A Study to Assess the Safety of GRF6019 Infusions in Subjects With Severe Alzheimer's Disease

NCT03765762 | Completed Phase 2 Results FDA Drug

• 1 other identifier: Alkahest study 6019-202
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 6

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.

Conditions

Severe Alzheimer Disease

Keywords

Brain Diseases; Nervous System Diseases; Central Nervous System Diseases; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Dementia; Tauopathy

Outcome Measures

Primary Outcomes (2)

• Frequency of Treatment-emergent Adverse Events (Safety)

  Number of Subjects with at Least One Treatment-emergent adverse event by MedDRA preferred term and grouped by MedDRA System Organ Class

  5 weeks

• Tolerability of GRF6019

  Tolerability of treatment defined by the number of subjects completing 4 weeks of study after receiving 5 daily infusions

  5 weeks

Secondary Outcomes (6)

• The Mini-Mental State Examination (MMSE) Score

  Baseline and 5 weeks

• Severe Impairment Battery (SIB) Total Score

  Baseline and 5 weeks

• Alzheimer's Disease Cooperative Study Group Activities of Daily Living Inventory for Severe Alzheimer's Disease (ADCS-ADL-Severe)

  Baseline and 5 weeks

• Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change Plus Caregiver Input (ADCS-CGIC)

  Baseline and 5 weeks

• Neuropsychiatric Inventory Nursing Home (NPI-NH) Total Score

  Baseline and 5 weeks

Study Arms (2)

GRF6019

EXPERIMENTAL

Subjects will receive intravenously 250 mL of GRF6019 each day for 5 consecutive days.

Drug: GRF6019

Placebo

PLACEBO COMPARATOR

Subjects will receive intravenously 250 mL of placebo each day for 5 consecutive days.

Other: Placebo

Interventions

GRF6019 DRUG

GRF6019 for IV infusion

GRF6019

Placebo OTHER

Placebo for IV infusion

Also known as: Normal Saline

Placebo

Eligibility Criteria

• Age: 60 Years - 95 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alkahest, Inc.: lead

Study Sites (6)

Cognitive Clinical Trials

Gilbert, Arizona, 85297, United States

Location

Cognitive Clinical Trials

Mesa, Arizona, 85209, United States

Location

Cognitive Clinical Trials

Phoenix, Arizona, 85037, United States

Location

Pacific Research Network

San Diego, California, 92103, United States

Location

Riverside Clinical Research

Edgewater, Florida, 32132, United States

Location

Bio Behavioral Health

Toms River, New Jersey, 08755, United States

Location

MeSH Terms

Conditions

Alzheimer Disease; Brain Diseases; Nervous System Diseases; Central Nervous System Diseases; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Dementia; Tauopathies

Interventions

Saline Solution

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: Head of Communications
• Organization: Alkahest, Inc.

info@alkahest.com

(650) 801-0474

Study Officials

• Alkahest Program Physician

  Alkahest, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 29, 2018
• First Posted: December 5, 2018
• Study Start: January 15, 2019
• Primary Completion: December 17, 2019
• Study Completion: December 17, 2019
• Last Updated: January 27, 2021
• Results First Posted: January 27, 2021

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)