NCT01411267 is a Phase 1 clinical trial of AC220 in Lymphoblastic Leukemia, Acute, Childhood, sponsored by Therapeutic Advances in Childhood Leukemia Consortium.

Who is sponsoring NCT01411267?

NCT01411267 is sponsored by Therapeutic Advances in Childhood Leukemia Consortium.

What drugs are being tested in NCT01411267?

Interventions in NCT01411267: AC220, Cytarabine, Etoposide, Methotrexate.

What condition does NCT01411267 study?

NCT01411267 studies Lymphoblastic Leukemia, Acute, Childhood, Myelogenous Leukemia, Acute, Childhood.

Is NCT01411267 still recruiting?

NCT01411267 is currently completed. Last updated 4 April 2022.

Are results published for NCT01411267?

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

Last reviewed 2022-04-04 · How we verify

NCT01411267

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

AC220 for Children With Relapsed/Refractory ALL or AML

Completed Phase 1 Results posted Last updated 4 April 2022

What this trial tests

Phase 1 trial testing AC220 in Lymphoblastic Leukemia, Acute, Childhood in 24 participants. Completed in 12 September 2013.

Timeline

1 September 2011

Primary endpoint
12 September 2013

12 September 2013

Quick facts

Lead sponsor	Therapeutic Advances in Childhood Leukemia Consortium
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	24
Start date	1 September 2011
Primary completion	12 September 2013
Estimated completion	12 September 2013
Sites	11 locations across United States

Drugs / interventions tested

AC220
Cytarabine — full drug profile →
Etoposide (etoposide) — full drug profile →
Methotrexate — full drug profile →

Conditions studied

Lymphoblastic Leukemia, Acute, Childhood — all drugs for Lymphoblastic Leukemia, Acute, Childhood →
Myelogenous Leukemia, Acute, Childhood — all drugs for Myelogenous Leukemia, Acute, Childhood →

Sponsor

Therapeutic Advances in Childhood Leukemia Consortium

Who can join

Adults 1 Month to 21, any sex, with Lymphoblastic Leukemia, Acute, Childhood or Myelogenous Leukemia, Acute, Childhood. 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.

The Dose of AC220 That is Safe and Biologically Active When Given in Sequential Combination With Ara-C/Etoposide Primary · 4 weeks from therapy start

The incidence of dose limiting toxicity (DLT) will be measured. The maximum tolerated dose will be the highest study dose at which 1 or fewer of six patients experience DLT during cycle 1 of therapy. Plasma inhibitor activity (PIA) will be measured Pre-treatment and on Days 7, 14, 21 and 28 of Course 1. For the MTD to be considered biologically active, we will require that 7 of 9 patients achieve PIA of \> 90% at 3 of 4 trough time points.

Group	Value	95% CI
ALL AC220 @ 25mg/m^2/Day (Dose Level 1)	0
AML AC220 @ 25mg/m^2/Day (Dose Level 1)	3
ALL AC220 @ 40mg/m^2/Day (Dose Level 2)	1
AML AC220 @ 40mg/m^2/Day (Dose Level 2)	4
ALL AC220 @ 60mg/m^2/Day (Dose Level 3)	0
AML AC220 @ 60mg/m^2/Day (Dose Level 3)	8
ALL AC220 @ 25mg/m^2/Day (Dose Level 1)	0
AML AC220 @ 25mg/m^2/Day (Dose Level 1)	0
ALL AC220 @ 40mg/m^2/Day (Dose Level 2)	1
AML AC220 @ 40mg/m^2/Day (Dose Level 2)	0
ALL AC220 @ 60mg/m^2/Day (Dose Level 3)	0
AML AC220 @ 60mg/m^2/Day (Dose Level 3)	1
ALL AC220 @ 25mg/m^2/Day (Dose Level 1)	0
AML AC220 @ 25mg/m^2/Day (Dose Level 1)	0
ALL AC220 @ 40mg/m^2/Day (Dose Level 2)	0
AML AC220 @ 40mg/m^2/Day (Dose Level 2)	1
ALL AC220 @ 60mg/m^2/Day (Dose Level 3)	2
AML AC220 @ 60mg/m^2/Day (Dose Level 3)	3

