Trial Outcomes & Findings for A Study to Compare CAELYX With Topotecan HCL in Patients With Recurrent Epithelial Ovarian Carcinoma Following Failure of First-Line, Platinum-Based Chemotherapy (NCT NCT01840943)

Last Updated: 2015-12-23

Results Overview

Progression-free survival incidence was be measured as number of participants who were progression-free and alive. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20 percent (%) increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

32 participants

Primary outcome timeframe

Week 24

Results posted on

2015-12-23

Participant Flow

Out of 32 participants screened, 26 were randomized to study treatment.

Participant milestones

Participant milestones
Measure	CAELYX Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Overall Study STARTED	14	12
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	13	12

Reasons for withdrawal

Reasons for withdrawal
Measure	CAELYX Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Overall Study Withdrawal by Subject	5	6
Overall Study Physician Decision	4	2
Overall Study Lost to Follow-up	4	4

Baseline Characteristics

A Study to Compare CAELYX With Topotecan HCL in Patients With Recurrent Epithelial Ovarian Carcinoma Following Failure of First-Line, Platinum-Based Chemotherapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CAELYX n=14 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.	Total n=26 Participants Total of all reporting groups
Age, Continuous	56.1 years STANDARD_DEVIATION 4.97 • n=5 Participants	51.6 years STANDARD_DEVIATION 8.32 • n=7 Participants	54.0 years STANDARD_DEVIATION 6.96 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	12 Participants n=7 Participants	26 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 24

Population: The modified intent-to-treat population included (mITT) included all randomized participants.

Outcome measures

Outcome measures
Measure	CAELYX n=14 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Number of Participants With Progression-free Survival Incidence at Week 24	6 participants	2 participants

SECONDARY outcome

Timeframe: 1 year after the last dose (24 weeks) administration

Population: The mITT population included all randomized participants.

It was calculated as the time, in weeks, from the day of randomization until documented disease progression or death due to any cause, whichever occurs first using a Kaplan-Meier curve for PFS.

Outcome measures

Outcome measures
Measure	CAELYX n=14 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Duration of Progression-free Survival	NA weeks Interval 8.0 to Insufficient number of participants with events.	24.6 weeks Interval 7.9 to 25.1

SECONDARY outcome

Timeframe: Up to Week 24

Population: The mITT population included all randomized participants.

Response rate was measured as number of participants with at least a durable response: Complete response (CR) or partial response (PR). Complete response is defined as the disappearance of all target lesions. Partial response is defined as at least a 30 percentage decrease in the sum of diameters of target lesions. Stable disease defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease. Progression in disease is defined as at least a 20% increase in the sum of diameters of target lesions. Not evaluable participants were those who were not analyzed. The reference of the baseline sum of diameters of lesions was considered.

Outcome measures

Outcome measures
Measure	CAELYX n=14 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Number of Participants With Response Not Evaluable (NE)	4 participants	2 participants
Number of Participants With Response Complete Response (CR)	0 participants	0 participants
Number of Participants With Response Partial Response (PR)	5 participants	3 participants
Number of Participants With Response Stable Disease (SD)	4 participants	6 participants
Number of Participants With Response Progression Disease (PD)	1 participants	1 participants

SECONDARY outcome

Timeframe: Up to Week 24

Population: The mITT population included all randomized participants.Time to response was analyzed for participants who achieved response.

It is calculated as the day of randomization to the first observation of a durable response (the first of the 2 confirmatory measurements).

Outcome measures

Outcome measures
Measure	CAELYX n=5 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=3 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Time to Response	16.0 weeks Interval 8.0 to 24.1	16.0 weeks Interval 6.0 to 16.1

SECONDARY outcome

Timeframe: Up to 1 year of last dose (Week 24) administration

Population: The mITT population included all randomized participants. Duration of response was analyzed for participants who achieved response.

It is calculated as the first observation of a durable response (the first of the 2 confirmatory measurements) to the first observation of disease progression or death due to any cause.

Outcome measures

Outcome measures
Measure	CAELYX n=5 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=3 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Duration of Response	NA weeks Interval 8.1 to Insufficient number of participants with events.	9.3 weeks Interval 8.6 to 10.6

SECONDARY outcome

Timeframe: Day 1 of each cycle of study medication and Week 4 after last dose of study medication

Population: Data was not collected for this outcome measure as the study was early terminated.

Calculation of each HQL domain scale will be performed according to the scoring guidelines for each of the HQL measures. The HQL analyses will include scales measuring physical functioning, pain, nausea, fatigue, and global quality of life. Each item is measured on a scale of 0 to 3, where 0 = no impact on quality of life and 3 = extreme impact on quality of life.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 4 after the last dose of the study medication and approximately up to 1 year after the disease progression or completion of the study treatment or death, whichever is earlier

Population: The mITT population included all randomized participants.

