemotherapy or targeted therapies, HMPL-306 is unique because it targets a specific mutation in cancer cells known as IDH1/2. Most current treatments don't specifically address this mutation, making HMPL-306 promising for patients with these genetic changes. Additionally, HMPL-306 is administered orally, which could offer a more convenient option compared to intravenous therapies. Researchers are excited about its potential to provide a more personalized and potentially effective treatment for individuals with these specific mutations in their blood cancers.

What evidence suggests that HMPL-306 might be an effective treatment for blood cancers?

Research has shown that HMPL-306, the treatment under study in this trial, is a promising option for blood cancers with specific genetic changes called IDH1 and IDH2 mutations. Studies have found that this treatment can lower levels of 2-HG, a harmful substance linked to these mutations, by about 90% after just one dose. This reduction indicates that HMPL-306 effectively targets the mutations driving the cancer. The treatment has demonstrated safety and positive early results in initial trials. These findings suggest that HMPL-306 could be a valuable option for patients with these specific types of blood cancers. ^{1 2 4 5 6}

Who Is on the Research Team?

Bo Zhang

Principal Investigator

Hutchison Medipharma Limited

Are You a Good Fit for This Trial?

This trial is for adults over 18 with advanced blood cancers that have come back or haven't responded to treatment, and who are not too sick to participate (ECOG ≤ 2). They mustn't be pregnant/breastfeeding, have severe infections, certain heart conditions, uncontrolled cancer complications, specific liver/gastrointestinal diseases, or poor organ function. Those allergic to HMPL-306 or its ingredients can't join.

Inclusion Criteria

My blood cancer has come back or hasn't responded to treatment.

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

Exclusion Criteria

I haven't had any new cancers in the last 2 years, except for certain skin cancers or early-stage cervical or breast cancer.

Subjects with a known hypersensitivity to HMPL-306 or to any of its excipients

I do not have a severe infection or unexplained fever over 38.3°C.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive HMPL-306 to determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D)

4 weeks

Weekly visits for dose escalation monitoring

Dose Expansion

Participants receive the MTD/RP2D of HMPL-306 to evaluate safety and efficacy

Up to 36 months

Monthly visits for treatment and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• HMPL-306

Trial Overview The study tests HMPL-306's safety and effectiveness in patients with blood cancers containing IDH mutations. It's an open-label trial where all participants receive the drug; researchers will monitor how the body processes it and any signs of cancer improvement.

How Is the Trial Designed?

Group I: Treatment Experimental Treatment 1 Intervention

All patients will be administered HMPL-306 orally QD

Who Is Running the Clinical Trial?

Hutchmed

Lead Sponsor

Trials

38

Recruited

6,700+

Hutchison Medipharma Limited

Lead Sponsor

Trials

104

Recruited

14,000+

Dr. Weiguo Su

Hutchison Medipharma Limited

Chief Executive Officer since 2022

PhD in Chemistry from Harvard University, BSc in Chemistry from Fudan University

Dr. Karen Atkin

Hutchison Medipharma Limited

Chief Medical Officer since 2023

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04764474

NCT04764474 | A Study of HMPL-306 in Advanced ... This is a phase 1, open-label, multicenter, single-arm study to evaluate safety, tolerability, PK, PD, and preliminary efficacy of HMPL-306 administered orally.

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2666634025000029

HMPL-306 in relapsed or refractory IDH1- and/or IDH2- ... The 2-HG level was reduced by ∼90% at 24 h after a single administration of HMPL-306 at 10 mg/kg, a clinically relevant dose level.

3. authorea.com authorea.com/users/902431/articles/1277458-hmpl-306-ranosidenib-the-next-frontier-in-midh-cancer-therapy

HMPL-306 (Ranosidenib): The Next Frontier in mIDH ... This review synthesizes HMPL-306's mechanistic innovation, clinical promise, and translational potential, advocating its role as a cornerstone ...

4. cell.com cell.com/cms/10.1016/j.medj.2025.100575/attachment/05a5d5bd-3a91-40ab-801c-6d4f906cafa6/mmc2.pdf

HMPL-306 in relapsed or refractory IDH1- and/or IDH2- ... Conclusions: HMPL-306 showed an acceptable safety profile and promising preliminary efficacy. A phase 3, randomized study of HMPL-306 in R/R AML ...

5. hutch-med.com hutch-med.com/raphael-phase-3-hmpl-306-initiation/

and/or IDH2-Mutated Relapsed/Refractory Acute Myeloid ... HMPL-306 is a novel dual-inhibitor of IDH1 and IDH2 enzymes. Mutations of IDH1 and IDH2 have been implicated as drivers of certain hematological ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39892383/

and/or IDH2-mutated acute myeloid leukemia: A phase 1 ... Conclusions: HMPL-306 showed an acceptable safety profile and promising preliminary efficacy. A phase 3, randomized study of HMPL-306 in R/R ...