NCT03236857

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

Brief summary

An open-label, global, multi-center study to evaluate the safety and pharmacokinetics of an active drug monotherapy, to determine the dose limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to assess the preliminary efficacy of an active drug in pediatric and young adult participants with relapsed or refractory malignancies.

Interventional study

Status:
Recruiting
Conditions:
Malignancies
Acute Lymphoblastic Leukemia (ALL)
Acute Myeloid Leukemia (AML)
Non-Hodgkin's Lymphoma
Neuroblastoma
Enrollment:
165 patients
Phase:
  • 1
  • 2
  • 3
  • 4
Protocol ID:
M13-833
Intervention model:
Single Group Assignment
Masking:
None (Open Label)
Purpose:
Treatment

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Eligibility criteria

Participant attributes:
Male and Female

Age:

0 to 25 Years.

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.

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 within 30 days

- Biologic agent (i.e., antibodies) for anti-neoplastic intent within 30 days

- CAR-T infusion or other cellular therapy within 30 days

- Anticancer therapy including blinatumomab or 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).

- Steroid therapy for anti-neoplastic intent within 5 days

- 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.

- Participants who have active, uncontrolled infections.

- Participants with malabsorption syndrome or any other condition that precludes enteral
administration.

All the cities where the clinical studies are located

Toronto - M5G 1X8

Montreal - H3T 1C5

Ontario

Quebec