Number of Participants With Overall survival were categorized as number of 1) Deaths, 2) Still alive, 3) Early termination from the study due to lost to follow up, 4) Early termination from the study due to withdraw of consent, 5) Other. Overall survival is defined as the time interval from randomization to death from any cause.

Outcome measures

Outcome measures
Measure	CAELYX n=14 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Number of Participants With Overall Survival Death	1 participants	0 participants
Number of Participants With Overall Survival Still alive	0 participants	0 participants
Number of Participants With Overall Survival Early termination: due to lost to follow up	4 participants	4 participants
Number of Participants With Overall Survival Early termination: due to withdraw of consent	5 participants	6 participants
Number of Participants With Overall Survival Other	4 participants	2 participants

SECONDARY outcome

Timeframe: Up to 30 days after the last dose of study medication

Population: Safety population included all randomized participants.

An AE is any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Grade 3 (Severe) events are symptoms causing inability to perform usual social \& functional activities. Grade 4 (Life-threatening) events are Symptoms causing inability to perform basic self-care functions or Medical or operative intervention indicated to prevent permanent impairment, persistent disability, or death.

Outcome measures

Outcome measures
Measure	CAELYX n=14 Participants Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 Participants Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Number of Participants With Adverse Events TEAEs	12 participants	12 participants
Number of Participants With Adverse Events TESAEs	1 participants	1 participants

Adverse Events

CAELYX

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Topotecan Hydrocloride (HCl)

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CAELYX n=14 participants at risk Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 participants at risk Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Infections and infestations Pulmonary tuberculosis	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Platelet count decreased	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.

Other adverse events

Additional Information

Senior Director Clin Development

Xian Janssen Pharmaceutical LTD

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If an investigator wishes to publish information from the study, a copy of the manuscript must be provided to the sponsor for review at least 60 days before submission for publication or presentation. If requested by the sponsor in writing, the investigator will withhold such publication for up to an additional 60 days.
Publication restrictions are in place

Restriction type: OTHER

Measure	CAELYX n=14 participants at risk Participants received 50 milligram per square meters (mg/m\^2) of Caelyx as a 90-minute or 60 to 90-minute intravenous infusion every 4 weeks.	Topotecan Hydrocloride (HCl) n=12 participants at risk Participants received topotecan HCl 1.25 mg per square meter per day, intravenously for 30-minutes duration, on Day 1 to Day 5 of each cycle.
Gastrointestinal disorders Abdominal distension	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Abdominal pain	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	16.7% 2/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Diarrhoea	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Gastrointestinal pain	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Dermatitis	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Dermatitis acneiform	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Purpura	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Ileus	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysaesthesia syndrome	35.7% 5/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Rash	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Rash maculo-papular	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Infections and infestations Pulmonary tuberculosis	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Infections and infestations Vaginal infection	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
General disorders Pyrexia	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
General disorders Fatigue	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
General disorders Irritability	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Infections and infestations Upper respiratory tract infection	14.3% 2/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Infections and infestations Folliculitis	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Infections and infestations Herpes zoster	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Respiratory, thoracic and mediastinal disorders Cough	14.3% 2/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Immune system disorders Anaphylactic reaction	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Immune system disorders Hypersensitivity	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Immune system disorders Infusion related reaction	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Psychiatric disorders Insomnia	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Metabolism and nutrition disorders Decreased appetite	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Metabolism and nutrition disorders Hyperglycaemia	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Metabolism and nutrition disorders Hypoalbuminaemia	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Stomatitis	21.4% 3/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Constipation	14.3% 2/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Mouth ulceration	21.4% 3/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Blood and lymphatic system disorders Anemia	35.7% 5/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	91.7% 11/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Blood and lymphatic system disorders Leukopenia	35.7% 5/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	16.7% 2/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Blood and lymphatic system disorders Neutropenia	35.7% 5/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Blood and lymphatic system disorders Thrombocytopenia	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	16.7% 2/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Leukocytosis	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Blood and lymphatic system disorders Neutrophilia	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations White blood cell count decreased	42.9% 6/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	83.3% 10/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Neutrophil count decreased	42.9% 6/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	75.0% 9/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Platelet count decreased	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	50.0% 6/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Alanine aminotransferase increased	14.3% 2/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Aspartate aminotransferase increased	21.4% 3/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Weight decreased	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Blood albumin decreased	7.1% 1/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	0.00% 0/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Lymphocyte percentage decreased	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations Platelet count increased	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Investigations White blood cell count increased	0.00% 0/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	8.3% 1/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Vomiting	35.7% 5/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	66.7% 8/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.
Gastrointestinal disorders Nausea	42.9% 6/14 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.	41.7% 5/12 • Up to 30 days after the last dose of study medication Safety population included all randomized participants.