A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies

• Register: ClinicalTrials.gov
• Last refreshed on: 29 May 2023
• Main ID: NCT03236857
• Date of registration: 31/07/2017
• Prospective Registration: Yes
• Primary sponsor: AbbVie
• Public title: A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
• Scientific title: A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
• Date of first enrolment: November 8, 2017
• Target sample size: 143
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03236857
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Australia, Canada, France, Germany, Netherlands, Switzerland, United Kingdom, United States

Contacts

• Name: ABBVIE INC.
• Affiliation: AbbVie

Key inclusion & exclusion criteria

Inclusion Criteria:

- Participants must have relapsed or refractory cancer.

- 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 with primary brain tumors or disease metastatic to the brain.

- 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.



Age minimum: 0 Years
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Acute Myeloid Leukemia (AML)

Neuroblastoma

Acute Lymphoblastic Leukemia (ALL)

Malignancies

Non-Hodgkin's Lymphoma

Intervention(s)

Drug: venetoclax

Drug: chemotherapy

Primary Outcome(s)

Number of Participants Experiencing Adverse Events [Time Frame: Up to 9 months]

AUC0-24 Post-Dose of Venetoclax [Time Frame: Up to approximately 2 weeks]

Cmax of Venetoclax [Time Frame: Up to approximately 2 weeks]

Number of Participants With Dose Limiting Toxicities (DLT) of Venetoclax Monotherapy [Time Frame: First 21 days venetoclax monotherapy]

Recommended Phase 2 dose (RPTD) of Venetoclax [Time Frame: First 21 days venetoclax monotherapy]

Tmax of venetoclax [Time Frame: Up to approximately 2 weeks]

Secondary Outcome(s)

Complete Response (CR) Rate [Time Frame: Up to 9 months]

Partial Response (PR) Rate [Time Frame: Up to 9 months]

Objective Response Rate (ORR) [Time Frame: Up to 9 months]

Secondary ID(s)

2017-000439-14

M13-833

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Roche-Genentech