Trial Outcomes & Findings for Study to Evaluate the Efficacy, Safety and Tolerability of Everolimus in de Novo Renal Transplant Recipients Participating in the Eurotransplant Senior Program (NCT NCT00956293)

NCT ID: NCT00956293

Last Updated: 2014-06-06

Results Overview

The study was terminated prematurely and not powered for efficacy.

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

207 participants

Primary outcome timeframe

Month 6

Results posted on

2014-06-06

Participant Flow

The study consisted of a main period and a 54 month observation follow-up period. The main period included a pre-randomized treatment phase (6 weeks) and a randomized treatment phase (18 weeks). All randomized participants, who participated in the main period, were eligible for the follow-up period.

At baseline (BL) 1 (pre-randomization), eligible participants received a CNI-based regimen for 6 weeks. At BL2 (randomization), eligible participants were randomized in a 1:2 ratio to the control group or everolimus group.

Participant milestones

Participant milestones
Measure
Control Group
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group continued with a CNI-based regimen of MPA and CsA.
Everolimus Group
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group made a stepwise switch to a CNI-free regimen of everolimus and MPA.
Pre-randomized Group
Participants, who met BL1 eligibility, were enrolled into the study.
Main Period (Pre-randomization)
STARTED
0
0
207
Main Period (Pre-randomization)
Enrolled Safety Set
0
0
203
Main Period (Pre-randomization)
COMPLETED
0
0
77
Main Period (Pre-randomization)
NOT COMPLETED
0
0
130
Main Period (Randomization)
STARTED
24
53
0
Main Period (Randomization)
Full Analysis Set
24
51
0
Main Period (Randomization)
Randomized Safety Set
24
51
0
Main Period (Randomization)
COMPLETED
23
26
0
Main Period (Randomization)
NOT COMPLETED
1
27
0
Follow-up Period
STARTED
20
32
0
Follow-up Period
COMPLETED
0
0
0
Follow-up Period
NOT COMPLETED
20
32
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Control Group
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group continued with a CNI-based regimen of MPA and CsA.
Everolimus Group
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group made a stepwise switch to a CNI-free regimen of everolimus and MPA.
Pre-randomized Group
Participants, who met BL1 eligibility, were enrolled into the study.
Main Period (Pre-randomization)
Not specified (data missing)
0
0
1
Main Period (Pre-randomization)
Death
0
0
1
Main Period (Pre-randomization)
Protocol Violation
0
0
1
Main Period (Pre-randomization)
Abnormal test procedure result
0
0
2
Main Period (Pre-randomization)
Administrative problems
0
0
6
Main Period (Pre-randomization)
Graft loss
0
0
7
Main Period (Pre-randomization)
Withdrawal by Subject
0
0
12
Main Period (Pre-randomization)
Lack of Efficacy
0
0
20
Main Period (Pre-randomization)
Adverse Event
0
0
36
Main Period (Pre-randomization)
Abnormal laboratory value
0
0
44
Main Period (Randomization)
Abnormal laboratory value
0
2
0
Main Period (Randomization)
Lack of Efficacy
1
10
0
Main Period (Randomization)
Adverse Event
0
15
0
Follow-up Period
Follow-up period was terminated.
20
32
0

Baseline Characteristics

Study to Evaluate the Efficacy, Safety and Tolerability of Everolimus in de Novo Renal Transplant Recipients Participating in the Eurotransplant Senior Program

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Control Group
n=24 Participants
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group continued with a CNI-based regimen of MPA and CsA.
Everolimus Group
n=51 Participants
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group made a stepwise switch to a CNI-free regimen of everolimus and MPA.
Total
n=75 Participants
Total of all reporting groups
Age, Continuous
69.3 Years
STANDARD_DEVIATION 3.1 • n=99 Participants
68.4 Years
STANDARD_DEVIATION 3.3 • n=107 Participants
68.7 Years
STANDARD_DEVIATION 3.3 • n=206 Participants
Sex: Female, Male
Female
8 Participants
n=99 Participants
26 Participants
n=107 Participants
34 Participants
n=206 Participants
Sex: Female, Male
Male
16 Participants
n=99 Participants
25 Participants
n=107 Participants
41 Participants
n=206 Participants

PRIMARY outcome

Timeframe: Month 6

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

The study was terminated prematurely and not powered for efficacy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 6

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

The study was terminated prematurely and not powered for efficacy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Months 6, 12, 24, 36, 48 and 60

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

The study was terminated prematurely and not powered for efficacy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Months 6, 12, 24, 36, 48 and 60

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

The study was terminated prematurely and not powered for efficacy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 6

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

The study was terminated prematurely and not powered for efficacy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 7, Month 6

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

The study was terminated prematurely and not powered for efficacy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Month 6

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Months 6, 12, 24, 36, 48 and 60

Population: Randomized Safety Set: This set included all randomized participants who received at least one dose of study medication.

