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A Study to Assess the Safety of GRF6021 Infusions in Subjects With Parkinson's Disease and Cognitive Impairment

Primary Purpose

Parkinson Disease

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

GRF6021

Placebo

About this trial

This is an interventional treatment trial for Parkinson Disease focused on measuring Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders, Parkinson's Disease, Dementia

Eligibility Criteria

40 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Diagnosis of Parkinson's Disease (PD), with at least 1 year of PD symptoms.
Diagnosis of PD with mild cognitive impairment (PD-MCI) or probable or possible Parkinson's disease dementia according to Movement Disorder Society's Clinical Diagnostic criteria.
Score on the Montreal Cognitive Assessment (MoCA) of 13-25.
Modified Hoehn and Yahr Stages 1-4.
Modified Hachinski Ischemic Scale (MHIS) score of 4 or less.

Exclusion Criteria:

History of blood coagulation disorders or hypercoagulability.
Current use of anticoagulant therapy. Use of antiplatelet drugs (e.g., aspirin or clopidogrel) is acceptable.
Prior hypersensitivity reaction to any human blood product or any IV infusion.
Treatment with any human blood product, including transfusions and IV immunoglobulin, during the 6 months prior to screening.
History of immunoglobulin A or haptoglobin deficiency; stroke, anaphylaxis, or thromboembolic complications of IV immunoglobulins.
Heart disease, as evidenced by myocardial infarction, unstable, new onset or severe angina, or congestive heart failure in the 6 months prior to dosing
Hemoglobin < 10 g/dL in women and < 11 g/dL in men.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

GRF6021

Placebo

Arm Description

Subjects will receive GRF6021 for 5 consecutive days at Week 1 and Week 13.

Subjects will receive Placebo for 5 consecutive days at Week 1 and Week 13.

Outcomes

Primary Outcome Measures

Incidence of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Treatment-emergent adverse events identified by MedDRA preferred term and grouped by MedDRA System Organ Class

Secondary Outcome Measures

The Montreal Cognitive Assessment (MoCA) Score.

Change from baseline in the The Montreal Cognitive Assessment (MoCA). The MoCA is a 30-point test, which assess the attention and concentration, executive functions, memory, visuospatial abilities, language abilities, conceptual thinking, calculations, and orientation. Higher scores indicate better cognitive function; the total possible score is 30 and a score of 26 or more is considered normal. A positive value of change means an improvement, and a negative value of change means deterioration. Score range [0 (min) - 30 (Max)].

Continuity of Attention, Reaction Time Variability, Working Memory, and Episodic Memory on the Cognitive Drug Research Computerized Cognition Battery (CDR-CCB) as Assessed by Change From Baseline in CDR-CCB.

The CDR-CCB is an automated cognitive function assessment system. The secondary efficacy outcomes involved the following composite scores: Continuity of Attention: Min: - 20 # ; 35 # Reaction Time Variability: Min: 0 #; Max: 900 # Quality of Working Memory: Min : 0 # ; Max: 2 # Quality of Episodic Memory: Min: -400 #; Max: 400 # Note: # denotes "no specific unit" Lower scores reflect poorer ability for Continuity of Attention, Quality of Working Memory, and Quality of Episodic Memory; thus, a negative change from baseline reflects impairment compared to baseline. Whereas, for Reaction Time Variability, higher scores reflect poorer ability, and a positive change from baseline reflects impairment compared to baseline.

The Delis-Kaplan Executive Function System (D-KEFS) Verbal Fluency.

Change from baseline in the Delis-Kaplan Executive Function System (D-KEFS). The D-KEFS Verbal Fluency test is used for assessment of executive function and has three conditions: Letter Fluency, Category Fluency, and Category Switching. Higher scores indicate more correct responses. A positive value of change means an improvement and a negative value of change means deterioration. The minimum score is 0 and there is no concrete maximum score.

The Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) 1, 2, 3, and Total Score.

