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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04901806




Registration number
NCT04901806
Ethics application status
Date submitted
20/05/2021
Date registered
26/05/2021
Date last updated
28/03/2024

Titles & IDs
Public title
Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors
Scientific title
A Phase 1/2 Study of PBI-200 in Subjects With NTRK-Fusion-Positive Advanced or Metastatic Solid Tumors
Secondary ID [1] 0 0
PBI-200-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Solid Tumor, Adult 0 0
Brain Tumor, Primary 0 0
Desmoplastic Small Round Cell Tumor 0 0
Condition category
Condition code
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Sarcoma (also see 'Bone') - soft tissue
Cancer 0 0 0 0
Children's - Other

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PBI-200

Experimental: Phase 1 Dose Escalation -

Experimental: Phase 2 Cohort Expansion -


Treatment: Drugs: PBI-200
PBI-200 will be administered orally over continuous 28-day cycles
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1: Number of patients with AEs
Timepoint [1] 0 0
Through study completion, estimated as an average of 36 months
Primary outcome [2] 0 0
Phase 1: Recommended Phase 2 Dose
Timepoint [2] 0 0
Approximately 12 months
Primary outcome [3] 0 0
Phase 2: Cohort A - Overall Response Rate (ORR)
Timepoint [3] 0 0
Through study completion, estimated as an average of 36 months
Primary outcome [4] 0 0
Phase 2: Cohort B - ORR
Timepoint [4] 0 0
Through study completion, estimated as an average of 36 months
Secondary outcome [1] 0 0
Phase 1: Area under the plasma drug concentration-time curve from 0 to 24 hours after one dose and after 28 doses
Timepoint [1] 0 0
29 days
Secondary outcome [2] 0 0
Phase 1: ORR
Timepoint [2] 0 0
Through study completion, estimated as an average of 36 months
Secondary outcome [3] 0 0
Duration of Response (DoR)
Timepoint [3] 0 0
Through study completion, estimated as an average of 36 months
Secondary outcome [4] 0 0
Progression-free Survival
Timepoint [4] 0 0
Through study completion, estimated as an average of 36 months

Eligibility
Key inclusion criteria
Key

- Subject has one of the following solid tumors which has progressed on or following at
least one systemic therapy regimen administered for advanced or metastatic disease or
for which no approved therapy exists:

- NTRK-fusion-positive, locally advanced (i.e., not amenable to surgical resection)
or metastatic solid tumor Note: Subjects with any grade of malignant glioma
previously treated with systemic therapy are eligible.

Phase 1

- NTRK-gene amplified, locally advanced or metastatic solid tumor

- EWSR1-WT1-positive DSRCTs.

- Subjects with NTRK-fusion-positive solid tumors other than primary brain tumors must
have previously received treatment with a TRK inhibitor, unless the subject does not
have access to TRK-inhibitor therapy (e.g., no TRK inhibitor is marketed and available
to the subject in the subject's country) or the subject has declined treatment with
available marketed TRK inhibitors.

- Subjects with NTRK-gene amplified solid tumors, primary brain tumors or
EWSR1-WT1-positive DSRCTs may have received prior treatment with a TRK inhibitor but
this is not required.

Phase 2

- Has measurable disease by RECIST v1.1 for subjects with non-brain primary tumors or
RANO criteria for subjects with primary brain tumors.

- Subjects with non-brain primary tumors must have previously received treatment with a
TRK inhibitor and a documented resistance mutation(s) (e.g., solvent front, gatekeeper
or xDFG mutation). Archival tissue from a prior biopsy taken after the subject
completed TRK inhibitor treatment but prior to additional systemic therapy may be used
to meet this eligibility criterion with Medical Monitor approval.

- Subjects with primary brain tumors may have received prior treatment with a TRK
inhibitor but this is not required. Biopsies of brain tumors are not required for
eligibility.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Cytotoxic chemotherapy, biologic agent, investigational agent, or radiation therapy =
3 weeks prior to the first dose of PBI-200 (6 weeks for nitrosoureas).

- Subjects with either primary brain tumors or brain metastasis must have completed
brain radiation therapy 12 weeks prior to the brain MRI obtained within 4 weeks
of the first dose of PBI-200.

- Small-molecule kinase inhibitors or hormonal agents = 14 days and within 5 half-lives
prior to the first dose of PBI-200.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/07/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/07/2023
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment postcode(s) [1] 0 0
3000 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Tennessee
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Denmark
State/province [9] 0 0
Copenhagen
Country [10] 0 0
France
State/province [10] 0 0
Bordeaux
Country [11] 0 0
France
State/province [11] 0 0
Lyon
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
France
State/province [13] 0 0
Poitiers
Country [14] 0 0
France
State/province [14] 0 0
Villejuif
Country [15] 0 0
Germany
State/province [15] 0 0
Frankfurt am Main
Country [16] 0 0
Germany
State/province [16] 0 0
Heidelberg
Country [17] 0 0
Germany
State/province [17] 0 0
Herne
Country [18] 0 0
Hong Kong
State/province [18] 0 0
Pok Fu Lam
Country [19] 0 0
Hong Kong
State/province [19] 0 0
Sha Tin
Country [20] 0 0
Italy
State/province [20] 0 0
Ancona
Country [21] 0 0
Italy
State/province [21] 0 0
Milano
Country [22] 0 0
Italy
State/province [22] 0 0
Napoli
Country [23] 0 0
Italy
State/province [23] 0 0
Verona
Country [24] 0 0
Korea, Republic of
State/province [24] 0 0
Gyeonggi-do
Country [25] 0 0
Korea, Republic of
State/province [25] 0 0
Seoul
Country [26] 0 0
Singapore
State/province [26] 0 0
Singapore
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
L'Hospitalet De Llobregat
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Spain
State/province [30] 0 0
Sant Cugat Del Vallès
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Manchester
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Sutton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Pyramid Biosciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04901806
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Chief Medical Officer
Address 0 0
Pyramid Biosciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04901806