Neoplasms

Nervous System Diseases

All Conditions

Rare Diseases

Brain Neoplasms

Desmoplastic Small Round Cell Tumor

Central Nervous System Neoplasms

Nervous System Neoplasms

Brain Diseases

Central Nervous System Diseases

Sarcoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Soft Tissue Sarcoma

PBI-200 will be administered orally over continuous 28-day cycles

PBI-200

Chief Medical Officer

This is a first-in-human, open-label, multicenter, dose-escalation, safety, PK, and biomarker study of PBI-200 in subjects with NTRK-fusion-positive advanced or metastatic solid tumors.

This is a first-in-human, open-label, multicenter, dose-escalation, safety, PK, and biomarker study of PBI-200 in subjects with NTRK-fusion-positive advanced or metastatic solid tumors. Phase 1 will also include subjects with NTRK-amplified advanced or metastatic solid tumors or refractory EWSR1-WT1-fusion-positive desmoplastic small round cell tumors (DSRCTs).

Phase 1 is the dose-escalation portion of the study in which the evaluation of safety and tolerability and establishing the RP2D are primary objectives. Once the RP2D has been established, two expansion cohorts will open to accrual, a Non-Brain Primary Tumor cohort and a Primary Brian Tumor cohort.

Although this was intended to be a Phase 1/2 trial, the trial was terminated without proceeding to Phase 2.

Dose Escalation: Single-subject cohorts will be enrolled initially, until a subject has a Grade 2 or greater adverse event (AE), at which time a 3+3 design will be utilized. Dose escalation will continue until the maximum-tolerated dose (MTD) is reached, or the Recommended Phase 2 Dose (RP2D) is established.

Cohort Expansion: Two cohorts will be opened to accrual. Cohort A will enroll subjects with a non-brain primary tumor and Cohort B will enroll subjects with a primary brain tumor.

Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors

A Phase 1/2 Study of PBI-200 in Subjects With NTRK-Fusion-Positive Advanced or Metastatic Solid Tumors

Severity of AEs will be assessed according to the NCI CTCAE v5.0

Assessed using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1

Assessed using Response Assessment in Neuro-Oncology (RANO) criteria

Assessed by RECIST for subjects with non-brain primary tumors and by RANO for subjects with primary brain tumors

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors	NCT04901806	Solid Tumor, Ad... Brain Tumor, Pr... Desmoplastic Sm...	PBI-200	18 Years -	Pyramid Biosciences
Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors	NCT04901806	Solid Tumor, Ad... Brain Tumor, Pr... Desmoplastic Sm...	PBI-200	18 Years -	Pyramid Biosciences

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