A Phase 1 interventional study of BH-30236 and Venetoclax in Leukemia, Leukemia, Myeloid and Leukemia, Myeloid, Acute, sponsored by BlossomHill Therapeutics. Recruiting at 13 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-09-24.
Sponsored by BlossomHill Therapeutics · Phase 1, Interventional, and Treatment
Study BH-30236-01 is a first-in-human (FIH), Phase 1/1b, open-label, dose escalation and expansion study in participants with relapsed/refractory acute myelogenous leukemia (R/R AML) or higher-risk myelodysplastic syndrome (HR-MDS).
Phase 1, Part 1 Dose Escalation - Monotherapy will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of BH-30236 administered orally. Approximately 50 participants may be enrolled in Phase 1, Part 1 Dose Escalation - Monotherapy.
Phase 1, Part 2 Dose Escalation - Combination with Venetoclax will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of BH-30236 administered as a combination therapy with venetoclax. Approximately 48 participants may be enrolled in Phase 1, Part 2 Dose Escalation - Combination with Venetoclax.
Phase 1b (Dose Expansion) will follow Phase 1 to further understand the relationships among dose, exposure, toxicity, tolerability, and clinical activity. Up to 72 participants may be enrolled in Phase 1b of the study as a monotherapy or in combination with venetoclax.
This is a Phase 1/1b, multi-center, open-label, dose escalation, first-in-human study to evaluate the safety, tolerability, PK, PD, and preliminary anti-leukemic activity of the CLK inhibitor, BH-30236 as a monotherapy or in combination with venetoclax, in adult participants with R/R AML or HR-MDS.
The study consists of three parts: Phase 1, Part 1 Dose Escalation - Monotherapy, Phase 1, Part 2 Dose Escalation - Combination with Venetoclax, and Phase 1b Dose Expansion.
Phase 1, Part 1 Dose Escalation - Monotherapy is anticipated to enroll approximately 50 participants to evaluate the safety, tolerability, PK, PD, and preliminary anti-leukemic activity of BH-30236, as well as determine the MTD and/or the preliminary recommended dose(s) for expansion (RDEs).
Phase 1, Part 2 Dose Escalation - Combination with Venetoclax is anticipated to enroll approximately 48 participants to evaluate the safety, tolerability, PK, PD, and preliminary anti-leukemic activity of BH-30236, as well as determine the MTD and/or the preliminary recommended dose(s) for expansion (RDEs).
Phase 1 will follow a Bayesian optimal interval (BOIN) design dose escalation, where participants will receive ascending doses of BH-30236 to determine the recommended RDEs.
Phase 1b Dose Expansion will enroll approximately 72 participants to evaluate the safety, tolerability, and preliminary anti-leukemic activity of BH-30236 as a monotherapy or in combination with venetoclax at selected RDEs determined in Phase 1 Dose Escalation.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's planned enrollment of 170 is above the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →BlossomHill Therapeutics is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
Compared within Leukemia, Myeloid, Acute, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Leukemia. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
The above are a summary, other inclusion criteria details may apply.
Exclusion Criteria:
The above is a summary, other exclusion criteria details may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BH-30236 Monotherapy for Dose Escalation
Drug: BH-30236
BH-30236 in Combination with Venetoclax for Dose Escalation.
Drug: BH-30236 · Drug: Venetoclax
BH-30236 based on Monotherapy dose escalation data.
Drug: BH-30236
BH-30236 in Combination with Venetoclax based on Dose Escalation data
Drug: BH-30236 · Drug: Venetoclax
BH-30236 will be provided as either a 5 mg, 15 mg or 30 mg tablet. Participants will take BH-30236 tablets orally depending on their dose level assignment.
Venetoclax will be provided as 10 mg, 50 mg or 100 mg tablets. Participants will take venetoclax orally per label instructions.
Also known as: venclexta, venclyxto
Dose Escalation: Frequency of dose limiting toxicities (DLTs)
DLTs are dose-limiting toxicities as defined in the study protocol.
Time frame: Dose-limiting toxicities are collected during the first treatment cycle (28 days)
Dose Escalation and Expansion: Safety evaluation of BH-30236: Number of participants with treatment-related adverse events as assessed by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Frequency, severity and relationship to study drug of AEs and SAEs
Time frame: From first dose until 28 days after last dose of BH-30236
Dose Expansion: Composite Complete Remission (CR) Rate
Composite CR rate disease assessment in accordance with the following guidelines: European Leukemia Network (ELN) 2022 for acute myelogenous leukemia (AML) and International Working Group (IWG) 2023 for myelodysplastic syndrome (MDS).
Time frame: From first dose of BH-30236 until disease progression (up to approximately 1 year)
Dose Escalation and Expansion: Maximum observed blood concentration (Cmax) of BH-30236.
Blood samples for PK analyses will be collected at predetermined time points and analyzed.
Time frame: Evaluation performed in Cycle 1 (cycle duration is 28 days).
Dose Escalation: Area under the blood concentration time curve (AUC) of BH-30236.
Blood samples for PK analyses will be collected at predetermined time points and analyzed.
Time frame: Evaluation performed in Cycle 1 (cycle duration is 28 days).
Dose Escalation and Expansion: Concentration before dose at steady state (Ctrough).
Blood samples for PK analyses will be collected at predetermined time points and analyzed.
Time frame: Evaluation performed in all treatment cycles up to one year (cycle duration is 28 days).
Dose Escalation and Expansion: Objective Response Rate (ORR)
Objective response rate disease assessments in accordance with the following guidelines: ELN 2022 recommendations for AML and IWG 2023 for MDS (CR, CR with partial hematologic recovery \[CRh\], CR with incomplete count recovery \[CRi\], CR with limited count recovery \[CRL\], morphologic leukemia-free state \[MLFS\], or partial response \[PR\]).
Time frame: From first dose of BH-30236 until disease progression (up to approximately 1 year)
Dose Escalation and Expansion: Duration of Response (DoR)
Time from first documented response until the date of relapse or death.
Time frame: Time from first documented response until disease progression or death (approximately 1 year).
Dose Escalation and Expansion: Time to remission (TTR)
Time from first dose to the achievement of first remission Disease assessments will follow the following guidelines: ELN 2022 for AML and IWG 2023 for MDS.
Time frame: From first dose of BH-30236 until complete remission, disease progression or death (approximately 1 year).
Dose Escalation and Expansion: Relapse-free Survival (RFS)
The time from the start of treatment date to disease progression, death, or initiation of a new anti-leukemic therapy.
Time frame: From first dose of BH-30236 until disease progression, death, or initiation of a new anti-leukemic therapy (approximately 1 year).
Dose Escalation and Expansion: Measurable Residual Disease (MRD)
For AML, using ELN 2022 criteria for disease assessment from Screening, then at the beginning of Cycle 2 and 3, and then every second cycle thereafter.
Time frame: From time of first dose until discontinuation of BH-30236 (approximately 1 year).
Dose Escalation and Expansion: Measurement of the change in RNA alternative splicing markers on BH-30236 treatment
Peripheral blood samples for pharmacodynamic (PD) analyses will be collected at predetermined time points and analyzed.
Time frame: From time of first dose until discontinuation of BH-30236 (approximately 1 year).
Dose Escalation and Expansion: Complete remission (CR) / complete remission with partial hematologic recovery (CRh) rate for AML and complete remission/partial remission (CR/PR) rate for HR-MDS
In accordance with the following guidelines: ELN 2022 recommendations for AML and IWG 2023 for MDS (CR, CR with partial hematologic recovery \[CRh\], CR with incomplete count recovery \[CRi\], CR with limited count recovery CRL, morphologic leukemia-free state \[MLFS\], or partial response \[PR\])
Time frame: Time from first documented response until disease progression or death (approximately 1 year)
Plan to share: No
Started Jun 2024 (actual). Primary completion is estimated for Jun 2026, 24 months after the start.
Of 183 industry-sponsored phase 1 interventional studies under Leukemia, Myeloid, Acute, half reached primary completion within 40 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 62. This study's planned 24 months is below that.
Counting every sponsor, half of 488 studies reached it within 49 months; a quarter within 31 and three quarters within 76.
How these figures are worked out, and which studies are left out of them.
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BlossomHill Therapeutics