Clinical trial NCT01745198: Effect of Cerefolin®/CerefolinNAC® on Biomarker Measurements
Official title: Effect of Cerefolin®/CerefolinNAC® on Biomarker Measurements in Patients With Mild Cognitive Impairment, Alzheimer's Disease and Related Disorders
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Mild Cognitive Impairment
Alzheimer's Disease
Alzheimer's Disease Related Disorders
Keywords: homocysteinemia
dementia
depression
vitamin B12
folate
mild cognitive impairment
alzheimer's
homocysteine
Lead sponsor: Pamlab, Inc.
Sponsor class: INDUSTRY
Collaborators: name: The Shankle Clinic
name: Hoag Memorial Hospital Presbyterian
First posted: 2012-12-10
Enrollment: 121
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with homocysteinemia plus past/current CFLN treatment (Treatment Group) will be matched to those without homocysteinemia plus no past/current B12, folate or CFLN treatment (Non-Treatment Group).

Brief summary: In a retrospective analysis of data from 1100 patients, disease-delaying effects of Cerefolin®/CerefolinNAC® were examined in terms of cognition. The purpose of the current study is to expand the retrospective study dataset by prospectively collecting additional biomarker and imaging data.

Detailed description: CerefolinNAC® is an orally administered prescription medical food, and is formulated as a combination of L-methylfolate calcium (as Metafolin®), methylcobalamin, and N-acetylcysteine. In a retrospective analysis, disease-delaying effects of Cerefolin®/CerefolinNAC® (CFLN) are examined in terms of cognition (measured by MCI Screen (MCIS)), and functional capacity (measured by Functional Assessment Staging Test (FAST)). - the treatment effect of CFLN on cognitive and functional measures, and on biomarker measures in patients with Alzheimer's disease and related disorders (ADRD).

The current study will expand the NAC-002b study dataset by prospectively collecting additional biomarker and imaging data in a more comprehensively assessed, matched sample of patients. This will allow more precise evaluation of cognitive and functional outcome measures, and biomarker measures will be assessed in an attempt to identify specific populations or conditions in which CFLN is most effective.

The sample will consist of patients with homocysteinemia plus past/current CFLN treatment (Treatment Group) matched to those without homocysteinemia plus no past/current B12, folate or CFLN treatment (Non-Treatment Group). Also 65 additional subjects will be recruited for the non-Treatment group, which will be used to improve the rate of decline estimates for the cognitive and functional outcome measures.

Eligibility criteria: Inclusion Criteria:

* With a diagnosis of normal aging (NL), cognitive impairment or dementia not otherwise specified (CI/D), or ADRD
* With at least one previous quantitative MRI (qMRI)
* With at least one previous homocysteine level
* Without homocysteinemia plus no past or current B12, folate or Cerefolin® treatment, OR with homocysteinemia plus past or current Cerefolin® treatment

Exclusion Criteria:

Subjects who do not meet the inclusion criteria will be excluded from the study.

Primary outcomes:
measure: Change in rate of cognitive decline as measured by the Memory Performance Index (MPI)
timeFrame: Baseline to end of study (estimated average of 48 months)
Secondary outcomes:
measure: Change in rate of cognitive decline as measured by the MCI Screen
timeFrame: Baseline to end of study (estimated average of 48 months)
measure: Change in rate of cognitive decline as measured by The Consortium to Establish a Registry for Alzheimer's Disease (CERAD) Drawings
timeFrame: Baseline to end of study (estimated average of 48 months)
measure: Change in rate of cognitive decline as measured by Trails A & B
timeFrame: Baseline to end of study (estimated average of 48 months)
measure: Rate of atrophy of hippocampal volume
timeFrame: Baseline to end of study (estimated average of 48 months)
measure: Rate of atrophy in cortical volume
timeFrame: Baseline to end of study (estimated average of 48 months)
measure: Rate of atrophy in ventricular volume
timeFrame: Baseline to end of study (estimated average of 48 months)
measure: Change in rate of cognitive decline as measured by Functional Assessment Staging Test (FAST)
timeFrame: Baseline to end of study (estimated average of 48 months)
Other outcomes:
Locations:
city: Newport Beach
country: United States
facility: Hoag Memorial Hospital
state: California
Study officials:
affiliation: Shankle Clinic
name: William R Shankle, MS, MD, FACP
role: PRINCIPAL_INVESTIGATOR
References: