Trial Outcomes & Findings for Evaluation of Safety & Tolerability of Multiple Dose Regimens of CHF 5074 (NCT NCT01303744)

NCT ID: NCT01303744

Last Updated: 2016-01-11

Results Overview

To assess if there were differences in ΔsCD40L levels between CHF 5074 doses and placebo

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

96 participants

Primary outcome timeframe

up to 12 weeks

Results posted on

2016-01-11

Participant Flow

Participant milestones

Participant milestones
Measure
CHF 5074 1x
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Overall Study
STARTED
24
24
24
24
Overall Study
COMPLETED
24
24
24
24
Overall Study
NOT COMPLETED
0
0
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Evaluation of Safety & Tolerability of Multiple Dose Regimens of CHF 5074

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
CHF 5074 1x
n=24 Participants
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
n=24 Participants
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
n=24 Participants
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
n=24 Participants
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Total
n=96 Participants
Total of all reporting groups
Age, Continuous
67.7 years
STANDARD_DEVIATION 8.1 • n=99 Participants
68.9 years
STANDARD_DEVIATION 8.5 • n=107 Participants
69.0 years
STANDARD_DEVIATION 9.2 • n=206 Participants
68.1 years
STANDARD_DEVIATION 8.0 • n=7 Participants
68.4 years
STANDARD_DEVIATION 8.3 • n=31 Participants
Sex: Female, Male
Female
10 Participants
n=99 Participants
11 Participants
n=107 Participants
11 Participants
n=206 Participants
8 Participants
n=7 Participants
40 Participants
n=31 Participants
Sex: Female, Male
Male
14 Participants
n=99 Participants
13 Participants
n=107 Participants
13 Participants
n=206 Participants
16 Participants
n=7 Participants
56 Participants
n=31 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
1 Participants
n=107 Participants
0 Participants
n=206 Participants
1 Participants
n=7 Participants
2 Participants
n=31 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=99 Participants
1 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
2 Participants
n=31 Participants
Race (NIH/OMB)
White
23 Participants
n=99 Participants
22 Participants
n=107 Participants
24 Participants
n=206 Participants
23 Participants
n=7 Participants
92 Participants
n=31 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
Region of Enrollment
United States
24 participants
n=99 Participants
24 participants
n=107 Participants
19 participants
n=206 Participants
24 participants
n=7 Participants
91 participants
n=31 Participants
Region of Enrollment
Italy
0 participants
n=99 Participants
0 participants
n=107 Participants
5 participants
n=206 Participants
0 participants
n=7 Participants
5 participants
n=31 Participants

PRIMARY outcome

Timeframe: up to 12 weeks

Population: ITT

To assess if there were differences in ΔsCD40L levels between CHF 5074 doses and placebo

Outcome measures

Outcome measures
Measure
CHF 5074 1x
n=24 Participants
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
n=24 Participants
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
n=24 Participants
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
n=24 Participants
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Differences in ∆sCD40L Levels Between CHF 5074 Doses and Placebo at Any Specific Time Point
270.28 pg/ml
Standard Error 371.09
403.22 pg/ml
Standard Error 377.79
-1293.42 pg/ml
Standard Error 362.69
242.49 pg/ml
Standard Error 361.32

SECONDARY outcome

Timeframe: Days 85

evaluate the pharmacokinetics (PK) of CHF 5074 in patients with MCI.