Change from baseline in the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS). The MDS-UPDRS contains 4 subscales: Part 1, Mentation, Behavior, and Mood; Part 2, Activities of Daily Living; Part 3, Motor; Part 4, Complications nonmotor experiences of daily living (13 items), motor experiences of daily living (13 items), motor examination (18 items), and motor complications (six items). The rating for each item is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores. For this study, Parts 1-3 will be completed. Part 1 score ranges from 0 to 52. Part 2 score ranges from 0 to 52. Part 3 score ranges from 0 to 132. Total score possible is 0 to 236.

The Schwab and England Activities of Daily Living (SE-ADL) Scale.

Change from baseline in the Schwab and England Activities of Daily Living (SE-ADL). The SE-ADL evaluates patients' perceptions of global functional capacity and dependence. Scoring is expressed in terms of percentage, in 10 steps from 100 to 0 (100%, normal status; 0%, bedridden with vegetative dysfunction), so that the lower the score, the worse the functional status. The range is 0% to 100%.

The Clinical Impression of Severity Index - PD (CISI-PD).

Change from baseline in The Clinical Impression of Severity Index PD (CISI-PD). The CISI-PD is a severity index formed by four items (motor signs, disability, motor complications, and cognitive status), rated 0 (not at all) to 6 (very severe or completely disabled); the possible scores range from 0 to 24. A total score is calculated by summing the item scores. Higher scores indicate worse severity. A negative value of change means an improvement and a positive value of change means deterioration.

The Parkinson's Disease Quality of Life Questionnaire-39 (PDQ-39).

Change from baseline in the Parkinson's Disease Quality of Life Questionnaire-39 (PDQ-39). The PDQ-39 is a self-administered questionnaire of 39 questions relating to 8 key areas of health and daily activities, including both motor and non-motor symptoms. It is scored on a scale of 0 -100 with lower scores indicating better health and high scores indicating more severe symptoms.

The Geriatric Depression Scale-15 (GDS-15).

Change from baseline in the Geriatric Depression Scale (GDS-15). The GDS-15 is a 15-item yes/no questionnaire of depression in older adults. Each depressive answer is 1 point. The final score is the tally of the number of depressive answers with the following scores indicating depression: 0-4 No depression; 5-10 Suggestive of a mild depression; 11 + Suggestive of severe depression. The possible scores range from 0 - 15.

The Digital Clock Drawing Test (dCDT).

Change from baseline in the digital clock drawing test (dCDT). The pen-like dCDT device will be used to gather the x-y coordinates that describe the movement of the stylus as it changes its position during the assessment. It also assesses when the stylus or writing device is not exerting pressure on the writing surface. The dCDT score is a number from 0 and 100 that represents a person's overall cognitive function as assessed by DCT clock. The total possible score is 100. A negative value of change means a deterioration and a positive value of change means an improvement.

Power of Attention, Cognitive Reaction Time, and Speed of Memory on the Cognitive Drug Research Computerized Cognition Battery (CDR-CCB) as Assessed by Change From Baseline in CDR-CCB.

The CDR-CCB is an automated cognitive function assessment system. The secondary efficacy outcomes involved the following composite scores: Power of Attention: Min: 350 ms ; Max: 60000 ms Cognitive Reaction Time: Min: - 30000 ms; Max : 30000 ms Speed of Memory: Min 800 ms; Max: 120000 ms Higher scores reflect poorer ability, and a positive change from baseline reflects impairment compared to baseline.

Full Information

NCT ID

NCT03713957

First Posted

October 18, 2018

Last Updated

May 5, 2022

Sponsor

Alkahest, Inc.

Collaborators

Michael J. Fox Foundation for Parkinson's Research

1. Study Identification

Unique Protocol Identification Number

NCT03713957

Brief Title

A Study to Assess the Safety of GRF6021 Infusions in Subjects With Parkinson's Disease and Cognitive Impairment

Official Title

A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Tolerability of GRF6021 Infusions in Subjects With Parkinson's Disease and Cognitive Impairment

Study Type

Interventional

2. Study Status

Record Verification Date

May 2022

Overall Recruitment Status

Completed

Study Start Date

November 12, 2018 (Actual)

Primary Completion Date

July 20, 2020 (Actual)

Study Completion Date

July 20, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alkahest, Inc.

Collaborators

Michael J. Fox Foundation for Parkinson's Research

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study will evaluate the safety, tolerability, and potential effects on cognition of GRF6021, a plasma-derived product, administered as an intravenous (IV) infusion, to subjects with Parkinson's disease and cognitive impairment.

Detailed Description

This is a randomized, double-blind, placebo-controlled study to assess the safety and tolerability of GRF6021, a plasma derived product, administered by intravenous (IV) infusion to subjects with Parkinson's disease (PD) and cognitive impairment. The study duration for the subjects will be approximately 7 months.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Parkinson Disease

Keywords

Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders, Parkinson's Disease, Dementia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

79 (Actual)

8. Arms, Groups, and Interventions

Arm Title

GRF6021

Arm Type

Experimental

Arm Description

Subjects will receive GRF6021 for 5 consecutive days at Week 1 and Week 13.

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Subjects will receive Placebo for 5 consecutive days at Week 1 and Week 13.

Intervention Type

Drug

Intervention Name(s)

GRF6021

Intervention Description

GRF6021 for IV infusion

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Placebo for IV infusion

Primary Outcome Measure Information:

Title

Incidence of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Description

Treatment-emergent adverse events identified by MedDRA preferred term and grouped by MedDRA System Organ Class

Time Frame

Approximately 24 Months

Secondary Outcome Measure Information:

Title

The Montreal Cognitive Assessment (MoCA) Score.

Description

Time Frame

Change from Baseline to Week 16

Title

Description

Time Frame

Change from Baseline to Week 20

Title

The Delis-Kaplan Executive Function System (D-KEFS) Verbal Fluency.

Description

Time Frame

Change from Baseline to Week 20

Title

The Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) 1, 2, 3, and Total Score.

Description

Time Frame

Change from Baseline to Week 16

Title

The Schwab and England Activities of Daily Living (SE-ADL) Scale.

Description

Time Frame

Change from Baseline to Week 24

Title

The Clinical Impression of Severity Index - PD (CISI-PD).

Description

Time Frame

Change from Baseline to Week 24

Title

The Parkinson's Disease Quality of Life Questionnaire-39 (PDQ-39).

Description

Time Frame

Change from Baseline to Week 20

Title

The Geriatric Depression Scale-15 (GDS-15).

Description

Time Frame

Change from Baseline to Week 20

Title

The Digital Clock Drawing Test (dCDT).

Description

Time Frame

Change from Baseline to Week 20

Title

Power of Attention, Cognitive Reaction Time, and Speed of Memory on the Cognitive Drug Research Computerized Cognition Battery (CDR-CCB) as Assessed by Change From Baseline in CDR-CCB.

Description

Time Frame

Change from Baseline to Week 20

10. Eligibility

Sex

All

Minimum Age & Unit of Time

40 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Diagnosis of Parkinson's Disease (PD), with at least 1 year of PD symptoms. Diagnosis of PD with mild cognitive impairment (PD-MCI) or probable or possible Parkinson's disease dementia according to Movement Disorder Society's Clinical Diagnostic criteria. Score on the Montreal Cognitive Assessment (MoCA) of 13-25. Modified Hoehn and Yahr Stages 1-4. Modified Hachinski Ischemic Scale (MHIS) score of 4 or less. Exclusion Criteria: History of blood coagulation disorders or hypercoagulability. Current use of anticoagulant therapy. Use of antiplatelet drugs (e.g., aspirin or clopidogrel) is acceptable. Prior hypersensitivity reaction to any human blood product or any IV infusion. Treatment with any human blood product, including transfusions and IV immunoglobulin, during the 6 months prior to screening. History of immunoglobulin A or haptoglobin deficiency; stroke, anaphylaxis, or thromboembolic complications of IV immunoglobulins. Heart disease, as evidenced by myocardial infarction, unstable, new onset or severe angina, or congestive heart failure in the 6 months prior to dosing Hemoglobin < 10 g/dL in women and < 11 g/dL in men.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Alkahest Medical Monitor

Organizational Affiliation

Alkahest, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Clinical Trials, Inc.

City

Little Rock

State/Province

Arkansas

ZIP/Postal Code

72205

Country

United States

Facility Name

Rocky Mountain Movement Disorders Center

City

Englewood

State/Province

Colorado

ZIP/Postal Code

80113

Country

United States

Facility Name

Moonshine Research Center

City

Doral

State/Province

Florida

ZIP/Postal Code

33166

Country

United States

Facility Name

Riverside Clinical Research

City

Edgewater

State/Province

Florida

ZIP/Postal Code

32132

Country

United States

Facility Name

MD Clinical

City

Hallandale Beach

State/Province

Florida

ZIP/Postal Code

33009

Country

United States

Facility Name

Research Centers of America, LLC

City

Hollywood

State/Province

Florida

ZIP/Postal Code

33024

Country

United States

Facility Name

Suncoast Research Group, LLC

City

Miami

State/Province

Florida

ZIP/Postal Code

33135

Country

United States

Facility Name

Qps_Mra, Llc

City

South Miami

State/Province

Florida

ZIP/Postal Code

33143

Country

United States

Facility Name

Atlanta Center for Medical Research

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30331

Country

United States

Facility Name

NeuroTrials Research Inc.

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30342

Country

United States

Facility Name

Quest Research Institute

City

Farmington Hills

State/Province

Michigan

ZIP/Postal Code

48334

Country

United States

Facility Name

SRI Biosciences

City

Plymouth

State/Province

Michigan

ZIP/Postal Code

48170

Country

United States

Facility Name

PsychCare Consultants Research

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63128

Country

United States

Facility Name

Wake Research

City

Raleigh

State/Province

North Carolina

ZIP/Postal Code

27612

Country

United States

Facility Name

Cleveland Clinic

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44195

Country

United States

Facility Name

Oregon Health & Science University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

UT Southwestern Medical Center

City

Dallas

State/Province

Texas

ZIP/Postal Code

75390

Country

United States

Facility Name

Centex Studies, INC.

City

Houston

State/Province

Texas

ZIP/Postal Code

77058

Country

United States

Facility Name

Northwest Clinical Research Center

City

Bellevue

State/Province

Washington

ZIP/Postal Code

98007

Country

United States

Facility Name

Alfred Hospital

City

Melbourne

State/Province

Victoria

ZIP/Postal Code

3004

Country

Australia

Facility Name

Hopital Neurologique

City

Bron

ZIP/Postal Code

69677

Country

France

Facility Name

Hopital Henri Mondor

City

Creteil

ZIP/Postal Code

94000

Country

France

Facility Name

CHU Grenoble Alpes

City

Grenoble

ZIP/Postal Code

38043

Country

France

Facility Name

Hopital Roger Salengro

City

Lille

ZIP/Postal Code

59000

Country

France

Facility Name

Hopital de la Timone

City

Marseille

ZIP/Postal Code

13385

Country

France

Facility Name

CHU Caremeau

City

Nimes

ZIP/Postal Code

30029

Country

France

Facility Name

CHU de Poitiers

City

Poitiers

ZIP/Postal Code

86021

Country

France

Facility Name

CHU Charles Nicolle

City

Rouen

ZIP/Postal Code

76000

Country

France

Facility Name

CHU Purpan - Hopital Pierre Paul Riquet

City

Toulouse

ZIP/Postal Code

31059

Country

France

12. IPD Sharing Statement

Learn more about this trial

A Study to Assess the Safety of GRF6021 Infusions in Subjects With Parkinson's Disease and Cognitive Impairment

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A Study to Assess the Safety of GRF6021 Infusions in Subjects With Parkinson's Disease and Cognitive Impairment

Parkinson Disease

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial