A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Tolerability of Pulsed GRF6019 Infusions in Subjects With Severe Alzheimer's Disease

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

Start Date15 Jan 2019

Sponsor / Collaborator

Alkahest, Inc.Startup

NCT03520998 / CompletedPhase 2

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

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.

Start Date16 Apr 2018

Sponsor / Collaborator

Alkahest, Inc.Startup

100 Clinical Results associated with GRF6019

100 Translational Medicine associated with GRF6019

100 Patents (Medical) associated with GRF6019

Literatures (Medical) associated with GRF6019

15 Jun 2021·Journal of Alzheimer's DiseaseQ3 · MEDICINE

Safety and Tolerability of GRF6019 Infusions in Severe Alzheimer’s Disease: A Phase II Double-Blind Placebo-Controlled Trial

Q3 · MEDICINE

Article

Author: Nikolich, Karoly ; Albensi, Benedict ; Koborsi, Katie ; Cummings, Jeffrey L. ; Thein, Stephen G. ; Duclos, Tiffanie ; Scott, James ; Chao, Whitney ; Braithwaite, Steven ; Patel, Ashok K. ; Kay, Gary ; Hannestad, Jonas ; Klutzaritz, Vicki ; Beck, Brian

Background: The plasma fraction GRF6019 shows multiple benefits on brain aging in mice, including enhanced cognition, neurogenesis, and synaptic density, as well as reduced neuroinflammation. Objective: To evaluate the safety, tolerability, and preliminary efficacy of GRF6019 in patients with severe Alzheimer’s disease (AD). Methods: A phase II, double-blind, placebo-controlled study in patients with severe AD (Mini-Mental State Examination score 0–10). Patients were randomized 2 : 1 to GRF6019 (N = 18) or placebo (N = 8) and received daily 250 mL intravenous infusions over 5 days. The primary endpoints were the rates of adverse events (AEs) and the tolerability of GRF6019 as assessed by the number of patients completing the study. Change from baseline in cognitive and functional assessments was also evaluated. Results: All patients completed 100%of study visits and infusions. The rate of AEs was similar in the GRF6019 (8/18 patients [44.4%]) and placebo (3/8 patients [37.5%]) groups, and there were no deaths or serious AEs. The most common AEs considered related to treatment were mild, transient changes in blood pressure in the GRF6019 group (hypotension: 2 patients [11.1%]; hypertension: 1 patient [5.6%]); there were no related AEs in the placebo group. The trial was not powered to detect statistically significant differences between treatment groups. At the end of the study, patients in both treatment groups remained stable or improved on all cognitive and functional endpoints. Conclusion: GRF6019 demonstrated excellent safety, feasibility, and tolerability. Future trials designed to characterize the potential functional benefits of GRF6019 and related plasma fractions in severe AD are warranted.

01 Jan 2020·Alzheimer's & Dementia: Translational Research & Clinical Interventions

Safety and tolerability of GRF6019 in mild‐to‐moderate Alzheimer's disease dementia

Article

Author: Páez, Antonio ; Jackson, Sam ; Hannestad, Jonas ; Nikolich, Karoly ; Ray, Rebecca ; Klutzaritz, Vicki ; Kay, Gary ; Koborsi, Katie ; Chao, Whitney ; Braithwaite, Steven ; Cummings, Jeffrey L. ; Lohr, Scott

AbstractIntroductionThis phase 2 trial evaluated the safety, tolerability, and feasibility of repeated infusions of the plasma fraction GRF6019 in mild‐to‐moderate Alzheimer's disease.MethodsIn this randomized, double‐blind, dose‐comparison trial, 47 patients were randomized 1:1 to receive daily infusions of 100 mL (n = 24) or 250 mL (n = 23) of GRF6019 for 5 consecutive days over two dosing periods separated by a treatment‐free interval of 3 months.ResultsThe mean (standard deviation [SD]) age of the enrolled patients was 74.3 (6.9), and 62% were women. Most adverse events (55%) were mild, with no clinically significant differences in safety or tolerability between the two dose levels. The mean (SD) baseline Mini‐Mental State Examination score was 20.6 (3.7) in the 100 mL group and 19.6 (3.7) in the 250 mL group; at 24 weeks, the within‐patient mean change from baseline was –1.0 points (95% confidence interval [CI], –3.1 to 1.1) in the 100 mL group and +1.5 points (95% CI, –0.4 to 3.3) in the 250 mL group. The within‐patient mean change from baseline on the Alzheimer's Disease Assessment Scale‐Cognitive subscale was –0.4 points (95% CI, –2.9 to 2.2) in the 100 mL group, while in the 250 mL group it was –0.9 points (95% CI, –3.0 to 1.2). The within‐patient mean change from baseline on the Alzheimer's Disease Cooperative Study‐Activities of Daily Living was –0.7 points in the 100 mL group (95% CI, –4.3 to 3.0) and –1.3 points (95% CI, –3.4 to 0.7) in the 250 mL group. The mean change from baseline on the Category Fluency Test, Clinical Dementia Rating Scale–Sum of Boxes, Alzheimer's Disease Cooperative Study–Clinical Global Impression of Change, and Neuropsychiatric Inventory Questionnaire was similar for both treatment groups and did not show any worsening.DiscussionGRF6019 was safe and well tolerated, and patients experienced no cognitive decline and minimal functional decline. These results support further development of GRF6019.

100 Deals associated with GRF6019

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Alzheimer Disease	Phase 2	US	Alkahest, Inc.Startup	16 Apr 2018

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


AAIC2022	Not Applicable	Alzheimer Disease	-	GRF6019 100 mL	mwnltsvgwb(bnwsiwuump) = pxnlacmxky qgmcjhwpwi (rqxosghkoj, > 0.05) View more	-	01 Feb 2022
				GRF6019 250 mL	mwnltsvgwb(bnwsiwuump) = djumjxkhqy qgmcjhwpwi (rqxosghkoj, > 0.05) View more
NCT03765762 (CTgov)	Phase 2	Alzheimer Disease	26	GRF6019 (GRF6019)	qpfhvabzmx(kxdnifrdbs) = kihiqlaegk wdienjobhn (uumfyaasxo, pdyfslvcbj - yiotvswywq) View more	-	27 Jan 2021
				Placebo (Placebo)	qpfhvabzmx(kxdnifrdbs) = kaytbxazfc wdienjobhn (uumfyaasxo, ueymwoycbz - xzylwlnkhj) View more
NCT03520998 (Pubmed) Manual	Phase 2	Alzheimer Disease	47	GRF6019 (100 mL)	(hxtdndmeor) = cwkssstmgc nshrsrzjek (wujodfsfge, -2.9 to 2.2) View more	Positive	16 Dec 2020
				GRF6019 (250 mL)	(hxtdndmeor) = sglqeoynfi nshrsrzjek (wujodfsfge, -3.0 to 1.2) View more
NCT03520998 (CTgov)	Phase 2	Alzheimer Disease	47	GRF6019 (GRF6019 Low Dose)	swyvwwjsxc(wchiowlykd) = imqgxiktej euxnaxjpxr (khafjlixgo, qecyqmntrg - rpceeacecm) View more	-	05 Nov 2020
				GRF6019 (GRF6019 High Dose)	swyvwwjsxc(wchiowlykd) = yjzqtyqqmf euxnaxjpxr (khafjlixgo, pyaivdzikg - hdhgappidt) View more

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