Trial Outcomes & Findings for Study of the Effects of Cerefolin NAC on Inflammation Blood Markers in Older Individuals With Memory Complaints (NCT NCT00597376)

NCT ID: NCT00597376

Last Updated: 2013-05-23

Results Overview

Primary outcome markers were plasma homocysteine (tHcy), glutathione, and the ratio of amyloid proteins, Aβ42 and Aβ40. Plasma tHcy and glutathione were assayed using a high performance liquid chromatography (HPLC) with fluorescence detection method. Enzyme-linked immunosorbent assays (ELISA) were used for Aβ42 (Wako Chemicals USA, Inc., Richmond, VA) and Aβ40 (Invitrogen Corporation, Canarillo, CA) detection. Primary analyses utilized Last Observation Carried Forward (LOCF) to assess the primary biomarker level at 6 months versus baseline. Six month levels in biomarker outcomes were compared using t-tests of the logarithmically transformed values and the antilogarithm was applied to the SDs obtain 95% confidence intervals (95% CIs). A significant difference between treatments was needed for at least one of the primary outcome variables (tHcy, glutathione, or the Aβ42 to Aβ40 ratio) to declare the study positive.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

104 participants

Primary outcome timeframe

6 months

Results posted on

2013-05-23

Participant Flow

Between March 2008 and September 2009, persons expressing study interest at community presentations or at the Rush Memory Clinic were contacted. Of 524 persons expressing interest, 400 were excluded before consenting for having excluding clinical diagnoses(227), participation refusal (119), and other exclusions medication (54).

Consented participants underwent a screening evaluation to ensure inclusion criteria were met prior to study treatment assignment and one month run-in treatment with a multivitamin only. Of 124 consented individuals, 20 discontinued prior to randomization due to new medical conditions (9), declining participation (5), and for other reasons (6).

Participant milestones

Participant milestones
Measure
Cerefolin NAC + Multivitamin
On Cerefolin NAC and open-label multivitamin supplement Cerefolin NAC (a medical food) : Cerefolin NAC one tablet each day
Cerefolin NAC Placebo + Multivitamin
On placebo and open label multivitamin supplement Cerefolin NAC placebo : Placebo tablet once a day
Overall Study
STARTED
51
53
Overall Study
COMPLETED
43
49
Overall Study
NOT COMPLETED
8
4

Reasons for withdrawal

Reasons for withdrawal
Measure
Cerefolin NAC + Multivitamin
On Cerefolin NAC and open-label multivitamin supplement Cerefolin NAC (a medical food) : Cerefolin NAC one tablet each day
Cerefolin NAC Placebo + Multivitamin
On placebo and open label multivitamin supplement Cerefolin NAC placebo : Placebo tablet once a day
Overall Study
Withdrawal by Subject
4
3
Overall Study
Adverse Event
4
1

Baseline Characteristics

Study of the Effects of Cerefolin NAC on Inflammation Blood Markers in Older Individuals With Memory Complaints

