A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
NCT ID: NCT03236857
Last Updated: 2023-05-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
143 participants
INTERVENTIONAL
2017-11-08
2023-04-19
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Venetoclax with or without chemotherapy
Venetoclax administered orally once daily (QD) with various doses and dosing regimens with or without chemotherapy at the discretion of the investigator. Allowed chemotherapy regimens as outlined in the study protocol.
chemotherapy
Dexamethasone and/or vincristine and/or pegasparaginase OR cytarabine and/or etoposide and/or pegasparaginase; tyrosine kinase inhibitor; cytarabine OR azacitidine OR decitabine; rituximab and/or dexamethasone and/or vincristine; cyclophosphamide and/or topotecan
venetoclax
Oral tablet for participants; Tablet for oral suspension (participants who cannot swallow a tablet)
Interventions
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chemotherapy
Dexamethasone and/or vincristine and/or pegasparaginase OR cytarabine and/or etoposide and/or pegasparaginase; tyrosine kinase inhibitor; cytarabine OR azacitidine OR decitabine; rituximab and/or dexamethasone and/or vincristine; cyclophosphamide and/or topotecan
venetoclax
Oral tablet for participants; Tablet for oral suspension (participants who cannot swallow a tablet)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participants must have adequate hepatic and kidney function.
* Participants less than or equal to 16 years of age must have performance status of Lansky greater than or equal to 50% and participants greater than 16 years of age must have performance status of Karnofsky greater than or equal to 50%.
* Participants with solid tumors (with the exception of neuroblastoma) must have adequate bone marrow function in Part 1.
* For the fifth cohort during Part 2 Cohort Expansion, participants with solid tumors must have evidence of BCL-2 expression (except participants with TCF3-HLF ALL).
Exclusion Criteria
* Participants who have central nervous system (CNS) disease with cranial involvement that requires radiation.
* Participants who have received any of the following within the listed time frame, prior to the first dose of study drug
* Inotuzumab ozogamicin or gemtuzumab ozogamicin within 30 days
* Biologic agent (i.e., antibodies) for anti-neoplastic intent within 30 days or 5 half-lives whichever is shorter.
* CAR-T infusion or other cellular therapy within 30 days
* Anticancer therapy including chemotherapy, radiation therapy, targeted small molecule agents, investigational agents within 14 days or 5 half-lives, whichever is shorter (Exceptions: Ph+ALL participants on Tyrosine Kinase Inhibitor (TKI) at Screening may enroll and remain on TKI therapy to control disease and TCF3-HLF ALL participants are allowed to have received chemotherapy within 14 days or 5 half-lives, whichever is shorter).
* Steroid therapy for anti-neoplastic intent within 5 days (with the exception of TCF3-HLF ALL participants).
* Requires ongoing hydroxyurea (hydroxyurea permitted up to first dose)
* Participants who are less than 100 days post-transplant, or greater than or equal to 100 days post-transplant with active graft versus host disease (GVHD), or are receiving immunosuppressant therapy within 7 days prior to first dose of study drug.
* Participants who are less than 6 weeks post-131 I-metaiodobenzylguanidine (mIBG) therapy.
* Participants who have received the following within 7 days prior to the first dose of study drug:
* Strong and moderate Cytochrome P450 3A (CYP3A) inhibitors (Part 1 Dose Determination);
* Strong and moderate CYP3A inducers (Part 1 Dose Determination and Part 2 Cohort Expansion).
* Participants who have not recovered from clinically significant adverse effect(s)/toxicity(s) of the previous therapy (Exception: Chemotherapy induced side effects that are expected to return to baseline in TCF3-HLF ALL participants).
* Participants who have active, uncontrolled infections.
* Participants with malabsorption syndrome or any other condition that precludes enteral administration.
* Participants with recent positive test for SARS-CoV-2 (COVID-19) and no follow up test with negative result cannot be enrolled. Participants with contact to persons with COVID-19 and participants with signs and symptoms for COVID-19 infection must be tested before enrolling.
0 Years
25 Years
ALL
No
Sponsors
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Roche-Genentech
INDUSTRY
AbbVie
INDUSTRY
Responsible Party
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Principal Investigators
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ABBVIE INC.
