Trial Outcomes & Findings for Safety and Efficacy of Pemigatinib in Patients With High-risk Urothelial Cancer After Radical Surgery (NCT NCT04294277)
NCT ID: NCT04294277
Last Updated: 2025-04-18
Results Overview
In the protocol, RFS at Year 2 was defined as the time from the start of treatment until disease relapse by Investigator determination, study-end or lost to follow-up, or death due to any cause. Due to the Covid-19 crisis causing significant delay of recruitment and the fact that we needed much more patients to identify patients with FGFR3 alterations, the study recruitment was prematurely stopped in November 2021 when 46 patients were screened and only 4 among 42 tested (9.5%) patients were identified with FGFR3 alterations. Two of the FGFR3 positive patients started treatment and the others were ineligible for the study. Since patients withdrawing from the study before completing the study period of 2 years cannot be included in the calculation of the 2-year RFS rate, the outcome results through premature study-end are presented.
TERMINATED
PHASE2
2 participants
Mean Relapse-free survival rate from start of treatment through premature study-end with a maximal follow-up of 62 weeks.
2025-04-18
Participant Flow
In 2020, the study was started in 5 Italian centers and recruitment commenced. The first patient was screened in July 2020.
Participant milestones
| Measure |
Treatment Arm
Treatment with Pemigatinib at the protocol-defined dose administered orally once daily as continuous therapy schedule until 12 months.
Pemigatinib: At Day 1, prior to the start of treatment, results from screening visit evaluations are reviewed to determine eligibility requirements. Subsequent visits during treatment phase will take place at Day 8, Day 15 and Day 21 and subsequently at 3 week intervals. Timing of subsequent visits can be prolonged to max. 9 week intervals if no problems exist during the first 12 weeks of therapy. Subjects will self-administer study drug using an oral QD regimen in a continuous dosing schedule. Each dose of Pemigatinib should be taken first thing in the morning upon waking or after a 2-hour fast; subjects should then fast for an additional 1 hour after taking study drug. The starting dose is 13.5 mg Pemigatinib. Study treatment will continue until 12 months, or until the evidence of disease relapse or onset of unacceptable toxicity.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Treatment Arm
n=2 Participants
Treatment with Pemigatinib at the protocol-defined dose administered orally once daily as continuous therapy schedule until 12 months.
Pemigatinib: At Day 1, prior to the start of treatment, results from screening visit evaluations are reviewed to determine eligibility requirements. Subsequent visits during treatment phase will take place at Day 8, Day 15 and Day 21 and subsequently at 3 week intervals. Timing of subsequent visits can be prolonged to max. 9 week intervals if no problems exist during the first 12 weeks of therapy. Subjects will self-administer study drug using an oral QD regimen in a continuous dosing schedule. Each dose of Pemigatinib should be taken first thing in the morning upon waking or after a 2-hour fast; subjects should then fast for an additional 1 hour after taking study drug. The starting dose is 13.5 mg Pemigatinib. Study treatment will continue until 12 months, or until the evidence of disease relapse or onset of unacceptable toxicity.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=2 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=2 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=2 Participants
|
|
Age, Continuous
|
73.5 years
n=2 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=2 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=2 Participants
|
|
Region of Enrollment
Italy
|
2 participants
n=2 Participants
|
PRIMARY outcome
Timeframe: Mean Relapse-free survival rate from start of treatment through premature study-end with a maximal follow-up of 62 weeks.Population: 2 patients started treatment with Pemigatinib and were followed-up according to protocol through premature study-end.
In the protocol, RFS at Year 2 was defined as the time from the start of treatment until disease relapse by Investigator determination, study-end or lost to follow-up, or death due to any cause. Due to the Covid-19 crisis causing significant delay of recruitment and the fact that we needed much more patients to identify patients with FGFR3 alterations, the study recruitment was prematurely stopped in November 2021 when 46 patients were screened and only 4 among 42 tested (9.5%) patients were identified with FGFR3 alterations. Two of the FGFR3 positive patients started treatment and the others were ineligible for the study. Since patients withdrawing from the study before completing the study period of 2 years cannot be included in the calculation of the 2-year RFS rate, the outcome results through premature study-end are presented.
Outcome measures
| Measure |
Mean RFS for Patients Treated With Pemigatinib.
n=2 Participants
Mean RFS for patients treated with Pemigatinib and followed through premature study-end with a maximal follow up of 62 weeks.
|
|---|---|
|
Mean Relapse-free Survival (RFS) After Start of Treatment With Pemigatinib.
|
26.6 weeks
Interval 6.9 to 46.4
|
SECONDARY outcome
Timeframe: Mean Overall Sturvival from start of treatment through premature study-end with a maximal follow-up of 62 weeks.Population: 2 patients started treatment with Pemigatinib and were followed-up according to protocol through premature study-end with a maximal follow-up of 62 weeks.
Mean Overall Survival of patients who started treatment with Pemigatinib is defined as the time from the start of treatment until the last date known alive, or death due to any cause through premature study-end.
Outcome measures
| Measure |
Mean RFS for Patients Treated With Pemigatinib.
n=2 Participants
Mean RFS for patients treated with Pemigatinib and followed through premature study-end with a maximal follow up of 62 weeks.
|
|---|---|
|
Mean Overall Survival After Start of Treatment With Pemigatinib.
|
34.2 weeks
Interval 6.9 to 61.6
|
Adverse Events
Treatment Arm
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment Arm
n=2 participants at risk
Treatment with Pemigatinib at the protocol-defined dose administered orally once daily as continuous therapy schedule until 12 months.
Pemigatinib: At Day 1, prior to the start of treatment, results from screening visit evaluations are reviewed to determine eligibility requirements. Subsequent visits during treatment phase will take place at Day 8, Day 15 and Day 21 and subsequently at 3 week intervals. Timing of subsequent visits can be prolonged to max. 9 week intervals if no problems exist during the first 12 weeks of therapy. Subjects will self-administer study drug using an oral QD regimen in a continuous dosing schedule. Each dose of Pemigatinib should be taken first thing in the morning upon waking or after a 2-hour fast; subjects should then fast for an additional 1 hour after taking study drug. The starting dose is 13.5 mg Pemigatinib. Study treatment will continue until 12 months, or until the evidence of disease relapse or onset of unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
high level of urea in blood
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Eye disorders
worsening visual acuity
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Eye disorders
photophobia
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
General disorders
dygeusia
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
General disorders
weight loss
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
General disorders
hypotension
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
General disorders
xerostomia
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Infections and infestations
mouth ulcers
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Musculoskeletal and connective tissue disorders
aching sole of foot
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Skin and subcutaneous tissue disorders
redness of basalioma
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Skin and subcutaneous tissue disorders
hairloss
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Skin and subcutaneous tissue disorders
xerosis cutis
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
Infections and infestations
mucositis mouth
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
|
General disorders
fatique
|
50.0%
1/2 • Number of events 1 • From start of treatment to premature study end with a maximal follow-up of 62 weeks.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place