Trial Outcomes & Findings for The Purpose of the Study is to Continue to Provide Pemigatinib to Patients With Advanced Malignancies. (NCT NCT04949191)

Last Updated: 2024-09-19

Results Overview

An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. TEAEs were defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

up to at least 30 days after the last dose of study treatment or until toxicities resolve, return to baseline, or are deemed irreversible, whichever was longer (up to 1010 days)

Results posted on

2024-09-19

Participant Flow

Participants who were actively receiving treatment with pemigatinib (monotherapy or in combination with pembrolizumab) under a parent protocol, were receiving clinical benefit in those trials, and who did not have access to pemigatinib (as monotherapy or as combination therapy) outside of a clinical trial were enrolled.

This study was conducted at 9 sites in 4 countries.

Participant milestones

Participant milestones
Measure	Pemigatinib 6 mg Monotherapy Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 6 milligrams (mg) once daily (QD), either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 9 mg Monotherapy Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 9 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 13.5 mg Monotherapy articipants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 13.5 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib Combination Participants originally enrolled in parent study 54828-101 (NCT02393248), who received clinical benefit from the combination of pemigatinib plus pembrolizumab, and who did not have access to the combination outside of a clinical trial received received the combination of pemigatinib and pembrolizumab. Participants received pembrolizumab intravenously at 200 mg once every 3 weeks on Day 1 of each 21-day cycle. The dose could have been adjusted for toxicity management per commercial labeling. The investigator could have interrupted, modified, or discontinued pembrolizumab with medical monitor approval. Participants self-administered oral pemigatinib 13.5 mg QD on a 2-weeks-on therapy and 1-week-off therapy schedule. The treatment cycle consisted of: Days 1 through 14: pemigatinib QD; Days 15 through 21: treatment break. It was permissible to continue pemigatinib administration during the toxicity break of pembrolizumab.
Overall Study STARTED	2	5	2	1
Overall Study COMPLETED	1	3	1	0
Overall Study NOT COMPLETED	1	2	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Pemigatinib 6 mg Monotherapy Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 6 milligrams (mg) once daily (QD), either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 9 mg Monotherapy Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 9 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 13.5 mg Monotherapy articipants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 13.5 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib Combination Participants originally enrolled in parent study 54828-101 (NCT02393248), who received clinical benefit from the combination of pemigatinib plus pembrolizumab, and who did not have access to the combination outside of a clinical trial received received the combination of pemigatinib and pembrolizumab. Participants received pembrolizumab intravenously at 200 mg once every 3 weeks on Day 1 of each 21-day cycle. The dose could have been adjusted for toxicity management per commercial labeling. The investigator could have interrupted, modified, or discontinued pembrolizumab with medical monitor approval. Participants self-administered oral pemigatinib 13.5 mg QD on a 2-weeks-on therapy and 1-week-off therapy schedule. The treatment cycle consisted of: Days 1 through 14: pemigatinib QD; Days 15 through 21: treatment break. It was permissible to continue pemigatinib administration during the toxicity break of pembrolizumab.
Overall Study Non-compliance with Study Drug	1	0	0	0
Overall Study Progressive Disease	0	1	0	0
Overall Study Transitioned to Commercial Pemigatinib	0	1	0	0
Overall Study Sponsor Terminated Study	0	0	1	1

Baseline Characteristics

The Purpose of the Study is to Continue to Provide Pemigatinib to Patients With Advanced Malignancies.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pemigatinib 6 mg and 13.5 mg Monotherapy n=4 Participants Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 6 milligrams (mg) or 13.5 mg once daily (QD), either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 9 mg Monotherapy n=5 Participants Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 9 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib Combination n=1 Participants Participants originally enrolled in parent study 54828-101 (NCT02393248), who received clinical benefit from the combination of pemigatinib plus pembrolizumab, and who did not have access to the combination outside of a clinical trial received received the combination of pemigatinib and pembrolizumab. Participants received pembrolizumab intravenously at 200 mg once every 3 weeks on Day 1 of each 21-day cycle. The dose could have been adjusted for toxicity management per commercial labeling. The investigator could have interrupted, modified, or discontinued pembrolizumab with medical monitor approval. Participants self-administered oral pemigatinib 13.5 mg QD on a 2-weeks-on therapy and 1-week-off therapy schedule. The treatment cycle consisted of: Days 1 through 14: pemigatinib QD; Days 15 through 21: treatment break. It was permissible to continue pemigatinib administration during the toxicity break of pembrolizumab.	Total n=10 Participants Total of all reporting groups
Age, Continuous	64.5 years STANDARD_DEVIATION 17.20 • n=5 Participants	61.2 years STANDARD_DEVIATION 6.30 • n=7 Participants	NA years STANDARD_DEVIATION NA • n=5 Participants	NA years STANDARD_DEVIATION NA • n=4 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	5 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Race/Ethnicity, Customized White/Caucasian	2 Participants n=5 Participants	4 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants
Race/Ethnicity, Customized Black/African-American	1 Participants n=5 Participants	0 Participants n=7 Participants	NA Participants n=5 Participants	NA Participants n=4 Participants