Role: STUDY_DIRECTOR
AbbVie
Locations
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Univ California, San Francisco /ID# 163460
San Francisco, California, United States
Children's Hospital Colorado /ID# 161551
Aurora, Colorado, United States
Children's Healthcare of Atlan /ID# 161552
Atlanta, Georgia, United States
Dana-Farber Cancer Institute /ID# 163440
Boston, Massachusetts, United States
Memorial Sloan Kettering Cancer Center-Koch Center /ID# 163444
New York, New York, United States
Cincinnati Children's Hospital /ID# 161550
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia /ID# 163445
Philadelphia, Pennsylvania, United States
St Jude Children's Research Hospital /ID# 163447
Memphis, Tennessee, United States
Primary Children's /ID# 164399
Salt Lake City, Utah, United States
Seattle Children's Hospital /ID# 163459
Seattle, Washington, United States
Medical College of Wisconsin /ID# 163461
Milwaukee, Wisconsin, United States
Sydney Children's Hospital /ID# 163148
Randwick, New South Wales, Australia
Queensland Children's Hospital /ID# 163146
South Brisbane, Queensland, Australia
Women and Childrens Hospital /ID# 163147
North Adelaide, South Australia, Australia
Royal Children's Hospital /ID# 163104
Parkville, Victoria, Australia
Hospital for Sick Children /ID# 163726
Toronto, Ontario, Canada
CHU Sainte-Justine /ID# 163725
Montreal, Quebec, Canada
AP-HM - Hopital de la Timone /ID# 161465
Marseille, Bouches-du-Rhone, France
Centre Leon Berard /ID# 163707
Lyon, Rhone, France
AP-HP - Hopital Armand-Trousseau /ID# 163728
Paris, , France
Robert Debre Hopital, FR /ID# 161464
Paris, , France
CHU Toulouse - Hôpital des enfants /ID# 163727
Toulouse, , France
Universitaetsklinikum Freiburg /ID# 164206
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Universitaetsklinikum Schleswig-Holstein Campus Kiel /ID# 161729
Kiel, Schleswig-Holstein, Germany
Charite Universitaetsklinikum Berlin - Campus Virchow /ID# 161730
Berlin, , Germany
Universitaetsklinikum Essen /ID# 164207
Essen, , Germany
Erasmus MC - Sophia /ID# 161579
Rotterdam, , Netherlands
Prinses Maxima Centrum /ID# 162670
Utrecht, , Netherlands
Kinderspital Zurich - Eleonorenstiftung /ID# 163037
Zurich, Canton of Zurich, Switzerland
Great Ormond Street Hospital for Children /ID# 169238
London, London, City of, United Kingdom
The Newcastle Upon Tyne Hospitals NHS Foundation Trust /ID# 162938
Newcastle upon Tyne, , United Kingdom
Countries
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References
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Karol SE, Khaw SL, Zwaan CM, Baruchel A, Bittencourt H, Cooper TM, Flotho C, Fraser C, Forlenza CJ, Goldsmith KC, Macy ME, Morgenstern DA, O'Brien MM, Petit A, Ziegler DS, Reinhardt D, Opferman JT, Rubnitz JE, Onishi M, Dunshee DR, Dunbar F, Vishwamitra D, Ross JA, Chen X, Unnebrink K, Kammerlander M, Salem AH, Palenski TL, Sunkersett G, Place AE. Venetoclax Alone or in Combination With Chemotherapy in Paediatric and Adolescent/Young Adult Patients With Relapsed/Refractory Acute Myeloid Leukaemia. Pediatr Blood Cancer. 2025 Jul;72(7):e31714. doi: 10.1002/pbc.31714. Epub 2025 Apr 23.
Place AE, Karol SE, Forlenza CJ, Cooper TM, Fraser C, Cario G, O'Brien MM, Gerber NU, Bourquin JP, Reinhardt D, Rubnitz JE, Opferman JT, Sunkersett G, Onishi M, Dunshee DR, Chen X, Unnebrink K, Vishwamitra D, Dunbar F, Badawi M, Ross JA, Loh ML. Venetoclax Combined With Chemotherapy in Pediatric and Adolescent/Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia. Pediatr Blood Cancer. 2025 Jun;72(6):e31630. doi: 10.1002/pbc.31630. Epub 2025 Mar 10.
Badawi M, Gopalakrishnan S, Engelhardt B, Palenski T, Karol SE, Rubnitz JE, Menon R, Salem AH. Dosing of Venetoclax in Pediatric Patients with Relapsed Acute Myeloid Leukemia: Analysis of Developmental Pharmacokinetics and Exposure-Response Relationships. Clin Ther. 2024 Oct;46(10):759-767. doi: 10.1016/j.clinthera.2024.09.008. Epub 2024 Oct 5.
Dalton KM, Krytska K, Lochmann TL, Sano R, Casey C, D'Aulerio A, Khan QA, Crowther GS, Coon C, Cai J, Jacob S, Kurupi R, Hu B, Dozmorov M, Greninger P, Souers AJ, Benes CH, Mosse YP, Faber AC. Venetoclax-based Rational Combinations are Effective in Models of MYCN-amplified Neuroblastoma. Mol Cancer Ther. 2021 Aug;20(8):1400-1411. doi: 10.1158/1535-7163.MCT-20-0710. Epub 2021 Jun 4.
Place AE, Goldsmith K, Bourquin JP, Loh ML, Gore L, Morgenstern DA, Sanzgiri Y, Hoffman D, Zhou Y, Ross JA, Prine B, Shebley M, McNamee M, Farazi T, Kim SY, Verdugo M, Lash-Fleming L, Zwaan CM, Vormoor J. Accelerating drug development in pediatric cancer: a novel Phase I study design of venetoclax in relapsed/refractory malignancies. Future Oncol. 2018 Sep;14(21):2115-2129. doi: 10.2217/fon-2018-0121. Epub 2018 Mar 29.
Other Identifiers
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2017-000439-14
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
M13-833
Identifier Type: -
Identifier Source: org_study_id
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