Clinical trial NCT03520998: A Randomized Study to Assess the Safety of GRF6019 Infusions in Subjects With Mild to Moderate Alzheimer's Disease
Official title: A Prospective, Randomized, Double-Blind, Dose-Comparison Concurrent Control Study to Assess the Safety and Tolerability of GRF6019 Infusions in Subjects With Mild to Moderate Alzheimer's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alzheimer Disease
Mild to Moderate Alzheimer Disease
Lead sponsor: Alkahest, Inc.
Sponsor class: INDUSTRY
First posted: 2018-05-11
Enrollment: 47
Sex: ALL
Minimum age (years): 60
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is evaluating the safety, tolerability, and feasibility of GRF6019, a plasma-derived product, administered as an intravenous (IV) infusion, to subjects with mild to moderate Alzheimer's disease.

Detailed description: This is a randomized, double-blind, dose-comparison concurrent control study to assess the safety, tolerability, and feasibility of GRF6019, a plasma-derived product, administered by intravenous (IV) infusion to subjects with mild to moderate Alzheimer's disease.

Subjects will be randomized 1:1 to a low dose or a high dose of active treatment in a double-blind manner. All subjects will receive one infusion per day at the randomized dose for 5 consecutive days during Week 1 and, again, during Week 13 (for a total of 10 doses per subject). All IV infusions will take place at an inpatient research unit while the follow-up visits after each treatment period will be on an outpatient basis. Subjects will participate for a total of 6 months in this study.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of probable AD based upon the National Institute on Aging-Alzheimer's Association (NIA-AA) Criteria
* MMSE Score 12-24 inclusive
* Modified Hachinski Ischemia Scale (MHIS) score of ≤ 4
* Provided a signed and dated informed consent form (either the subject and/or subject's legal representative as well as the trial partner)

Exclusion Criteria:

* Evidence of clinically relevant neurological disorder(s) other than probable AD
* History of blood coagulation disorders or hypercoagulability; any concurrent use of an anticoagulant therapy. (e.g., heparin, warfarin, thrombin inhibitors, Factor Xa inhibitors). Use of antiplatelet drugs (e.g., aspirin or clopidogrel) is acceptable.
* Initiation or change in the dosage of cholinesterase inhibitors (AChEI), memantine, Axona, vitamin E supplementation or selegiline within 3 months prior to screening.
* Heart disease (or history thereof), as evidenced by myocardial infarction, unstable, new onset or severe angina, or congestive heart failure (New York Association Class II, III or IV) in the 6 months prior to dosing; uncontrolled high blood pressure (systolic blood pressure of 160 mmHg or higher and/or diastolic blood
* Prior hypersensitivity reaction to any human blood product or intravenous infusion; any known clinically significant drug allergy.
* Treatment with any human blood product, including transfusions and intravenous immunoglobulin, during the 6 months prior to screening.
* History of immunoglobulin A (IgA), haptoglobulin or C1 inhibitor deficiency; stroke, anaphylaxis, or thromboembolic complications of intravenous immunoglobulins.
* Hemoglobin \<10 g/dL in women; and \<11 g/dL in men.

Primary outcomes:
measure: Frequency of Treatment-emergent Adverse Events (Safety)
timeFrame: Baseline to 6 months
Secondary outcomes:
measure: The Mini-Mental State Examination (MMSE)
timeFrame: Baseline and 6 months
measure: Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADASCog/11)
timeFrame: Baseline and 6 months
measure: The Clinical Dementia Rating Scale - Sum of Boxes (CDR-SOB)
timeFrame: Baseline and 6 months
measure: The Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL23)
timeFrame: Baseline and 6 months
measure: The Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change (ADCS-CGIC)
timeFrame: Baseline and 6 months
measure: The Neuropsychiatric Inventory Questionnaire (NPI-Q)
timeFrame: Baseline and 6 months
Other outcomes:
Locations:
city: Lemon Grove
country: United States
facility: Synergy East
state: California
city: Long Beach
country: United States
facility: CNS Network
state: California
city: San Diego
country: United States
facility: Pacific Research Network
state: California
city: Hallandale Beach
country: United States
facility: MD Clinical
state: Florida
city: Miami
country: United States
facility: Miami Jewish Health Systems
state: Florida
city: North Miami
country: United States
facility: Behavioral Clinical Research
state: Florida
city: Orlando
country: United States
facility: Bioclinica Research
state: Florida
city: Princeton
country: United States
facility: Princeton Medical Institute
state: New Jersey
city: DeSoto
country: United States
facility: Serenity Inpatient
state: Texas
city: Salt Lake City
country: United States
facility: PRA Health Sciences
state: Utah
Study officials:
affiliation: Alkahest, Inc.
name: Alkahest Medical Monitor
role: STUDY_DIRECTOR
References: