NCT04029688 is a Phase 1, PHASE2 clinical trial of Idasanutlin in Acute Myeloid Leukemia (AML), sponsored by Hoffmann-La Roche.

Who is sponsoring NCT04029688?

NCT04029688 is sponsored by Hoffmann-La Roche.

What drugs are being tested in NCT04029688?

Interventions in NCT04029688: Idasanutlin, Venetoclax, Cyclophosphamide, Topotecan, Fludarabine, Cytarabine, Intrathecal Chemotherapy.

What condition does NCT04029688 study?

NCT04029688 studies Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Neuroblastoma, Solid Tumors.

Is NCT04029688 still recruiting?

NCT04029688 is currently terminated. Last updated 20 December 2024.

Are results published for NCT04029688?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2024-12-20 · How we verify

NCT04029688

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Study Evaluating the Safety, Tolerability, Pharmacokinetics and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in Treatment of Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias or Solid Tumors

Terminated Phase 1, PHASE2 Results posted Last updated 20 December 2024

What this trial tests

Phase 1, PHASE2 trial testing Idasanutlin in Acute Myeloid Leukemia (AML) in 38 participants. Terminated before completion.

Timeline

27 January 2020

Primary endpoint
6 May 2024

6 May 2024

Quick facts

Lead sponsor	Hoffmann-La Roche
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	38
Start date	27 January 2020
Primary completion	6 May 2024
Estimated completion	6 May 2024
Sites	14 locations across France, Netherlands, United Kingdom, Canada, United States, Spain

Drugs / interventions tested

Idasanutlin
Venetoclax (venetoclax) — full drug profile →
Cyclophosphamide (cyclophosphamide) — full drug profile →
Topotecan (topotecan) — full drug profile →
Fludarabine (FLUDARABINE) — full drug profile →
Cytarabine — full drug profile →
Intrathecal Chemotherapy

Conditions studied

Acute Myeloid Leukemia (AML) — all drugs for Acute Myeloid Leukemia (AML) →
Acute Lymphoblastic Leukemia (ALL) — all drugs for Acute Lymphoblastic Leukemia (ALL) →
Neuroblastoma — all drugs for Neuroblastoma →
Solid Tumors — all drugs for Solid Tumors →

Sponsor

Hoffmann-La Roche — full company profile →

Who can join

Adults 0 to 30, any sex, with Acute Myeloid Leukemia (AML) or Acute Lymphoblastic Leukemia (ALL). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Part 1a and 1b: Number of Participants With Adverse Events (AEs) and Severity of AEs Determined According to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE v5.0) Primary · From screening up to 30 days after study treatment discontinuation (approximately 7 months)

An AE is any untoward medical occurrence in participant administered a pharmaceutical product \& regardless of causal relationship with this treatment. An AE can therefore be any unfavorable \& unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with use of investigational product, whether or not considered related to investigational product. AEs were graded as per NCI CTCAE v5.0. Grade 1=Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; or intervention not indicated; Grade 2=Moderate; minimal, local or non-invasive int

AEs, Any Grade

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	8
Part 1a: Idasanutlin 3 mg/kg	3
Part 1a: Idasanutlin 4.5 mg/kg	6
Part 1a: Idasanutlin 6.4 mg/kg	3
Part 1a: Idasanutlin 8 mg/kg	6
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	3
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	3
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	6

AEs, Grade 1

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	1
Part 1a: Idasanutlin 3 mg/kg	0
Part 1a: Idasanutlin 4.5 mg/kg	0
Part 1a: Idasanutlin 6.4 mg/kg	0
Part 1a: Idasanutlin 8 mg/kg	0
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	0

AEs, Grade 2

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	0
Part 1a: Idasanutlin 3 mg/kg	0
Part 1a: Idasanutlin 4.5 mg/kg	0
Part 1a: Idasanutlin 6.4 mg/kg	0
Part 1a: Idasanutlin 8 mg/kg	0
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	1

AEs, Grade 3

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	2
Part 1a: Idasanutlin 3 mg/kg	1
Part 1a: Idasanutlin 4.5 mg/kg	3
Part 1a: Idasanutlin 6.4 mg/kg	2
Part 1a: Idasanutlin 8 mg/kg	1
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	3

AEs, Grade 4

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	5
Part 1a: Idasanutlin 3 mg/kg	2
Part 1a: Idasanutlin 4.5 mg/kg	3
Part 1a: Idasanutlin 6.4 mg/kg	1
Part 1a: Idasanutlin 8 mg/kg	5
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	3
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	3
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	1

AEs, Grade 5

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	0
Part 1a: Idasanutlin 3 mg/kg	0
Part 1a: Idasanutlin 4.5 mg/kg	0
Part 1a: Idasanutlin 6.4 mg/kg	0
Part 1a: Idasanutlin 8 mg/kg	0
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	0
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	1

Parts 1a and 1b: Number of Participants With Dose-Limiting Toxicities (DLTs) Primary · Cycle 1 (one cycle is 28 days)

DLTs were assessed for single-agent idasanutlin and idasanutlin in combination with chemotherapy or venetoclax. A DLT was defined as any AE that occurred during the DLT assessment window and was assessed by the investigator as related or possibly related to idasanutlin. An AE is an untoward medical occurrence in participant administered a pharmaceutical product \& regardless of causal relationship with this treatment. Following events were considered to be DLTs: any treatment-related death; elevation of serum hepatic transaminase; severe liver injury, in the absence of cholestasis or other cau

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	0
Part 1a: Idasanutlin 3 mg/kg	0
Part 1a: Idasanutlin 4.5 mg/kg	1
Part 1a: Idasanutlin 6.4 mg/kg	0
Part 1a: Idasanutlin 8 mg/kg	4
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	1
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	2
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	0

Part 1b: Objective Response Rate (ORR) in Participants With TP53 Wild-Type (WT) Neuroblastoma Assessed According to International Neuroblastoma Response Criteria (INRC) Primary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

ORR was defined as the percentage of participants with complete response (CR) or partial response (PR) at any time during study treatment, on 2 consecutive occasions ≥ 4 weeks apart, as determined by the investigator per INRC. Primary tumor: CR = \<10 millimeters (mm) residual soft tissue at primary site and complete resolution of meta-iodobenzylguanidine (MIBG) or fluorodeoxyglucose-positron emission tomography (FDG-PET) uptake at primary site. PR = ≥ 30% decrease in longest diameter of primary site and MIBG or FDG-PET uptake at primary site stable, improved, or resolved. Soft tissue \& bone

Group	Value	95% CI
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	0	0.0 – 84.19
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	33.3	0.84 – 90.57
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	0	0.0 – 60.24

Part 1a: Clinical Benefit Rate (CBR) in Participants With Solid Tumors From SE Population Assessed According to Response Evaluation Criteria Version 1.1 (RECIST v1.1) or INRC Secondary · From screening (maximum 28 days) up to Cycle 5 (cycle length=28 days)

CBR was defined as the percentage of participants achieving confirmed CR, PR, or stable disease (SD) on 2 consecutive occasions ≥4 weeks apart during the total study period. Per RECIST, CR was defined as disappearance of all target lesions. PR was defined as at least a 30% decrease in the sum of diameters (SOD) of target lesions, taking as reference the baseline SOD. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), taking as reference smallest sum on study. PD was defined as at least a 20% increase in the SOD of t

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	12.5	0.32 – 52.65
Part 1a: Idasanutlin 3 mg/kg	33.3	0.84 – 90.57
Part 1a: Idasanutlin 4.5 mg/kg	0	0.0 – 45.93
Part 1a: Idasanutlin 6.4 mg/kg	66.7	9.43 – 99.16
Part 1a: Idasanutlin 8 mg/kg	0	0.0 – 45.93

Part 1b: CBR in Participants With Neuroblastoma From SE Population Assessed According to INRC Secondary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

CBR=percentage of participants with CR, PR, or SD on 2 consecutive occasions ≥ 4 weeks apart per INRC. Primary tumor: CR=residual soft tissue at primary site is \<10 mm, with complete resolution of MIBG/FDG-PET uptake; PR= ≥30% decrease in longest diameter (LD) of primary site, with stable/improved MIBG/FDG-PET uptake; SD=Insufficient shrinkage for PR/increase for PD. PD= \>20% increase in LD from smallest sum \& minimum 5 mm increase in LD. Soft tissue \& bone metastases: CR=resolution of all disease sites; PR= ≥30% decrease in sum of non-primary target lesions, with no new lesions or ≥50% re

Group	Value	95% CI
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	33.3	0.84 – 90.57
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	33.3	0.84 – 90.57
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	0	0.0 – 45.93

Part 1b: CBR in Participants With TP53 WT Neuroblastoma Assessed According to INRC Secondary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

CBR=percentage of participants with CR, PR, or SD on 2 consecutive occasions ≥ 4 weeks apart per INRC. Primary tumor: CR=residual soft tissue at primary site is \<10 mm, with complete resolution of MIBG/FDG-PET uptake; PR= ≥30% decrease in longest diameter (LD) of primary site, with stable/improved MIBG/FDG-PET uptake; SD=Insufficient shrinkage for PR or increase for PD. PD= \>20% increase in LD from smallest sum \& minimum 5 mm increase in LD. Soft tissue \& bone metastases: CR=resolution of all disease sites; PR= ≥30% decrease in sum of non-primary target lesions, with no new lesions or ≥50%

Group	Value	95% CI
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	50.0	1.26 – 98.74
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	33.3	0.84 – 90.57
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	0	0.0 – 60.24

Part 1b: DOR in Participants With Neuroblastoma From SE Population Assessed According to INRC Secondary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

DOR=time from the first tumor assessment that supports a participant's objective response (CR or PR) to the time of PD or death from any cause (whichever occurs first), as determined by the investigator using INRC for participants with neuroblastoma. Primary tumor: CR=residual soft tissue at primary site is \<10 mm, with complete resolution of MIBG/FDG-PET uptake; PR= ≥30% decrease in LD of primary site and MIBG or FDG-PET uptake at primary site stable, improved, or resolved; PD= \>20% increase in LD from smallest sum \& minimum 5 mm increase in LD. Soft tissue \& bone metastases: CR = resolut

Group	Value	95% CI
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	4.5	NA – NA

Part 1b: DOR in in Participants With TP53 WT Neuroblastoma Assessed According to INRC Secondary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

Group	Value	95% CI
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	4.5	NA – NA

Part 1a: Progression Free Survival (PFS) in Participants With Solid Tumors From SE Population Assessed According to RECIST v1.1 or INRC Secondary · From screening (maximum 28 days) up to Cycle 5 (cycle length=28 days)

PFS was defined as the time from initiation of study drug to the first documented occurrence of PD or death from any cause (whichever occurs first), as determined by the investigator using RECIST or INRC. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference smallest sum on study (nadir), including baseline. Participants who had neuroblastoma were assessed by INRC. PD per INRC: Primary tumor = \>20% increase in LD from smallest sum \& minimum 5 mm increase in LD; Soft tissue \& bone metastases = new soft tissue lesions per CT/MRI or MIBG/FDG-PET avid bone

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	0.8	0.7 – 1.3
Part 1a: Idasanutlin 3 mg/kg	0.9	0.9 – NA
Part 1a: Idasanutlin 4.5 mg/kg	0.8	0.8 – 0.9
Part 1a: Idasanutlin 6.4 mg/kg	3.5	2.8 – NA
Part 1a: Idasanutlin 8 mg/kg	1.9	0.8 – NA

Part 1b: PFS in Participants With Neuroblastoma From SE Population Assessed According to INRC Secondary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

PFS was defined as the time from initiation of study drug to the first documented occurrence of PD or death from any cause (whichever occurs first), as determined by the investigator using INRC for participants with neuroblastoma. Primary tumor: PD= \>20% increase in LD from smallest sum \& minimum 5 mm increase in LD. Soft tissue \& bone metastases: PD=new soft tissue lesions per CT/MRI or MIBG/FDG-PET avid bone site. Bone marrow: PD=new tumor infiltration \>5% or infiltration increased \>2-fold with \>20% tumor infiltration.

