Parkinson DiseasePhase 2NCT03713957
A Study to Assess the Safety of GRF6021 Infusions in Subjects With Parkinson's Disease and Cognitive Impairment
A Phase 2 study testing GRF6021 in people with Parkinson Disease, run by Alkahest, Inc. at 29 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 40 to 85 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- GRF6021
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 79 participants
- Where
- 29 sitesin 3 countries
- Study dates
- 12 Nov 2018to 20 Jul 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
29 sites in 3 countries.
29 sites in 3 countries
United States19 sites
Atlanta Center for Medical Research
Atlanta, Georgia, 30331
NeuroTrials Research Inc.
Atlanta, Georgia, 30342
Northwest Clinical Research Center
Bellevue, Washington, 98007
Cleveland Clinic
Cleveland, Ohio, 44195
UT Southwestern Medical Center
Dallas, Texas, 75390
Moonshine Research Center
Doral, Florida, 33166
Riverside Clinical Research
Edgewater, Florida, 32132
Rocky Mountain Movement Disorders Center
Englewood, Colorado, 80113
Quest Research Institute
Farmington Hills, Michigan, 48334
MD Clinical
Hallandale, Florida, 33009
Research Centers of America, LLC
Hollywood, Florida, 33024
Centex Studies, INC.
Houston, Texas, 77058
Clinical Trials, Inc.
Little Rock, Arkansas, 72205
Suncoast Research Group, LLC
Miami, Florida, 33135
SRI Biosciences
Plymouth, Michigan, 48170
Oregon Health & Science University
Portland, Oregon, 97239
Wake Research
Raleigh, North Carolina, 27612
Qps_Mra, Llc
South Miami, Florida, 33143
PsychCare Consultants Research
St Louis, Missouri, 63128
France9 sites
Hopital Neurologique
Bron, 69677
Hopital Henri Mondor
Créteil, 94000
CHU Grenoble Alpes
Grenoble, 38043
Hopital Roger Salengro
Lille, 59000
Hopital de la Timone
Marseille, 13385
CHU Caremeau
Nîmes, 30029
CHU de Poitiers
Poitiers, 86021
CHU Charles Nicolle
Rouen, 76000
CHU Purpan - Hopital Pierre Paul Riquet
Toulouse, 31059
About this study
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
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.
Read the 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.
- Conditions
- Parkinson Disease
- Being tested
- GRF6021, Placebo
- Keywords
- Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders, Parkinson's Disease, Dementia
- Medical subject headings
- Parkinson Disease, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders, Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GRF6021Drug
GRF6021 for IV infusion
PlaceboOther
Placebo for IV infusion
| Study group | Role | What they receive |
|---|---|---|
| GRF6021 | Experimental | Subjects will receive GRF6021 for 5 consecutive days at Week 1 and Week 13. Receives: Drug: GRF6021 |
| Placebo | Placebo comparator | Subjects will receive Placebo for 5 consecutive days at Week 1 and Week 13. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Approximately 24 Months
Secondary outcomes (10)
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)].
Measured: Change from Baseline to Week 16
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.
Measured: Change from Baseline to Week 20
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.
Measured: Change from Baseline to Week 20
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.
Measured: Change from Baseline to Week 16
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%.
Measured: Change from Baseline to Week 24
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.
Measured: Change from Baseline to Week 24
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.
Measured: Change from Baseline to Week 20
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.
Measured: Change from Baseline to Week 20
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.
Measured: Change from Baseline to Week 20
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.
Measured: Change from Baseline to Week 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2018
- Study start12 Nov 2018
- Primary completion20 Jul 2020
- Study completion20 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alkahest, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Michael J. Fox Foundation for Parkinson's Research
- Responsible party
- Sponsor
- Investigators
- Alkahest Medical Monitor, Study director, Alkahest, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03713957
- Sponsor's protocol ID
- Alkahest study 6021-201
- Status last verified
- May 2022
- First submitted
- 18 Oct 2018
- Last updated
- 9 May 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03713957
- Acronym / sponsor ID
- Alkahest study 6021-201
- Conditions
- Parkinson Disease
- Keywords
- Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neurocognitive Disorders, Mental Disorders, Parkinson's Disease, Dementia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- Alkahest, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Michael J. Fox Foundation for Parkinson's Research
- Study start
- 12 Nov 2018 (actual)
- Primary completion
- 20 Jul 2020 (actual)
- Study completion
- 20 Jul 2020 (actual)
- First posted
- 22 Oct 2018 (actual)
- Results
- Posted 9 May 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GRF6021 | Experimental | Drug: GRF6021Subjects will receive GRF6021 for 5 consecutive days at Week 1 and Week 13. |
| Placebo | Placebo comparator | Other: PlaceboSubjects will receive Placebo for 5 consecutive days at Week 1 and Week 13. |
Who may qualify
- Ages
- 40 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GRF6021GRF6021 for IV infusion | Drug | GRF6021 |
| PlaceboPlacebo for IV infusion | Other | Placebo |
Primary outcome
Incidence of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Approximately 24 Months
Treatment-emergent adverse events identified by MedDRA preferred term and grouped by MedDRA System Organ Class
10 secondary outcomes
The Montreal Cognitive Assessment (MoCA) Score.
Time frame: Change from Baseline to Week 16
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.
Time frame: Change from Baseline to Week 20
The Delis-Kaplan Executive Function System (D-KEFS) Verbal Fluency.
Time frame: Change from Baseline to Week 20
The Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) 1, 2, 3, and Total Score.
Time frame: Change from Baseline to Week 16
The Schwab and England Activities of Daily Living (SE-ADL) Scale.
Time frame: Change from Baseline to Week 24
The Clinical Impression of Severity Index - PD (CISI-PD).
Time frame: Change from Baseline to Week 24
The Parkinson's Disease Quality of Life Questionnaire-39 (PDQ-39).
Time frame: Change from Baseline to Week 20
The Geriatric Depression Scale-15 (GDS-15).
Time frame: Change from Baseline to Week 20
The Digital Clock Drawing Test (dCDT).
Time frame: Change from Baseline to Week 20
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.
Time frame: Change from Baseline to Week 20
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alkahest Medical Monitor study director
Alkahest, Inc.
29 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GRF6021 in people with Parkinson Disease, run by Alkahest, Inc. at 29 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, France, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 May 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Approximately 24 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 May 2022.