Outcome measures

Outcome measures
Measure
CHF 5074 1x
n=24 Participants
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
n=20 Participants
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
n=20 Participants
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Measurement of Trough CHF 5074 Plasma Levels
5497 ng/ml
Standard Deviation 4940
7882 ng/ml
Standard Deviation 8231
19999 ng/ml
Standard Deviation 17474

SECONDARY outcome

Timeframe: 29 days

Population: ITT

Outcome measures

Outcome measures
Measure
CHF 5074 1x
n=24 Participants
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
n=24 Participants
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
n=24 Participants
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
n=24 Participants
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Changes in Plasma ΔTNFα Concentrations
2.57 pg/ml
Standard Error 1.1
-0.339 pg/ml
Standard Error 1.09
-1.568 pg/ml
Standard Error 1.05
0.039 pg/ml
Standard Error 1.05

Adverse Events

CHF 5074 1x

Serious events: 2 serious events
Other events: 9 other events
Deaths: 0 deaths

CHF 5074 2x

Serious events: 2 serious events
Other events: 18 other events
Deaths: 0 deaths

CHF 5074 3x

Serious events: 1 serious events
Other events: 11 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
CHF 5074 1x
n=24 participants at risk
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
n=24 participants at risk
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
n=24 participants at risk
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
n=24 participants at risk
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Gastrointestinal disorders
intestinal obstruction
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
Cardiac disorders
Acute congestive heart failure
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
Cardiac disorders
cardiac failure congestive
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
Respiratory, thoracic and mediastinal disorders
bronchitis
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
Cardiac disorders
cerebrovascular accident
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
penis carcinoma
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
Musculoskeletal and connective tissue disorders
femur fracture
0.00%
0/24 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
adenocarcinoma f transverse colon
0.00%
0/24 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration

Other adverse events

Other adverse events
Measure
CHF 5074 1x
n=24 participants at risk
oral tablet, multidose CHF 5074 1x: oral tablet, 1x, once a day in the morning for 12 weeks
CHF 5074 2x
n=24 participants at risk
oral tablet, multidose CHF 5074 2x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 8 weeks
CHF 5074 3x
n=24 participants at risk
oral tablet, multidose CHF 5074 3x: oral tablet, 1x, once a day in the morning for 4 weeks, followed by oral tablet, 2x, once a day in the morning for 4 weeks, followed by oral tablet, 3x, once a day in the morning for 4 weeks
Placebo
n=24 participants at risk
placebo, oral tablet, multidose Placebo: oral tablet, once a day in the morning for 12 weeks
Gastrointestinal disorders
diarrhoea
8.3%
2/24 • Number of events 2 • Trial duration
12.5%
3/24 • Number of events 7 • Trial duration
20.8%
5/24 • Number of events 5 • Trial duration
8.3%
2/24 • Number of events 2 • Trial duration
Metabolism and nutrition disorders
back pain
4.2%
1/24 • Number of events 1 • Trial duration
12.5%
3/24 • Number of events 3 • Trial duration
8.3%
2/24 • Number of events 3 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
Nervous system disorders
Dizziness
4.2%
1/24 • Number of events 1 • Trial duration
8.3%
2/24 • Number of events 2 • Trial duration
12.5%
3/24 • Number of events 4 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
General disorders
headache
4.2%
1/24 • Number of events 1 • Trial duration
8.3%
2/24 • Number of events 2 • Trial duration
0.00%
0/24 • Trial duration
8.3%
2/24 • Number of events 4 • Trial duration
Gastrointestinal disorders
nausea
4.2%
1/24 • Number of events 1 • Trial duration
16.7%
4/24 • Number of events 4 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
Infections and infestations
Urinary tract infection
12.5%
3/24 • Number of events 5 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration
4.2%
1/24 • Number of events 2 • Trial duration
Gastrointestinal disorders
vomiting
0.00%
0/24 • Trial duration
8.3%
2/24 • Number of events 2 • Trial duration
4.2%
1/24 • Number of events 1 • Trial duration
0.00%
0/24 • Trial duration
Respiratory, thoracic and mediastinal disorders
nasopharyngitis
0.00%
0/24 • Trial duration
8.3%
2/24 • Number of events 2 • Trial duration
0.00%
0/24 • Trial duration
0.00%
0/24 • Trial duration

Additional Information

Richard Margolin

Cerespir

Phone: (646) 202-2562

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place