Disease Response Secondary · 10 weeks

Possible outcomes are: Complete Remission (CR), Complete Remission without Platelet Recovery (CRp), complete response with incomplete hematologic recovery (CRi), Stable Disease, Progressive Disease, Induction Death, or Relapse

Group	Value	95% CI
Patients With ALL	0
Patients With AML Having FLT3-WT	1
Patients With AML Having FLT3-ITD	1
Patients With ALL	0
Patients With AML Having FLT3-WT	0
Patients With AML Having FLT3-ITD	1
Patients With ALL	0
Patients With AML Having FLT3-WT	0
Patients With AML Having FLT3-ITD	1
Patients With ALL	1
Patients With AML Having FLT3-WT	5
Patients With AML Having FLT3-ITD	4

Count of Participants According to Inhibition of FLT3 Phosphorylation Secondary · 4 weeks from therapy start

PIA samples will be collected pre-treatment and on Days 7, 14, 21 and 28 of Course 1.

Group	Value	95% CI
Patients Receiving Protocol Therapy	9
Patients Receiving Protocol Therapy	9
Patients Receiving Protocol Therapy	1

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events and suspected adverse reactions will be collected and reported on the electronic CRFs beginning with the first dose of study therapy until 30 days following the last dose of study therapy (a period of approximately 90 days).. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

ALL Dose Level 1

Serious: 0

Deaths: 0

AML Dose Level 1

Serious: 2/3 (67%)

Deaths: 2/3

ALL Dose Level 2

Serious: 1/2 (50%)

Deaths: 2/2

AML Dose Level 2

Serious: 3/5 (60%)

Deaths: 3/5

ALL Dose Level 3

Serious: 0/2 (0%)

Deaths: 1/2

AML Dose Level 3

Serious: 4/10 (40%)

Deaths: 3/10

Serious adverse events (21 terms)

Reaction	System	ALL Dose Level 1	AML Dose Level 1	ALL Dose Level 2	AML Dose Level 2	ALL Dose Level 3	AML Dose Level 3
Infections and infestations - Other, Not Specified	Infections and infestations	—	—	—	—	—	—
Skin infection	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Anaphylaxis	Immune system disorders	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—
Fever	General disorders	—	—	—	—	—	—
Hypokalemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—	—	—
Lipase increased	Investigations	—	—	—	—	—	—
Lung infection	Infections and infestations	—	—	—	—	—	—
Pancreatitis	Gastrointestinal disorders	—	—	—	—	—	—
Pleural effusion	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Pulmonary edema	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Respiratory failure	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—	—	—
Infection and Infestations - Rhinovirus	Blood and lymphatic system disorders	—	—	—	—	—	—
Infections and Infestations - Staphylococcus epidermidis	Blood and lymphatic system disorders	—	—	—	—	—	—
Infections and Infestations - streptococcal pharyngitis	Blood and lymphatic system disorders	—	—	—	—	—	—
Infections and Infestations - Pseudomonas	Blood and lymphatic system disorders	—	—	—	—	—	—
Chest wall pain	General disorders	—	—	—	—	—	—
Skin & subcutaneous tissue disorder-Other	Skin and subcutaneous tissue disorders	—	—	—	—	—	—

Other adverse events (57 terms — click to expand)

Reaction	System	ALL Dose Level 1	AML Dose Level 1	ALL Dose Level 2	AML Dose Level 2	ALL Dose Level 3	AML Dose Level 3
Hypokalemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypomagnesemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Platelet count decreased	Blood and lymphatic system disorders	—	—	—	—	—	—
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—
Neutrophil count decreased	Blood and lymphatic system disorders	—	—	—	—	—	—
White blood cell decreased	Investigations	—	—	—	—	—	—
Lymphocyte count decreased	Blood and lymphatic system disorders	—	—	—	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Fever	General disorders	—	—	—	—	—	—
Hyperglycemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—
Anorexia	Metabolism and nutrition disorders	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—	—	—
Mucositis oral	Gastrointestinal disorders	—	—	—	—	—	—
Pain	General disorders	—	—	—	—	—	—
Rash maculo-papular	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Rectal pain	Gastrointestinal disorders	—	—	—	—	—	—
Blood Culture infection	Infections and infestations	—	—	—	—	—	—
Headache	General disorders	—	—	—	—	—	—
Activated partial thromboplastin time prolonged	Investigations	—	—	—	—	—	—
Alkaline phosphatase increased	Investigations	—	—	—	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—
Catheter related infection	Infections and infestations	—	—	—	—	—	—
Creatinine increased	Investigations	—	—	—	—	—	—
Diarrhea	Gastrointestinal disorders	—	—	—	—	—	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Hypermagnesemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—
Hypoalbuminemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypocalcemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hyponatremia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypophosphatemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Infections and infestations - Other - Not otherwise Specified	Infections and infestations	—	—	—	—	—	—
Lung infection	Infections and infestations	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—