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Cerefolin NAC + Multivitamin
n=51 Participants
On Cerefolin NAC and open-label multivitamin supplement Cerefolin NAC (a medical food) : Cerefolin NAC one tablet each day
Cerefolin NAC Placebo + Multivitamin
n=53 Participants
On placebo and open label multivitamin supplement Cerefolin NAC placebo : Placebo tablet once a day
Total
n=104 Participants
Total of all reporting groups
Age Continuous
80.9 years
STANDARD_DEVIATION 6.6 • n=5 Participants
79.4 years
STANDARD_DEVIATION 8.7 • n=7 Participants
80.1 years
STANDARD_DEVIATION 7.7 • n=5 Participants
Sex: Female, Male
Female
46 Participants
n=5 Participants
42 Participants
n=7 Participants
88 Participants
n=5 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
11 Participants
n=7 Participants
16 Participants
n=5 Participants
Region of Enrollment
United States
51 participants
n=5 Participants
53 participants
n=7 Participants
104 participants
n=5 Participants
Education Level
15 years
STANDARD_DEVIATION 3 • n=5 Participants
15 years
STANDARD_DEVIATION 3 • n=7 Participants
15 years
STANDARD_DEVIATION 3 • n=5 Participants
total plasma homocysteine
8.35 micromol/L
STANDARD_DEVIATION 1.32 • n=5 Participants
8.10 micromol/L
STANDARD_DEVIATION 1.33 • n=7 Participants
8.22 micromol/L
STANDARD_DEVIATION 1.33 • n=5 Participants
glutathione
4.01 micromol/L
STANDARD_DEVIATION 1.67 • n=5 Participants
4.02 micromol/L
STANDARD_DEVIATION 1.59 • n=7 Participants
4.02 micromol/L
STANDARD_DEVIATION 1.63 • n=5 Participants
ratio of amyloid beta 1-42 to 1-40
0.027 pmol/L:pmol/L
STANDARD_DEVIATION 1.73 • n=5 Participants
0.034 pmol/L:pmol/L
STANDARD_DEVIATION 1.81 • n=7 Participants
0.031 pmol/L:pmol/L
STANDARD_DEVIATION 1.77 • n=5 Participants
highly sensitive c-Reactive Protein
0.20 mg/mL
STANDARD_DEVIATION 18.93 • n=5 Participants
0.23 mg/mL
STANDARD_DEVIATION 22.37 • n=7 Participants
0.22 mg/mL
STANDARD_DEVIATION 20.68 • n=5 Participants
tumor necrosis factor alpha
1.45 pg/mL
STANDARD_DEVIATION 1.61 • n=5 Participants
1.37 pg/mL
STANDARD_DEVIATION 1.53 • n=7 Participants
1.41 pg/mL
STANDARD_DEVIATION 1.57 • n=5 Participants
interleukin-6
2.12 pg/mL
STANDARD_DEVIATION 1.98 • n=5 Participants
2.12 pg/mL
STANDARD_DEVIATION 2.35 • n=7 Participants
2.12 pg/mL
STANDARD_DEVIATION 2.17 • n=5 Participants
malondialdehyde
0.90 micromol/L
STANDARD_DEVIATION 1.67 • n=5 Participants
0.93 micromol/L
STANDARD_DEVIATION 1.75 • n=7 Participants
0.92 micromol/L
STANDARD_DEVIATION 1.71 • n=5 Participants
potential anti-oxidant (PAO)
979 micromol/L
STANDARD_DEVIATION 2 • n=5 Participants
1016 micromol/L
STANDARD_DEVIATION 2 • n=7 Participants
997 micromol/L
STANDARD_DEVIATION 2 • n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: The Intent-to-Treat (ITT) cohort included randomized subjects taking at least one study treatment dose. Primary analyses utilized Last Observation Carried Forward (LOCF) to assess 6 month levels in the primary biomarkers. 2-sided, t-test p-value for group differences was only significant (\<0.05) for 6-month homocysteine level.

Primary outcome markers were plasma homocysteine (tHcy), glutathione, and the ratio of amyloid proteins, Aβ42 and Aβ40. Plasma tHcy and glutathione were assayed using a high performance liquid chromatography (HPLC) with fluorescence detection method. Enzyme-linked immunosorbent assays (ELISA) were used for Aβ42 (Wako Chemicals USA, Inc., Richmond, VA) and Aβ40 (Invitrogen Corporation, Canarillo, CA) detection. Primary analyses utilized Last Observation Carried Forward (LOCF) to assess the primary biomarker level at 6 months versus baseline. Six month levels in biomarker outcomes were compared using t-tests of the logarithmically transformed values and the antilogarithm was applied to the SDs obtain 95% confidence intervals (95% CIs). A significant difference between treatments was needed for at least one of the primary outcome variables (tHcy, glutathione, or the Aβ42 to Aβ40 ratio) to declare the study positive.