PRIMARY outcome

Timeframe: up to at least 30 days after the last dose of study treatment or until toxicities resolve, return to baseline, or are deemed irreversible, whichever was longer (up to 1010 days)

Population: Safety Population: all participants who received at least 1 dose of study treatment in this study

Outcome measures

Outcome measures
Measure	Pemigatinib 6 mg Monotherapy n=2 Participants Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 6 milligrams (mg) once daily (QD), either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 9 mg Monotherapy n=5 Participants Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 9 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 13.5 mg Monotherapy n=2 Participants Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 13.5 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib Combination n=1 Participants Participants originally enrolled in parent study 54828-101 (NCT02393248), who received clinical benefit from the combination of pemigatinib plus pembrolizumab, and who did not have access to the combination outside of a clinical trial received received the combination of pemigatinib and pembrolizumab. Participants received pembrolizumab intravenously at 200 mg once every 3 weeks on Day 1 of each 21-day cycle. The dose could have been adjusted for toxicity management per commercial labeling. The investigator could have interrupted, modified, or discontinued pembrolizumab with medical monitor approval. Participants self-administered oral pemigatinib 13.5 mg QD on a 2-weeks-on therapy and 1-week-off therapy schedule. The treatment cycle consisted of: Days 1 through 14: pemigatinib QD; Days 15 through 21: treatment break. It was permissible to continue pemigatinib administration during the toxicity break of pembrolizumab.
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)	1 Participants	5 Participants	1 Participants	1 Participants

Adverse Events

Pemigatinib 6 mg Monotherapy

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Pemigatinib 9 mg Monotherapy

Serious events: 2 serious events

Other events: 5 other events

Deaths: 0 deaths

Pemigatinib 13.5 mg Monotherapy

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Pemigatinib Combination

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Pemigatinib 6 mg Monotherapy n=2 participants at risk Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 6 milligrams (mg) once daily (QD), either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 9 mg Monotherapy n=5 participants at risk Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 9 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib 13.5 mg Monotherapy n=2 participants at risk Participants originally enrolled in a parent study (54828-201 \[NCT02872714\], 54828-202 \[NCT02924376\], or 54828-207 \[NCT03822117\]), who received clinical benefit from pemigatinib, and who did not have access to pemigatinib outside of a clinical trial self-administered oral pemigatinib 13.5 mg QD, either continuously in 21-day cycles or on a 2-weeks-on therapy/1-week-off therapy schedule in 21-day cycles. Pemigatinib was administered at the same dose received in the parent studies until documented disease progression or unacceptable toxicity.	Pemigatinib Combination n=1 participants at risk Participants originally enrolled in parent study 54828-101 (NCT02393248), who received clinical benefit from the combination of pemigatinib plus pembrolizumab, and who did not have access to the combination outside of a clinical trial received received the combination of pemigatinib and pembrolizumab. Participants received pembrolizumab intravenously at 200 mg once every 3 weeks on Day 1 of each 21-day cycle. The dose could have been adjusted for toxicity management per commercial labeling. The investigator could have interrupted, modified, or discontinued pembrolizumab with medical monitor approval. Participants self-administered oral pemigatinib 13.5 mg QD on a 2-weeks-on therapy and 1-week-off therapy schedule. The treatment cycle consisted of: Days 1 through 14: pemigatinib QD; Days 15 through 21: treatment break. It was permissible to continue pemigatinib administration during the toxicity break of pembrolizumab.
Renal and urinary disorders Acute kidney injury	0.00% 0/2 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	20.0% 1/5 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/2 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/1 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.
Cardiac disorders Cardiac failure	50.0% 1/2 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/5 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/2 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/1 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.
Nervous system disorders Seizure	0.00% 0/2 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	20.0% 1/5 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/2 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.	0.00% 0/1 • up to 1010 days Treatment-emergent adverse events (TEAEs), defined as AEs either reported for the first time or the worsening of pre-existing events after the first dose of study drug and until 30 days after the last dose of study drug, have been reported.

Other adverse events

Additional Information

Study Director

Incyte Corporation

Phone: 1-855-463-3463

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
Publication restrictions are in place

Restriction type: OTHER