Trial Outcomes & Findings for A Randomized Study to Assess the Safety of GRF6019 Infusions in Subjects With Mild to Moderate Alzheimer's Disease (NCT NCT03520998)
NCT ID: NCT03520998
Last Updated: 2021-01-29
Results Overview
Treatment-emergent adverse events identified by MedDRA preferred term and grouped by MedDRA System Organ Class
COMPLETED
PHASE2
47 participants
Baseline to 6 months
2021-01-29
Participant Flow
Participant milestones
| Measure |
GRF6019 Low Dose
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
Overall Study
STARTED
|
24
|
23
|
|
Overall Study
COMPLETED
|
18
|
21
|
|
Overall Study
NOT COMPLETED
|
6
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Randomized Study to Assess the Safety of GRF6019 Infusions in Subjects With Mild to Moderate Alzheimer's Disease
Baseline characteristics by cohort
| Measure |
GRF6019 Low Dose
n=24 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=23 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
Total
n=47 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
75.9 years
STANDARD_DEVIATION 6.26 • n=5 Participants
|
72.7 years
STANDARD_DEVIATION 7.21 • n=7 Participants
|
74.3 years
STANDARD_DEVIATION 6.85 • n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
23 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=5 Participants
|
23 participants
n=7 Participants
|
47 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to 6 monthsPopulation: Safety Set
Treatment-emergent adverse events identified by MedDRA preferred term and grouped by MedDRA System Organ Class
Outcome measures
| Measure |
GRF6019 Low Dose
n=24 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=23 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
Frequency of Treatment-emergent Adverse Events (Safety)
|
18 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Evaluable Set and Per Protocol Set
Changes in scores on the MMSE. The MMSE consists of 5 components: orientation to time and place, registration of 3 words, attention and calculation, recall of 3 words, and language. The scores from the 5 components are summed to obtain the overall MMSE total score. The MMSE total score can range from 0 to 30, with higher scores indicating better cognition.
Outcome measures
| Measure |
GRF6019 Low Dose
n=21 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=22 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
The Mini-Mental State Examination (MMSE)
|
-1.0 score on a scale
Standard Deviation 4.3
|
1.5 score on a scale
Standard Deviation 3.9
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Evaluable Set and Per Protocol Set
Changes in scores on the 11-item ADASCog/11. The ADAS-Cog/11 includes 11 items assessing cognitive function. The domains include memory, language, praxis, and orientation. There are 70 possible points. Higher scores reflect greater cognitive impairment.
Outcome measures
| Measure |
GRF6019 Low Dose
n=21 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=22 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADASCog/11)
|
-0.4 score on a scale
Standard Deviation 5.1
|
-0.9 score on a scale
Standard Deviation 4.3
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Evaluable Set and Per Protocol Set
Changes in the CDR-SOB. The CDR characterizes functioning in 6 domains: memory, orientation, judgment and problem solving, community affairs, home and hobbies and personal care. The score is obtained by summing each of the domain box scores. Scores range from 0 to 18 with higher scores reflecting worse cognition.
Outcome measures
| Measure |
GRF6019 Low Dose
n=21 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=22 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
The Clinical Dementia Rating Scale - Sum of Boxes (CDR-SOB)
|
-0.03 score on a scale
Standard Deviation 2.3
|
0.21 score on a scale
Standard Deviation 1.7
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Evaluable Set and Per Protocol Set
Changes in the ADCS-ADL23. The ADCS-ADL23 assesses basic and instrumental activities of daily living covering physical and mental functioning and independence in self-care. The score ranges from 0 to 78 with higher scores indicating less functional impairment.
Outcome measures
| Measure |
GRF6019 Low Dose
n=21 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=22 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
The Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL23)
|
-0.7 score on a scale
Standard Deviation 7.4
|
-1.3 score on a scale
Standard Deviation 4.6
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Evaluable Set and Per Protocol Set
The ADCS-CGIC focuses on clinicians' observations of change in the subject's cognitive, functional, and behavioral performance since the beginning of a trial. The ADCS-CGIC is a 7-point scale with lower values (\<4) representing an improvement, higher values (\>4) representing a worsening, and a value of 4 indicating no change.
Outcome measures
| Measure |
GRF6019 Low Dose
n=21 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=22 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
The Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change (ADCS-CGIC)
|
4.1 score on a scale
Standard Deviation 0.8
|
4.1 score on a scale
Standard Deviation 0.7
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Evaluable Set and Per Protocol Set
Change on the NPI-Q. The NPI-Q comprises 12 domains: delusions, hallucinations, dysphoria, apathy, euphoria, disinhibition, aggressivity and restlessness, irritability, anxiety aberrant motor behavior, appetite and eating disorders, and nocturnal behavior. The severity of the reported symptoms is assessed on a 3-point scale. The total severity score can range from 0 to 36 with higher scores representing worse severity.
Outcome measures
| Measure |
GRF6019 Low Dose
n=21 Participants
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=22 Participants
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
The Neuropsychiatric Inventory Questionnaire (NPI-Q)
|
-2.3 score on a scale
Standard Deviation 3.8
|
-1.2 score on a scale
Standard Deviation 3.1
|
Adverse Events
GRF6019 Low Dose
GRF6019 High Dose
Serious adverse events
| Measure |
GRF6019 Low Dose
n=24 participants at risk
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=23 participants at risk
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/24 • 6 months
|
4.3%
1/23 • Number of events 1 • 6 months
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
0.00%
0/24 • 6 months
|
4.3%
1/23 • Number of events 1 • 6 months
|
Other adverse events
| Measure |
GRF6019 Low Dose
n=24 participants at risk
Low dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
GRF6019 High Dose
n=23 participants at risk
High dose of GRF6019 for 5 consecutive days at Weeks 1 and 13
|
|---|---|---|
|
General disorders
Infusion site extravasation
|
12.5%
3/24 • Number of events 3 • 6 months
|
8.7%
2/23 • Number of events 2 • 6 months
|
|
General disorders
Infusion site haematoma
|
4.2%
1/24 • Number of events 1 • 6 months
|
17.4%
4/23 • Number of events 4 • 6 months
|
|
Nervous system disorders
Headache
|
12.5%
3/24 • Number of events 4 • 6 months
|
21.7%
5/23 • Number of events 7 • 6 months
|
|
Investigations
Amylase increased
|
4.2%
1/24 • Number of events 1 • 6 months
|
8.7%
2/23 • Number of events 3 • 6 months
|
|
Investigations
Lipase increased
|
4.2%
1/24 • Number of events 1 • 6 months
|
8.7%
2/23 • Number of events 3 • 6 months
|
|
Gastrointestinal disorders
Diarrhoea
|
4.2%
1/24 • Number of events 1 • 6 months
|
8.7%
2/23 • Number of events 2 • 6 months
|
|
Injury, poisoning and procedural complications
Fall
|
4.2%
1/24 • Number of events 1 • 6 months
|
8.7%
2/23 • Number of events 2 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.3%
2/24 • Number of events 2 • 6 months
|
0.00%
0/23 • 6 months
|
|
Vascular disorders
Hypertension
|
4.2%
1/24 • Number of events 1 • 6 months
|
8.7%
2/23 • Number of events 3 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60