Outcome measures

Outcome measures
Measure
Cerefolin NAC + Multivitamin
n=51 Participants
On Cerefolin NAC and open-label multivitamin supplement
Cerefolin NAC Placebo + Multivitamin
n=53 Participants
On placebo and open-label multivitamin supplement
Six Month Blood Levels of Homocysteine, Glutathione, and the Ratio of Aβ42 to Aβ40 (as a Percent of Baseline Levels) After Daily Intake of Cerefolin NAC Plus a Multivitamin Versus a Multivitamin Only
6-Month Homocysteine Level
71 percent of baseline level
Interval 66.0 to 77.0
101 percent of baseline level
Interval 96.0 to 107.0
Six Month Blood Levels of Homocysteine, Glutathione, and the Ratio of Aβ42 to Aβ40 (as a Percent of Baseline Levels) After Daily Intake of Cerefolin NAC Plus a Multivitamin Versus a Multivitamin Only
6-Month Glutathione Level
115 percent of baseline level
Interval 101.0 to 129.0
101 percent of baseline level
Interval 85.0 to 117.0
Six Month Blood Levels of Homocysteine, Glutathione, and the Ratio of Aβ42 to Aβ40 (as a Percent of Baseline Levels) After Daily Intake of Cerefolin NAC Plus a Multivitamin Versus a Multivitamin Only
6-Month AB42/AB40 Ratio
101 percent of baseline level
Interval 92.0 to 110.0
104 percent of baseline level
Interval 97.0 to 108.0

SECONDARY outcome

Timeframe: 6 months

Population: All participants in Intent-to-Treat cohort were included.

Mean study product compliance was measured as the actual number of study product tablets taken as a percent of the maximum study product tablets that could have been taken during the intervention period.

Outcome measures

Outcome measures
Measure
Cerefolin NAC + Multivitamin
n=51 Participants
On Cerefolin NAC and open-label multivitamin supplement
Cerefolin NAC Placebo + Multivitamin
n=53 Participants
On placebo and open-label multivitamin supplement
Tolerability of Cerefolin NAC and a Multivitamin Versus a Multivitamin Only
88 percent of study drug taken
Standard Error 15
88 percent of study drug taken
Standard Error 21

SECONDARY outcome

Timeframe: 6 months

Population: All participants in Intent-to-Treat were included with last observation carried forward.

Outcome measures were 6-month levels of highly sensitive c-reactive protein (hs-CRP), tumor necrosis factor alpha (TNF-alpha), interleukin-6 (IL-6), malondialdehyde, and potential anti-oxidant (PAO)in blood samples as a percent of baseline value.

Outcome measures

Outcome measures
Measure
Cerefolin NAC + Multivitamin
n=51 Participants
On Cerefolin NAC and open-label multivitamin supplement
Cerefolin NAC Placebo + Multivitamin
n=53 Participants
On placebo and open-label multivitamin supplement
Six Month Levels of Inflammation and Oxidative Stress Markers(as a Percent of Baseline Levels) After Daily Treatment With Cerefolin NAC and a Multivitamin or a Multivitamin Only
6-month TNF-alpha Level
99 percent of baseline level
Interval 93.0 to 106.0
108 percent of baseline level
Interval 98.0 to 118.0
Six Month Levels of Inflammation and Oxidative Stress Markers(as a Percent of Baseline Levels) After Daily Treatment With Cerefolin NAC and a Multivitamin or a Multivitamin Only
6-month hs-CRP Level
110 percent of baseline level
Interval 51.0 to 238.0
111 percent of baseline level
Interval 53.0 to 234.0
Six Month Levels of Inflammation and Oxidative Stress Markers(as a Percent of Baseline Levels) After Daily Treatment With Cerefolin NAC and a Multivitamin or a Multivitamin Only
6-month IL-6 Level
114 percent of baseline level
Interval 97.0 to 134.0
105 percent of baseline level
Interval 88.0 to 125.0
Six Month Levels of Inflammation and Oxidative Stress Markers(as a Percent of Baseline Levels) After Daily Treatment With Cerefolin NAC and a Multivitamin or a Multivitamin Only
6-month Malondialdehyde Level
93 percent of baseline level
Interval 83.0 to 104.0
90 percent of baseline level
Interval 79.0 to 102.0
Six Month Levels of Inflammation and Oxidative Stress Markers(as a Percent of Baseline Levels) After Daily Treatment With Cerefolin NAC and a Multivitamin or a Multivitamin Only
6-month PAO Level
109 percent of baseline level
Interval 96.0 to 123.0
115 percent of baseline level
Interval 100.0 to 131.0

Adverse Events

Cerefolin NAC + Multivitamin

Serious events: 11 serious events
Other events: 30 other events
Deaths: 0 deaths

Cerefolin NAC Placebo + Multivitamin

Serious events: 8 serious events
Other events: 28 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Cerefolin NAC + Multivitamin
n=51 participants at risk
On Cerefolin NAC and open-label multivitamin supplement
Cerefolin NAC Placebo + Multivitamin
n=53 participants at risk
On placebo and open-label multivitamin supplement
Cardiac disorders
Cardiac disorders
5.9%
3/51 • Number of events 3
5.7%
3/53 • Number of events 3
Gastrointestinal disorders
Gastrointestinal disorders
3.9%
2/51 • Number of events 2
0.00%
0/53
General disorders
General Disorders
3.9%
2/51 • Number of events 2
3.8%
2/53 • Number of events 2
Metabolism and nutrition disorders
Metabolism and nutrition disorders
2.0%
1/51 • Number of events 1
0.00%
0/53
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders
2.0%
1/51 • Number of events 1
1.9%
1/53 • Number of events 1
Nervous system disorders
Nervous system disorders
0.00%
0/51
1.9%
1/53 • Number of events 1
Renal and urinary disorders
Renal and urinary disorders
5.9%
3/51 • Number of events 3
1.9%
1/53 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
2.0%
1/51 • Number of events 1
0.00%
0/53

Other adverse events

Other adverse events
Measure
Cerefolin NAC + Multivitamin
n=51 participants at risk
On Cerefolin NAC and open-label multivitamin supplement
Cerefolin NAC Placebo + Multivitamin
n=53 participants at risk
On placebo and open-label multivitamin supplement
Cardiac disorders
Cardiac disorders
2.0%
1/51 • Number of events 1
3.8%
2/53 • Number of events 3
Eye disorders
Eye disorders
2.0%
1/51 • Number of events 1
3.8%
2/53 • Number of events 2
Gastrointestinal disorders
Gastrointestinal disorders
11.8%
6/51 • Number of events 6
5.7%
3/53 • Number of events 4
General disorders
General disorders
7.8%
4/51 • Number of events 4
9.4%
5/53 • Number of events 7
Metabolism and nutrition disorders
Metabolism and nutrition disorders
2.0%
1/51 • Number of events 1
3.8%
2/53 • Number of events 2
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders
9.8%
5/51 • Number of events 6
11.3%
6/53 • Number of events 7
Nervous system disorders
Nervous system disorders
3.9%
2/51 • Number of events 2
9.4%
5/53 • Number of events 5
Renal and urinary disorders
Renal and urinary disorders
2.0%
1/51 • Number of events 1
3.8%
2/53 • Number of events 2
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
11.8%
6/51 • Number of events 7
7.5%
4/53 • Number of events 4
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
7.8%
4/51 • Number of events 4
9.4%
5/53 • Number of events 5

Additional Information

Raj C. Shah, MD

Rush University Medical Center

Phone: 312-563-2902

Results disclosure agreements

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