Group	Value	95% CI
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	1.9	1.8 – NA
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	1.8	1.6 – NA
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	1.7	1.0 – NA

Part 1b: PFS in Participants With TP53 WT Neuroblastoma Assessed According to INRC Secondary · From screening (maximum 28 days) up to Cycle 6 (cycle length=28 days)

PFS was defined as the time from initiation of study drug to the first documented occurrence of PD or death from any cause (whichever occurs first), as determined by the investigator using INRC for participants with neuroblastoma. Primary tumor: PD= \>20% increase in LD from smallest sum \& minimum 5 mm increase in LD. Soft tissue \& bone metastases: PD=new soft tissue lesions per CT/MRI or MIBG/FDG-PET avid bone site. Bone marrow: PD=new tumor infiltration \>5% or infiltration increased \>2-fold with \>20% tumor infiltration.

Group	Value	95% CI
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	NA	1.8 – NA
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	1.8	1.6 – NA
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	1.7	1.5 – NA

Parts 1a and 1b: Overall Survival (OS) in SE Population Secondary · Up to approximately 29 months

OS was defined as the time from initiation of the study drug to death from any cause. Estimates for median OS were computed using Kaplan-Meier methodology

Group	Value	95% CI
Part 1a: Idasanutlin 2 mg/kg	5.6	2.1 – 20.8
Part 1a: Idasanutlin 3 mg/kg	12.5	1.1 – NA
Part 1a: Idasanutlin 4.5 mg/kg	3.6	2.3 – 9.8
Part 1a: Idasanutlin 6.4 mg/kg	16.4	7.2 – NA
Part 1a: Idasanutlin 8 mg/kg	5.4	3.4 – NA
Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy	NA	NA – NA
Part 1b: Idasanutlin (3.6mg/kg) +Chemotherapy	3.2	1.6 – NA
Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax	NA	2.9 – NA

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse Events: From screening up to 30 days after study treatment discontinuation (approximately 7 months) All-cause mortality: From screening up to end of follow up (approximately 29 months). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Part 1a: Idasanutlin 2 mg/kg

Serious: 6/8 (75%)

Deaths: 8/8

Part 1a: Idasanutlin 3 mg/kg

Serious: 3/3 (100%)

Deaths: 3/3

Part 1a: Idasanutlin 4.5 mg/kg

Serious: 4/6 (67%)

Deaths: 6/6

Part 1a: Idasanutlin 6.4 mg/kg

Serious: 2/3 (67%)

Deaths: 2/3

Part 1a: Idasanutlin 8 mg/kg

Serious: 6/6 (100%)

Deaths: 5/6

Part 1b: Idasanutlin (2.8mg/kg) +Chemotherapy

Serious: 2/3 (67%)

Deaths: 0/3

Part 1b: Idasanutlin (3.6 mg/kg) +Chemotherapy

Serious: 3/3 (100%)

Deaths: 2/3

Part 1b: Idasanutlin (3.6mg/kg) + Venetoclax

Serious: 4/6 (67%)

Deaths: 3/6

Serious adverse events (38 terms)

Reaction	System	Part 1a: Idasanutlin 2 mg/kg	Part 1a: Idasanutlin 3 mg/kg	Part 1a: Idasanutlin 4.5 m…	Part 1a: Idasanutlin 6.4 m…	Part 1a: Idasanutlin 8 mg/kg	Part 1b: Idasanutlin (2.8m…	Part 1b: Idasanutlin (3.6 …	Part 1b: Idasanutlin (3.6m…
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Leukopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Hematotoxicity	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Lymphopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Pancytopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Sinus tachycardia	Cardiac disorders	—	—	—	—	—	—	—	—
Hypoacusis	Ear and labyrinth disorders	—	—	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Stomatitis	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—	—	—
Appendicitis	Infections and infestations	—	—	—	—	—	—	—	—
Bacteraemia	Infections and infestations	—	—	—	—	—	—	—	—
Cellulitis	Infections and infestations	—	—	—	—	—	—	—	—
Central nervous system infection	Infections and infestations	—	—	—	—	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—	—	—	—	—
Sinusitis	Infections and infestations	—	—	—	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—	—	—	—
Eschar	Injury, poisoning and procedural complications	—	—	—	—	—	—	—	—
Femoral neck fracture	Injury, poisoning and procedural complications	—	—	—	—	—	—	—	—
Lymphocyte count decreased	Investigations	—	—	—	—	—	—	—	—
Platelet count decreased	Investigations	—	—	—	—	—	—	—	—

