Participant flow

Phase I Study of APX005M in Pediatric Central Nervous System Tumors · Source last updated 2026-08-13

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

groups

1. description

   Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.1 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

   id

   FG000

   title

   Stratum 1, Dose Level 1

2. description

   Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

   id

   FG001

   title

   Stratum 1, Dose Level 2

3. description

   Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

   id

   FG002

   title

   Stratum 1, Dose Level 3

4. description

   Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

   id

   FG003

   title

   Stratum 2, Dose Level 2

5. description

   Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.

   id

   FG004

   title

   Stratum 2, Dose Level 3

periods

1. drop Withdraws

   1. reasons

      1. group Id

         FG000

         num Subjects

         3

      2. group Id

         FG001

         num Subjects

         3

      3. group Id

         FG002

         num Subjects

         13

      4. group Id

         FG003

         num Subjects

         6

      5. group Id

         FG004

         num Subjects

         1

      type

      Lack of Efficacy

   2. reasons

      1. group Id

         FG000

         num Subjects

         0

      2. group Id

         FG001

         num Subjects

         0

      3. group Id

         FG002

         num Subjects

         1

      4. group Id

         FG003

         num Subjects

         0

      5. group Id

         FG004

         num Subjects

         4

      type

      Adverse Event

   3. reasons

      1. group Id

         FG000

         num Subjects

         0

      2. group Id

         FG001

         num Subjects

         0

      3. group Id

         FG002

         num Subjects

         1

      4. group Id

         FG003

         num Subjects

         0

      5. group Id

         FG004

         num Subjects

         0

      type

      Subject deemed ineligible after enrollment

   milestones

   1. achievements

      1. group Id

         FG000

         num Subjects

         3

      2. group Id

         FG001

         num Subjects

         3

      3. group Id

         FG002

         num Subjects

         15

      4. group Id

         FG003

         num Subjects

         6

      5. group Id

         FG004

         num Subjects

         5

      type

      STARTED

   2. achievements

      1. group Id

         FG000

         num Subjects

         0

      2. group Id

         FG001

         num Subjects

         0

      3. group Id

         FG002

         num Subjects

         0

      4. group Id

         FG003

         num Subjects

         0

      5. group Id

         FG004

         num Subjects

         0

      type

      COMPLETED

   3. achievements

      1. group Id

         FG000

         num Subjects

         3

      2. group Id

         FG001

         num Subjects

         3

      3. group Id

         FG002

         num Subjects

         15

      4. group Id

         FG003

         num Subjects

         6

      5. group Id

         FG004

         num Subjects

         5

      type

      NOT COMPLETED

   title

   Overall Study

pre Assignment Details

Patients with recurrent or refractory primary malignant CNS tumors were enrolled on stratum 1, and patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) were enrolled on stratum 2. A total of 32 patients were enrolled (31 eligible). Twenty-one (21) stratum 1 patients were enrolled; 1 of these patients was deemed to be ineligible as an eligibility assessment was not performed. Eleven (11) stratum 2 patients were enrolled (all deemed eligible).

recruitment Details

Patients ≥1 and ≤21 years of age with recurrent or refractory primary malignant central nervous system (CNS) tumors (Stratum 1) or newly diagnosed diffuse intrinsic pontine glioma (DIPG) (Stratum 2) were enrolled at Pediatric Brain Tumor Consortium (PBTC) member institutions. The first patient was enrolled on 3/1/2018 and the last patient was enrolled on 1/20/2023. Accrual was closed after the maximum tolerated dose/recommended phase II dose was determined for both strata.

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice