Trial Outcomes & Findings for Proof-of-concept, Open-label Study in Patients With Early Alzheimer's Disease (NCT NCT05161715)
NCT ID: NCT05161715
Last Updated: 2025-01-29
Results Overview
Mean percent change from screening (V1) to end of treatment (V6) in ApoA-I in CSF
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
24 weeks
Results posted on
2025-01-29
Participant Flow
Participant milestones
| Measure |
10mg Obicetrapib
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
10mg Obicetrapib
n=13 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Age, Continuous
|
64.5 years
STANDARD_DEVIATION 6.2 • n=13 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=13 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=13 Participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis.
Mean percent change from screening (V1) to end of treatment (V6) in ApoA-I in CSF
Outcome measures
| Measure |
10mg Obicetrapib
n=12 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Mean Percent Change in Apolipoprotein A-I (ApoA-I) in Cerebrospinal Fluid (CSF)
|
-8.4 percent change from screening visit
Standard Deviation 13.6
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: For the PD endpoints in plasma, analyses were conducted on all 13 patients.
Mean percent change in ApoA-I in plasma from baseline (V2) to week 24 (V6)
Outcome measures
| Measure |
10mg Obicetrapib
n=13 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Mean Percent Change in Apolipoprotein A-I (ApoA-I) in Plasma
|
37.5 percent change from baseline
Standard Deviation 25.2
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis.
Mean percent change from screening (V1) to end of treatment (V6) in ApoE
Outcome measures
| Measure |
10mg Obicetrapib
n=12 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Mean Percent Change in Apolipoprotein-E (ApoE) in Cerebrospinal Fluid (CSF)
|
3.9 percent change from screening
Standard Deviation 10.3
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: For the PD endpoints in plasma, analyses were conducted on all 13 patients.
Mean percent change from baseline (V2) to week 24 (V6) in plasma in ApoE
Outcome measures
| Measure |
10mg Obicetrapib
n=13 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Mean Percent Change in Apolipoprotein-E (ApoE) in Plasma
|
47.8 percent change from baseline
Standard Deviation 46.9
|
PRIMARY outcome
Timeframe: baselinePopulation: Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis.
Small high-density lipoprotein (s-HDL) particle concentration in cerebrospinal fluid (CSF) measured by Ion Mobility Assay (https://doi.org/10.1002/alz.12649)
Outcome measures
| Measure |
10mg Obicetrapib
n=12 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Small HDL (s-HDL) Particle Concentration in Cerebrospinal Fluid (CSF) at Baseline
|
0.542 average relative abundance
Standard Deviation 0.138
|
PRIMARY outcome
Timeframe: Week 24Population: Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis.
Small high-density lipoprotein (s-HDL) particle concentration in CSF measured by Ion Mobility Assay (https://doi.org/10.1002/alz.12649)
Outcome measures
| Measure |
10mg Obicetrapib
n=12 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Small HDL (s-HDL) Particle Concentration in Cerebrospinal Fluid (CSF) at Week 24
|
0.542 average relative abundance
Standard Deviation 0.106
|
PRIMARY outcome
Timeframe: baselinePopulation: Due to a non-optimal sample preparation procedure, a number of the samples allocated for the small HDL-C particle analysis were lost and therefore no samples were assessed for this measure.
Small high-density lipoprotein (s-HDL) particle concentration plasma measured by Ion Mobility Assay. For more information on the measurement used please refer to this article: https://doi.org/10.1002/alz.12649
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Week 24Population: Due to a non-optimal sample preparation procedure, a number of the samples allocated for the small HDL-C particle analysis were lost and therefore no samples were assessed for this measure.
Small high-density lipoprotein (s-HDL) particle concentration in plasma measured by Ion Mobility Assay at Week 24 For more information on the measurement used please refer to this article: https://doi.org/10.1002/alz.12649
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 24 weeksPopulation: Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis
Mean percent change in cholesterol efflux capacity in CSF from screening (V1) to week 24 (V6)
Outcome measures
| Measure |
10mg Obicetrapib
n=12 Participants
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Mean Percent Change in Cholesterol Efflux Capacity in Cerebrospinal Fluid (CSF)
|
1.9 percent change from screening
Standard Deviation 24.1
|
Adverse Events
10mg Obicetrapib Tablets
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
10mg Obicetrapib Tablets
n=13 participants at risk
10mg obicetrapib (5mg tablets) administered orally daily for 24 weeks
Obicetrapib: 10mg obicetrapib
|
|---|---|
|
Blood and lymphatic system disorders
Normocytic anemia
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Cardiac disorders
Angina pectoris
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Ear and labyrinth disorders
Benign paroxysmal positional vertigo
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Gastrointestinal disorders
Constipation
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Gastrointestinal disorders
Loose stools
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Gastrointestinal disorders
Obstipation
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Gastrointestinal disorders
Reflux esophagitis
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
General disorders
Unilateral leg swelling
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Infections and infestations
Covid-19
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Infections and infestations
Gastroenteritis
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Infections and infestations
Herpes zoster
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Infections and infestations
Laryngitis
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Infections and infestations
Upper respiratory tract infection
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Injury, poisoning and procedural complications
Fall
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Injury, poisoning and procedural complications
Lumbar puncture headache
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Injury, poisoning and procedural complications
Rib contusion
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Investigations
Weight loss
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Musculoskeletal and connective tissue disorders
Hand arthritis
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Musculoskeletal and connective tissue disorders
Lateral epicondylitis
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Musculoskeletal and connective tissue disorders
Low back pain
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Musculoskeletal and connective tissue disorders
Lumbago
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma face
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Nervous system disorders
Headache
|
15.4%
2/13 • Number of events 2 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
|
Skin and subcutaneous tissue disorders
Hair loss
|
7.7%
1/13 • Number of events 1 • From first dose of study drug through Week 24.5
Safety Population included all participants who received at least 1 dose of any study drug. Analysis of PD endpoints in CSF were conducted on 12 patients as the lumbar puncture for one patient was unsuccessful at End of Treatment (Day 168, Visit 6) and no CSF was available for analysis. For the PD endpoints in plasma, analyses were conducted on all 13 patients.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee After the multicenter publication or 12 months after completion of the study, whichever occurs first, Institution may publish the results of its study data. Institution and PI shall provide Sponsor with an advance copy of any proposed communications at least 30 days prior and Sponsor shall have 30 days to review. Sponsor may request (a) the deletion of any Confidential Information, (b) reasonable changes, or (c) a delay for an additional 60 day period.
- Publication restrictions are in place
Restriction type: OTHER