Other adverse events (112 terms — click to expand)

Reaction	System	Part 1a: Idasanutlin 2 mg/kg	Part 1a: Idasanutlin 3 mg/kg	Part 1a: Idasanutlin 4.5 m…	Part 1a: Idasanutlin 6.4 m…	Part 1a: Idasanutlin 8 mg/kg	Part 1b: Idasanutlin (2.8m…	Part 1b: Idasanutlin (3.6 …	Part 1b: Idasanutlin (3.6m…
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Diarrhea	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—	—	—
Leukopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—	—	—
Pain	General disorders	—	—	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—	—	—
Gamma-glutamyltransferase increased	Investigations	—	—	—	—	—	—	—	—
Lymphopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Oral pain	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Asthenia	General disorders	—	—	—	—	—	—	—	—
Mucosal inflammation	General disorders	—	—	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—	—	—
White blood cell count decreased	Investigations	—	—	—	—	—	—	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—
Hypoalbuminaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—
Hyponatraemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—
Hypophosphataemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—
Groin pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—	—	—
Lethargy	Nervous system disorders	—	—	—	—	—	—	—	—
Seizure	Nervous system disorders	—	—	—	—	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—
Gastritis	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Lip swelling	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Stomatitis	Gastrointestinal disorders	—	—	—	—	—	—	—	—
Non-cardiac chest pain	General disorders	—	—	—	—	—	—	—	—
Bacteraemia	Infections and infestations	—	—	—	—	—	—	—	—
Mucosal infection	Infections and infestations	—	—	—	—	—	—	—	—
Oral candidiasis	Infections and infestations	—	—	—	—	—	—	—	—
Skin infection	Infections and infestations	—	—	—	—	—	—	—	—

Most-reported serious reactions: Thrombocytopenia, Febrile neutropenia, Neutropenia, Leukopenia, Vomiting, Anemia, Hematotoxicity, Lymphopenia.

Data from ClinicalTrials.gov NCT04029688 adverse events section.

Sponsor's own description

This is a Phase I/II, multicenter, open-label, multi-arm study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of idasanutlin, administered as a single agent or in combination with chemotherapy or venetoclax, in pediatric and young adult participants with acute leukemias or solid tumors. This study is divided into three parts: Part 1 will begin with dose escalation of idasanutlin as a single agent in pediatric participants with relapsed or refractory solid tumors to identify the maximum tolerated dose (MTD)/maximum administered dose (MAD) and to characterize dose-limiting toxicities (DLTs). Following MTD/MAD identification, three separate safety run-in cohorts in neuroblastoma, acute myeloid leukemia (AML), and acute lymphoblastic leukemia (ALL) will be conducted to identify the recommended Phase 2 dose (RP2D) of idasanutlin in each combination, with chemotherapy or venetoclax. Part 2 will evaluate the safety and early efficacy of idasanutlin in combination with chemotherapy or venetoclax in newly enrolled pediatric and young adult participants in neuroblastoma, AML,and ALL cohorts at idasanutlin RP2D. Part 3 will potentially be conducted as an additional expansion phase of the idasanutlin combination cohorts in neuroblastoma, AML, or ALL for further response and safety assessment.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Regulated cell death (RCD) in cancer: key pathways and targeted therapies.
Peng F, Liao M, Qin R, Zhu S, et al · · 2022 · cited 586× · PMID 35963853 · DOI 10.1038/s41392-022-01110-y
Targeting p53 pathways: mechanisms, structures, and advances in therapy.
Wang H, Guo M, Wei H, Chen Y. · · 2023 · cited 580× · PMID 36859359 · DOI 10.1038/s41392-023-01347-1
Drugging p53 in cancer: one protein, many targets.
Hassin O, Oren M. · · 2023 · cited 532× · PMID 36216888 · DOI 10.1038/s41573-022-00571-8
Tumor biomarkers for diagnosis, prognosis and targeted therapy.
Zhou Y, Tao L, Qiu J, Xu J, et al · · 2024 · cited 379× · PMID 38763973 · DOI 10.1038/s41392-024-01823-2
Molecular targeting therapies for neuroblastoma: Progress and challenges.
Zafar A, Wang W, Liu G, Wang X, et al · · 2021 · cited 291× · PMID 33155698 · DOI 10.1002/med.21750
Recent advances in targeting the "undruggable" proteins: from drug discovery to clinical trials.
Xie X, Yu T, Li X, Zhang N, et al · · 2023 · cited 246× · PMID 37669923 · DOI 10.1038/s41392-023-01589-z
The Role of p53 Signaling in Colorectal Cancer.
Liebl MC, Hofmann TG. · · 2021 · cited 231× · PMID 33924934 · DOI 10.3390/cancers13092125
Targeting p53-MDM2 interaction by small-molecule inhibitors: learning from MDM2 inhibitors in clinical trials.
Zhu H, Gao H, Ji Y, Zhou Q, et al · · 2022 · cited 141× · PMID 35831864 · DOI 10.1186/s13045-022-01314-3

Verify or expand the search:

Other trials of Idasanutlin

Trials testing the same drug.

NCT03850535 — A Study Evaluating the Safety and Efficacy of Idasanutlin in Combination With Cytarabine and Daunorubicin in Patients Ne · Phase 1, PHASE2 · terminated
NCT03555149 — A Study Evaluating the Efficacy and Safety of Multiple Immunotherapy-Based Treatment Combinations in Patients With Metas · Phase 1, PHASE2 · terminated
NCT03566485 — Atezolizumab and Cobimetinib or Idasanutlin in Participants With Stage IV or Unresectable Recurrent Estrogen Receptor Po · Phase 1, PHASE2 · terminated
NCT03158389 — NCT Neuro Master Match - N²M² (NOA-20) · Phase 1, PHASE2 · completed
NCT03287245 — A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Idasanutlin Monotherapy in Participan · Phase 2 · terminated

Other recruiting trials for Acute Myeloid Leukemia (AML)

Currently open trials in the same condition.

NCT07463651 — MRD-guided Maintenance Post-HCT: Gilteritini vs Sorafenib · Phase 3 · recruiting
NCT07500441 — Digital PCR of CHIP and MR for MRD Monitoring After Allo-HSCT in AML · recruiting
NCT07410494 — Biomarker-Guided Allogeneic Single-Target or Dual-Target CAR-NK Cell Therapy for Advanced Solid Tumors · Phase 1, PHASE2 · recruiting
NCT06707493 — Ivosidenib as Post-HSCT Maintenance for AML · Phase 2 · recruiting
NCT07458542 — Effectiveness and Safety of ONUREG (Oral Azacitidine) in Chinese Patients With Acute Myeloid Leukemia · active not recruiting

Other Hoffmann-La Roche trials

Trials by the same sponsor.

NCT07503340 — A Study to Evaluate Pharmacokinetics, Safety, Tolerability, Immunogenicity and Pharmacodynamic Effects of Subcutaneous O · Phase 2 · not yet recruiting
NCT07298421 — A Study to Assess the Pharmacokinetics, Effectiveness and Safety of Afimkibart for Induction and Maintenance Therapy in · Phase 3 · recruiting
NCT07059273 — A COPD Data Registry for Participants With Frequent Exacerbations · not yet recruiting
NCT07416526 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A · Phase 3 · recruiting
NCT05199688 — A Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric · Phase 3 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04029688 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Hoffmann-La Roche
Last refreshed: 20 December 2024

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04029688.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing