Trial Outcomes & Findings for PCI-24781 in Combination With Doxorubicin to Treat Sarcoma (NCT NCT01027910)

NCT ID: NCT01027910

Last Updated: 2025-07-24

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 20 participants
• Primary outcome timeframe: up to 30 days after starting study drugs
• Results posted on: 2025-07-24

Participant Flow

Participant milestones

Measure	PCI-24781 + Doxorubicin Without Mandatory GCSF PCI-24781 + Doxorubicin without mandatory GCSF PCI-24781: Capsules taken orally for 5 consecutive days starting on Day 1 of each 3 week cycle Doxorubicin: Administered intravenously on Day 4 of each 3 week cycle	PCI-24781 + Doxorubicin With Mandatory GCSF PCI-24781 + Doxorubicin with mandatory GCSF PCI-24781: Capsules taken orally for 5 consecutive days starting on Day 1 of each 3 week cycle Doxorubicin: Administered intravenously on Day 4 of each 3 week cycle
Overall Study STARTED	6	14
Overall Study COMPLETED	6	14
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PCI-24781 in Combination With Doxorubicin to Treat Sarcoma

Baseline characteristics by cohort

Measure	PCI-24781 + Doxorubicin Without Mandatory GCSF n=6 Participants Study participants were enrolled into two arms. Arm A administered abexinostat and doxorubicin with optional GCSF support. Arm B administered abexinostat and doxorubicin with required GCSF support to all participants. The study uses the standard 3 + 3 phase I dose escalation design. Three cohorts of 3-6 participants were enrolled in each arm and separate inter-cohort dose escalations were performed in up to three cohorts of 3-6 participants enrolled sequentially until the maximum tolerated dose (MTD) of the combination abexinostat with doxorubicin, without (Arm A) mandatory G-CSF support was established.	PCI-24781 + Doxorubicin With Mandatory GCSF n=14 Participants Study participants were enrolled into two arms. Arm A administered abexinostat and doxorubicin with optional GCSF support. Arm B administered abexinostat and doxorubicin with required GCSF support to all participants. The study uses the standard 3 + 3 phase I dose escalation design. Three cohorts of 3-6 participants were enrolled in each arm and separate inter-cohort dose escalations were performed in up to three cohorts of 3-6 participants enrolled sequentially until the maximum tolerated dose (MTD) of the combination abexinostat with doxorubicin, with (Arm B) mandatory G-CSF support was established.	Total n=20 Participants Total of all reporting groups
Age, Continuous	52 years STANDARD_DEVIATION 14 • n=5 Participants	56 years STANDARD_DEVIATION 14 • n=7 Participants	54 years STANDARD_DEVIATION 14 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	7 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	7 Participants n=7 Participants	12 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 30 days after starting study drugs

Population: The two groups differ in that GCSF was offered if clinically indicated in arm 1 while it was administered to all participants in arm 2.

Outcome measures

Measure	PCI-24781 + Doxorubicin Without Mandatory GCSF n=6 Participants	PCI-24781 With Mandatory GCSF n=14 Participants
Maximum Tolerated Dose	15 mg/m2	45 mg/m2

SECONDARY outcome

Timeframe: 1 year

number of patients who experienced dose limiting toxicities

Outcome measures

Measure	PCI-24781 + Doxorubicin Without Mandatory GCSF n=6 Participants	PCI-24781 With Mandatory GCSF n=14 Participants
Dose Limiting Toxicities	2 participants	2 participants

SECONDARY outcome

Timeframe: 1 year

number of patients who demonstrated partial response to therapy as determined by RECIST v1.0 for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Outcome measures

Measure	PCI-24781 + Doxorubicin Without Mandatory GCSF n=6 Participants	PCI-24781 With Mandatory GCSF n=14 Participants
Number of Partial Responses (PR)	0 participants	3 participants

SECONDARY outcome

Timeframe: 2 years

Outcome measures

Measure	PCI-24781 + Doxorubicin Without Mandatory GCSF n=6 Participants	PCI-24781 With Mandatory GCSF n=14 Participants
Rate of Progression-free Survival at 6 Months in Participants Who Received PCI-24781/Doxorubicin Combination Administration.	1 participants	6 participants

Adverse Events

PCI-24781+Dox Without Mandated GCSF

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

PCI-24781+Dox With Mandated GCSF

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	PCI-24781+Dox Without Mandated GCSF n=6 participants at risk Patients in this arm were not mandated treatment with GCSF	PCI-24781+Dox With Mandated GCSF n=14 participants at risk Patients in this are were mandated treatment with GCSF
Blood and lymphatic system disorders grade 3 or 4 neutropenia	0.00% 0/6	14.3% 2/14 • Number of events 2
Infections and infestations infection	16.7% 1/6 • Number of events 1	0.00% 0/14
Blood and lymphatic system disorders thrombocytopenia	16.7% 1/6 • Number of events 1	0.00% 0/14

Other adverse events

Measure	PCI-24781+Dox Without Mandated GCSF n=6 participants at risk Patients in this arm were not mandated treatment with GCSF	PCI-24781+Dox With Mandated GCSF n=14 participants at risk Patients in this are were mandated treatment with GCSF
Gastrointestinal disorders diarrhea	16.7% 1/6 • Number of events 1	0.00% 0/14

Additional Information

Dr. Edwin Choy

Massachusetts General Hospital

Phone: 617-724-4000

Email: echoy@mgh.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place