Participants with adverse events (serious plus non-serious), serious adverse events and death were reported.

Outcome measures

Outcome measures
Measure
Control Group
n=24 Participants
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group continued with a CNI-based regimen of MPA and CsA.
Everolimus Group
n=51 Participants
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group made a stepwise switch to a CNI-free regimen of everolimus and MPA.
Number of Participants Who Experienced Adverse Events, Serious Adverse Events and Death
Adverse events (serious and non-serious)
21 Participants
50 Participants
Number of Participants Who Experienced Adverse Events, Serious Adverse Events and Death
Serious adverse events
11 Participants
28 Participants
Number of Participants Who Experienced Adverse Events, Serious Adverse Events and Death
Deaths
0 Participants
0 Participants

SECONDARY outcome

Timeframe: Months 12, 24, 36, 48 and 60

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Months12, 24, 36, 48 and 60

Population: This outcome measure was not analyzed because a total of 244 completed subjects were needed to have a power of 80% in detecting a significant difference between treatment groups. Due to early termination, the study was limited by a small sample size; hence, the planned analysis was not done.

Outcome measures

Outcome data not reported

Adverse Events

Control Goup

Serious events: 11 serious events
Other events: 15 other events
Deaths: 0 deaths

Everolimus Group

Serious events: 28 serious events
Other events: 50 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Control Goup
n=24 participants at risk
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group continued with a CNI-based regimen of MPA and CsA.
Everolimus Group
n=51 participants at risk
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group made a stepwise switch to a CNI-free regimen of everolimus and MPA.
Blood and lymphatic system disorders
ANAEMIA
4.2%
1/24
3.9%
2/51
Blood and lymphatic system disorders
LEUKOPENIA
0.00%
0/24
5.9%
3/51
Blood and lymphatic system disorders
PANCYTOPENIA
0.00%
0/24
3.9%
2/51
Cardiac disorders
MITRAL VALVE INCOMPETENCE
4.2%
1/24
0.00%
0/51
Gastrointestinal disorders
ABDOMINAL MASS
0.00%
0/24
2.0%
1/51
Gastrointestinal disorders
APHTHOUS STOMATITIS
0.00%
0/24
2.0%
1/51
Gastrointestinal disorders
CROHN'S DISEASE
0.00%
0/24
2.0%
1/51
Gastrointestinal disorders
DIARRHOEA
0.00%
0/24
3.9%
2/51
Gastrointestinal disorders
ENTEROCOLITIS HAEMORRHAGIC
0.00%
0/24
2.0%
1/51
Gastrointestinal disorders
ILEUS PARALYTIC
0.00%
0/24
2.0%
1/51
Gastrointestinal disorders
INGUINAL HERNIA
0.00%
0/24
2.0%
1/51
General disorders
GENERAL PHYSICAL HEALTH DETERIORATION
0.00%
0/24
2.0%
1/51
General disorders
PYREXIA
0.00%
0/24
2.0%
1/51
Immune system disorders
TRANSPLANT REJECTION
4.2%
1/24
0.00%
0/51
Infections and infestations
BK VIRUS INFECTION
0.00%
0/24
2.0%
1/51
Infections and infestations
BRONCHOPULMONARY ASPERGILLOSIS
0.00%
0/24
2.0%
1/51
Infections and infestations
CYTOMEGALOVIRUS COLITIS
0.00%
0/24
2.0%
1/51
Infections and infestations
FEBRILE INFECTION
0.00%
0/24
2.0%
1/51
Infections and infestations
GASTROENTERITIS VIRAL
0.00%
0/24
2.0%
1/51
Infections and infestations
GENITAL HERPES
0.00%
0/24
2.0%
1/51
Infections and infestations
HEPATIC CYST INFECTION
4.2%
1/24
0.00%
0/51
Infections and infestations
HERPES ZOSTER OTICUS
0.00%
0/24
2.0%
1/51
Infections and infestations
INFECTION
8.3%
2/24
2.0%
1/51
Infections and infestations
PNEUMONIA
8.3%
2/24
7.8%
4/51
Infections and infestations
PYELONEPHRITIS ACUTE
0.00%
0/24
2.0%
1/51
Infections and infestations
URINARY TRACT INFECTION
12.5%
3/24
9.8%
5/51
Infections and infestations
UROSEPSIS
0.00%
0/24
2.0%
1/51
Injury, poisoning and procedural complications
GRAFT DYSFUNCTION
0.00%
0/24
2.0%
1/51
Injury, poisoning and procedural complications
INCISIONAL HERNIA
0.00%
0/24
2.0%
1/51
Injury, poisoning and procedural complications
POST PROCEDURAL HAEMORRHAGE
0.00%
0/24
2.0%
1/51
Injury, poisoning and procedural complications
WOUND DEHISCENCE
0.00%
0/24
2.0%
1/51
Investigations
BLOOD CREATININE INCREASED
16.7%
4/24
9.8%
5/51
Metabolism and nutrition disorders
HYPOGLYCAEMIA
0.00%
0/24
2.0%
1/51
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
BASAL CELL CARCINOMA
0.00%
0/24
2.0%
1/51
Nervous system disorders
FACIAL NERVE DISORDER
0.00%
0/24
2.0%
1/51
Renal and urinary disorders
ACUTE PRERENAL FAILURE
0.00%
0/24
2.0%
1/51
Renal and urinary disorders
ANURIA
4.2%
1/24
0.00%
0/51
Renal and urinary disorders
NEPHROSCLEROSIS
4.2%
1/24
0.00%
0/51
Renal and urinary disorders
RENAL ARTERY STENOSIS
0.00%
0/24
2.0%
1/51
Renal and urinary disorders
RENAL FAILURE ACUTE
4.2%
1/24
2.0%
1/51
Renal and urinary disorders
URETERIC STENOSIS
0.00%
0/24
3.9%
2/51
Renal and urinary disorders
URINARY TRACT DISORDER
0.00%
0/24
2.0%
1/51
Respiratory, thoracic and mediastinal disorders
DYSPNOEA EXERTIONAL
0.00%
0/24
2.0%
1/51
Respiratory, thoracic and mediastinal disorders
PULMONARY ARTERIAL HYPERTENSION
4.2%
1/24
0.00%
0/51
Vascular disorders
ARTERIOVENOUS FISTULA
4.2%
1/24
2.0%
1/51
Vascular disorders
LYMPHOCELE
0.00%
0/24
2.0%
1/51

Other adverse events

Other adverse events
Measure
Control Goup
n=24 participants at risk
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group continued with a CNI-based regimen of MPA and CsA.
Everolimus Group
n=51 participants at risk
During the pre-randomized treatment phase, all participants received a CNI-based regimen consisting of basiliximab, mycophenolic acid (MPA), cyclosporin A (CsA) and corticosteroids (optional). Upon randomization, participants in this group made a stepwise switch to a CNI-free regimen of everolimus and MPA.
Blood and lymphatic system disorders
ANAEMIA
8.3%
2/24
11.8%
6/51
Blood and lymphatic system disorders
LEUKOPENIA
16.7%
4/24
39.2%
20/51
Gastrointestinal disorders
APHTHOUS STOMATITIS
0.00%
0/24
17.6%
9/51
Gastrointestinal disorders
CONSTIPATION
0.00%
0/24
5.9%
3/51
Gastrointestinal disorders
DIARRHOEA
0.00%
0/24
19.6%
10/51
Gastrointestinal disorders
FLATULENCE
0.00%
0/24
7.8%
4/51
Gastrointestinal disorders
NAUSEA
4.2%
1/24
5.9%
3/51
General disorders
OEDEMA PERIPHERAL
29.2%
7/24
25.5%
13/51
General disorders
PYREXIA
16.7%
4/24
9.8%
5/51
Infections and infestations
CYTOMEGALOVIRUS INFECTION
8.3%
2/24
7.8%
4/51
Infections and infestations
NASOPHARYNGITIS
4.2%
1/24
9.8%
5/51
Infections and infestations
URINARY TRACT INFECTION
25.0%
6/24
43.1%
22/51
Investigations
BLOOD CREATININE INCREASED
4.2%
1/24
9.8%
5/51
Metabolism and nutrition disorders
HYPERLIPIDAEMIA
4.2%
1/24
5.9%
3/51
Metabolism and nutrition disorders
HYPERURICAEMIA
4.2%
1/24
7.8%
4/51
Metabolism and nutrition disorders
HYPOCALCAEMIA
0.00%
0/24
5.9%
3/51
Metabolism and nutrition disorders
HYPOKALAEMIA
0.00%
0/24
23.5%
12/51
Metabolism and nutrition disorders
HYPOMAGNESAEMIA
4.2%
1/24
5.9%
3/51
Metabolism and nutrition disorders
HYPOPHOSPHATAEMIA
0.00%
0/24
7.8%
4/51
Metabolism and nutrition disorders
IRON DEFICIENCY
0.00%
0/24
9.8%
5/51
Metabolism and nutrition disorders
VITAMIN D DEFICIENCY
0.00%
0/24
5.9%
3/51
Psychiatric disorders
INSOMNIA
4.2%
1/24
5.9%
3/51
Renal and urinary disorders
PROTEINURIA
0.00%
0/24
15.7%
8/51
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
12.5%
3/24
5.9%
3/51
Respiratory, thoracic and mediastinal disorders
PLEURAL EFFUSION
0.00%
0/24
5.9%
3/51
Skin and subcutaneous tissue disorders
PRURITUS
8.3%
2/24
2.0%
1/51
Vascular disorders
HYPERTENSION
12.5%
3/24
11.8%
6/51

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: +1 (862) 778-1873

Results disclosure agreements

  • Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
  • Publication restrictions are in place

Restriction type: OTHER