Most-reported serious reactions: Infections and infestations - Other, Not Specified, Skin infection, Anaphylaxis, Blood bilirubin increased, Febrile neutropenia, Fever, Hypokalemia, Hypotension.

Data from ClinicalTrials.gov NCT01411267 adverse events section.

Sponsor's own description

This is a phase I study of the investigational drug AC220 combined with cytarabine and etoposide in pediatric patients with relapsed acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML).

Publications & conference data

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

Recent advances in targeted therapies in acute myeloid leukemia.
Bhansali RS, Pratz KW, Lai C. · · 2023 · cited 151× · PMID 36966300 · DOI 10.1186/s13045-023-01424-6
Childhood acute lymphoblastic leukemia: Integrating genomics into therapy.
Tasian SK, Loh ML, Hunger SP. · · 2015 · cited 54× · PMID 26194091 · DOI 10.1002/cncr.29573
The Biology and Targeting of FLT3 in Pediatric Leukemia.
Annesley CE, Brown P. · · 2014 · cited 51× · PMID 25295230 · DOI 10.3389/fonc.2014.00263
Novel agents for the treatment of childhood acute leukemia.
Annesley CE, Brown P. · · 2015 · cited 41× · PMID 25830014 · DOI 10.1177/2040620714565963
Advances in biology of acute lymphoblastic leukemia (ALL) and therapeutic implications.
Mohseni M, Uludag H, Brandwein JM. · · 2018 · cited 34× · PMID 30697448
Molecular therapeutic approaches for pediatric acute myeloid leukemia.
Tasian SK, Pollard JA, Aplenc R. · · 2014 · cited 29× · PMID 24672775 · DOI 10.3389/fonc.2014.00055
Targeting FLT3 Signaling in Childhood Acute Myeloid Leukemia.
Sexauer AN, Tasian SK. · · 2017 · cited 24× · PMID 29209600 · DOI 10.3389/fped.2017.00248
Molecular profiling of childhood cancer: Biomarkers and novel therapies.
Saletta F, Wadham C, Ziegler DS, Marshall GM, et al · · 2014 · cited 24× · PMID 26675306 · DOI 10.1016/j.bbacli.2014.06.003

Verify or expand the search:

Other recruiting trials for Lymphoblastic Leukemia, Acute, Childhood

Currently open trials in the same condition.

NCT05043571 — CARTALL: Chimeric-Antigen Receptor (CAR) T-Cell Therapy for Relapsed/ Refractory T-Lineage Acute Lymphoblastic Leukaemia · Phase 1 · recruiting
NCT05038696 — ALaCART-B: Acute Leukemia and Chimeric Antigen Receptor-T Cell Therapy for B-lymphoblastic Leukemia. · Phase 1 · recruiting
NCT03817320 — PO Ixazomib in Combination With Chemotherapy for Childhood Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymph · Phase 1, PHASE2 · active not recruiting

Other Therapeutic Advances in Childhood Leukemia Consortium trials

Trials by the same sponsor.

NCT05476770 — Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies · Phase 1 · recruiting
NCT03825367 — Nivolumab in Combination With 5-azacytidine in Childhood Relapsed/Refractory AML · Phase 1, PHASE2 · unknown
NCT03817320 — PO Ixazomib in Combination With Chemotherapy for Childhood Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymph · Phase 1, PHASE2 · active not recruiting
NCT03263936 — Epigenetic Reprogramming in Relapse/Refractory AML · Phase 1 · completed
NCT02879643 — Vincristine Sulfate Liposome Injection (Marqibo®) in Combination With UK ALL R3 Induction Chemotherapy · Phase 1 · completed

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 NCT01411267 (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 Therapeutic Advances in Childhood Leukemia Consortium
Last refreshed: 4 April 2022

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/NCT01411267.

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