Trial Outcomes & Findings for Testing Palbociclib (PD-0332991) as a Potential Targeted Treatment in Cancers With CCND1, 2, 3 Amplification (MATCH-Subprotocol Z1B) (NCT NCT04439201)

Last Updated: 2025-07-08

Results Overview

ORR is defined as the percentage of patients whose tumors have a complete or partial response to treatment among analyzable patients. Objective response is defined consistent with Response Evaluation Criteria in Solid Tumors version 1.1, the Cheson (2014) criteria for lymphoma patients, and the Response Assessment in Neuro-Oncology criteria for glioblastoma patients. Details about how to define complete response and partial response can be found in the master protocol. 90% two-sided binomial exact confidence interval is calculated for ORR.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

Tumor assessments occurred at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Results posted on

2025-07-08

Participant Flow

Subprotocol Z1B was activated on May 31, 2016. Forty patients were enrolled between August 2016 and December 2017.

To be eligible for this subprotocol, tumors had to contain both an amplification in CCND1, 2, or 3 and Rb expression. CCND amplification was defined as seven or more copies of the gene; Rb expression was defined as 1+ or greater staining by IHC. The mutation status was determined by a CLIA-approved assay performed in a NCI-MATCH approved laboratory for all patients in this arm. Confirmatory central testing was required in order for these patients to be included in the primary analysis.

Participant milestones

Participant milestones
Measure	Treatment (Palbociclib) Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Overall Study STARTED	40
Overall Study Eligible	37
Overall Study Started Protocol Therapy	38
Overall Study Eligible and Treated	36
Overall Study Mutation Status Confirmed	36
Overall Study Eligible, Treated and Mutation Status Confirmed	32
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	40

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Palbociclib) Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Overall Study Ineligible	2
Overall Study Never start protocol therapy	1
Overall Study Ineligible and never start protocol therapy	1
Overall Study Mutation status not confirmed	4
Overall Study Adverse Event	1
Overall Study Death	7
Overall Study Withdrawal by Subject	2
Overall Study Disease progression	20
Overall Study Alternative therapy	1
Overall Study Physician Decision	1

Baseline Characteristics

Testing Palbociclib (PD-0332991) as a Potential Targeted Treatment in Cancers With CCND1, 2, 3 Amplification (MATCH-Subprotocol Z1B)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Palbociclib) n=32 Participants Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Age, Continuous	62 years n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	28 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Tumor assessments occurred at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Population: Eligible, treated and mutation status confirmed

Outcome measures

Outcome measures
Measure	Treatment (Palbociclib) n=32 Participants Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Objective Response Rate (ORR)	0 Participants

SECONDARY outcome

Timeframe: Assessed at baseline, then every 2 cycles for the first 26 cycles, and every 3 cycles thereafter until disease progression, up to 3 years post registration, from which 6-month PFS rate is determined

Population: Eligible, treated and mutation status confirmed

Progression free survival is defined as time from treatment start date to date of progression or death from any cause, whichever occurs first. Disease progression was evaluated using the Response Evaluation Criteria in Solid Tumors version 1.1, the Cheson (2014) criteria for lymphoma patients, and the Response Assessment in Neuro-Oncology criteria for glioblastoma patients. Please refer to the protocol for detailed definitions of disease progression. 6 month PFS rate was estimated using the Kaplan-Meier method, which can provide a point estimate for any specific time point.

Outcome measures

Outcome measures
Measure	Treatment (Palbociclib) n=32 Participants Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
6-month Progression-free Survival (PFS) Rate	16 percentage of participants Interval 8.0 to 35.0

SECONDARY outcome

Timeframe: Assessed at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration

Population: Eligible, treated and mutation status confirmed

PFS was defined as time from treatment start date to date of disease progression or death from any causes, whichever occurred first. Median PFS was estimated using the Kaplan-Meier method. Disease progression was evaluated using the Response Evaluation Criteria in Solid Tumors version 1.1, the Cheson (2014) criteria for lymphoma patients, and the Response Assessment in Neuro-Oncology criteria for glioblastoma patients. Please refer to the protocol for detailed definitions of disease progression.

Outcome measures

Outcome measures
Measure	Treatment (Palbociclib) n=32 Participants Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Progression Free Survival (PFS)	1.8 months Interval 1.5 to 2.7

Adverse Events

Treatment (Palbociclib)

Serious events: 20 serious events

Other events: 25 other events

Deaths: 38 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Palbociclib) n=38 participants at risk Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Blood and lymphatic system disorders Anemia	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
General disorders Death NOS	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
General disorders Fatigue	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Diarrhea	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Nausea	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Small intestinal obstruction	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Infections and infestations Urinary tract infection	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Alanine aminotransferase increased	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Alkaline phosphatase increased	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Aspartate aminotransferase increased	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Blood bilirubin increased	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Lymphocyte count decreased	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Neutrophil count decreased	31.6% 12/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Platelet count decreased	10.5% 4/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations White blood cell decreased	18.4% 7/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Metabolism and nutrition disorders Dehydration	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Metabolism and nutrition disorders Hypokalemia	2.6% 1/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).

Other adverse events

Other adverse events
Measure	Treatment (Palbociclib) n=38 participants at risk Patients receive palbociclib 125 mg PO QD days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity Palbociclib: Given PO
Blood and lymphatic system disorders Anemia	31.6% 12/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
General disorders Fatigue	23.7% 9/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
General disorders Fever	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Skin and subcutaneous tissue disorders Rash maculo-papular	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Constipation	10.5% 4/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Dry mouth	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Mucositis oral	13.2% 5/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Nausea	18.4% 7/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Gastrointestinal disorders Vomiting	7.9% 3/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Alanine aminotransferase increased	7.9% 3/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Alkaline phosphatase increased	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Aspartate aminotransferase increased	10.5% 4/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Lymphocyte count decreased	21.1% 8/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Neutrophil count decreased	34.2% 13/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Platelet count decreased	28.9% 11/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Weight loss	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations White blood cell decreased	44.7% 17/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Investigations Investigations - Other, specify	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Metabolism and nutrition disorders Anorexia	13.2% 5/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Metabolism and nutrition disorders Hypoalbuminemia	7.9% 3/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Metabolism and nutrition disorders Hyponatremia	7.9% 3/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).
Vascular disorders Hypertension	5.3% 2/38 • Assessed every 28 days while on treatment and for 30 days after the end of treatment, up to 3 years post registration. All patients enrolled were included in the all-cause mortality analysis. Thirty-eight patients were included in the toxicity analysis (excluding two who did not receive treatment).

Additional Information

Study Statistician

ECOG-ACRIN Cancer Research Group

Phone: 617-632-3012

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60