Clinical trial NCT03765762: A Study to Assess the Safety of GRF6019 Infusions in Subjects With Severe Alzheimer's Disease
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Tolerability of Pulsed GRF6019 Infusions in Subjects With Severe Alzheimer's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Alzheimer Disease
Keywords: Brain Diseases
Nervous System Diseases
Central Nervous System Diseases
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders
Dementia
Tauopathy
Lead sponsor: Alkahest, Inc.
Sponsor class: INDUSTRY
First posted: 2018-12-05
Enrollment: 26
Sex: ALL
Minimum age (years): 60
Maximum age (years): 95
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the safety, tolerability, and potential cognitive benefit of the experimental treatment GRF6019 in subjects with severe Alzheimer's disease.

Detailed description: This is a randomized, double-blind, placebo-controlled study to assess the safety, tolerability and potential cognitive benefit of GRF6019, a human plasma protein fraction. GRF6019 or placebo will be administered intravenously to subjects with severe Alzheimer's disease every day for 5 consecutive days. The total study duration for each subject is approximately 9 weeks.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of probable AD according to the National Institute on Aging-Alzheimer's Association (NIA-AA) Criteria
* MMSE Score 0-10 inclusive
* Modified Hachinski Ischemia Scale (MHIS) score of 4 or less
* Provided a signed and dated informed consent form (either the subject and/or subject's legal representative)

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.
* Unstable coronary heart disease, e.g. myocardial infarction or severe or unstable angina in the 6 months prior to dosing.
* Moderate to severe congestive heart failure (New York Association Class III or IV).
* Poorly controlled high blood pressure (systolic blood pressure of 160 mmHg or higher and/or diastolic blood pressure of 100 mmHg or higher) despite treatment during the 3 months prior to dosing, or treatment refractory high blood pressure, defined as treatment requiring 3 or more antihypertensives from different classes.
* 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: 5 weeks
measure: Tolerability of GRF6019
timeFrame: 5 weeks
Secondary outcomes:
measure: The Mini-Mental State Examination (MMSE) Score
timeFrame: Baseline and 5 weeks
measure: Severe Impairment Battery (SIB) Total Score
timeFrame: Baseline and 5 weeks
measure: Alzheimer's Disease Cooperative Study Group Activities of Daily Living Inventory for Severe Alzheimer's Disease (ADCS-ADL-Severe)
timeFrame: Baseline and 5 weeks
measure: Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change Plus Caregiver Input (ADCS-CGIC)
timeFrame: Baseline and 5 weeks
measure: Neuropsychiatric Inventory Nursing Home (NPI-NH) Total Score
timeFrame: Baseline and 5 weeks
measure: Neuropsychiatric Inventory (NPI) Caregiver Total Score
timeFrame: Baseline and 5 weeks
Other outcomes:
Locations:
city: Gilbert
country: United States
facility: Cognitive Clinical Trials
state: Arizona
city: Mesa
country: United States
facility: Cognitive Clinical Trials
state: Arizona
city: Phoenix
country: United States
facility: Cognitive Clinical Trials
state: Arizona
city: San Diego
country: United States
facility: Pacific Research Network
state: California
city: Edgewater
country: United States
facility: Riverside Clinical Research
state: Florida
city: Toms River
country: United States
facility: Bio Behavioral Health
state: New Jersey
Study officials:
affiliation: Alkahest, Inc.
name: Alkahest Program Physician
role: STUDY_DIRECTOR
References: