Trial Outcomes & Findings for HERV-K Suppression Using Antiretroviral Therapy in Volunteers With Amyotrophic Lateral Sclerosis (ALS) (NCT NCT02437110)
NCT ID: NCT02437110
Last Updated: 2024-01-09
Results Overview
Blood samples were obtained during the screening visit and week 24 (post-treatment with antiretroviral drugs). The percent change in HERV-K level was measured using quantitative PCR: 100% x (screening visit - week 24 visit measurement) / screening visit.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
122 participants
Primary outcome timeframe
24 weeks
Results posted on
2024-01-09
Participant Flow
Participants were referred to the study by their provider or self-referred between April 2019 and June 2022. In total,122 participants were screened to determine HERV-K levels and eligibility to the treatment arm of the study. 118 participants were screened in protocol 15N0126 (Period 1) and 4 were screened in a companion protocol.
34 participants qualified and were interested in participating in the treatment portion of the protocol (Period 2). The follow-up period (Period 3), included a post treatment follow-up period from week 24 to week 48. Of the 20 participants who completed the treatment portion of the protocol,19 qualified for the follow-up period. One participant was excluded from the follow-up period (Period 3) due to a deviation from the protocol.
Participant milestones
| Measure |
Participants With ALS
Adults with sporadic or familial ALS
|
|---|---|
|
Screening
STARTED
|
118
|
|
Screening
COMPLETED
|
114
|
|
Screening
NOT COMPLETED
|
4
|
|
ART
STARTED
|
34
|
|
ART
COMPLETED
|
20
|
|
ART
NOT COMPLETED
|
14
|
|
Post-ART
STARTED
|
19
|
|
Post-ART
COMPLETED
|
9
|
|
Post-ART
NOT COMPLETED
|
10
|
Reasons for withdrawal
| Measure |
Participants With ALS
Adults with sporadic or familial ALS
|
|---|---|
|
Screening
Lost to Follow-up
|
3
|
|
Screening
Physician Decision
|
1
|
|
ART
Adverse Event
|
3
|
|
ART
Protocol Violation
|
3
|
|
ART
Withdrawal by Subject
|
4
|
|
ART
Lost to follow-up prior to start of intervention
|
1
|
|
ART
Physician decision prior to start of intervention
|
2
|
|
ART
Withdrawal by subject prior to starting intervention
|
1
|
|
Post-ART
Lost to Follow-up
|
3
|
|
Post-ART
Protocol Violation
|
4
|
|
Post-ART
Withdrawal by Subject
|
3
|
Baseline Characteristics
HERV-K Suppression Using Antiretroviral Therapy in Volunteers With Amyotrophic Lateral Sclerosis (ALS)
Baseline characteristics by cohort
| Measure |
ALS ART Treatment
n=34 Participants
Participants with ALS participating in the treatment period of the trial.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
25 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
31 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeksBlood samples were obtained during the screening visit and week 24 (post-treatment with antiretroviral drugs). The percent change in HERV-K level was measured using quantitative PCR: 100% x (screening visit - week 24 visit measurement) / screening visit.
Outcome measures
| Measure |
Patients With HERV-K 13
n=20 Participants
20 participants with ALS and a level of HERV-K:RPP30 greater than or equal to 13
|
|---|---|
|
Percent Change in HERV-K Level
|
-30.7 Percent change
Standard Error 0.06
|
Adverse Events
Screening Period
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
ART Treatment Period
Serious events: 0 serious events
Other events: 30 other events
Deaths: 0 deaths
Post-ART Follow-Up Period
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Screening Period
n=118 participants at risk
Adverse events noted in participants during the Screening period
|
ART Treatment Period
n=34 participants at risk
Adverse events noted in participants during the ART Treatment period
|
Post-ART Follow-Up Period
n=19 participants at risk
Adverse events noted in participants following the ART Treatment period
|
|---|---|---|---|
|
Investigations
Alanine Aminotransferase Increased
|
0.00%
0/118 • 48 weeks
|
23.5%
8/34 • Number of events 8 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Aspartate Aminotransferase Increased
|
0.00%
0/118 • 48 weeks
|
8.8%
3/34 • Number of events 3 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Blood Bicarbonate Decreased
|
0.00%
0/118 • 48 weeks
|
5.9%
2/34 • Number of events 2 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Nervous system disorders
Cerebrospinal Fluid Leakage
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Cholesterol High
|
0.00%
0/118 • 48 weeks
|
41.2%
14/34 • Number of events 14 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
CPK Increased
|
0.00%
0/118 • 48 weeks
|
5.9%
2/34 • Number of events 2 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Creatinine Increased
|
0.00%
0/118 • 48 weeks
|
5.9%
2/34 • Number of events 2 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/118 • 48 weeks
|
23.5%
8/34 • Number of events 8 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Gastrointestinal disorders
Gastrointestinal Disorders - other, bowel urgency
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Gastrointestinal disorders
Gastrointestinal Disorders - other, large bowel movement
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Glucose Intolerance
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/118 • 48 weeks
|
32.4%
11/34 • Number of events 11 • 48 weeks
|
5.3%
1/19 • Number of events 1 • 48 weeks
|
|
Metabolism and nutrition disorders
Hyperlipidemia
|
0.00%
0/118 • 48 weeks
|
32.4%
11/34 • Number of events 11 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
0.00%
0/118 • 48 weeks
|
32.4%
11/34 • Number of events 11 • 48 weeks
|
5.3%
1/19 • Number of events 1 • 48 weeks
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Hypokalemia
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Hyponatremia
|
0.00%
0/118 • 48 weeks
|
11.8%
4/34 • Number of events 4 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/118 • 48 weeks
|
5.9%
2/34 • Number of events 2 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Lipase Increased
|
0.00%
0/118 • 48 weeks
|
8.8%
3/34 • Number of events 3 • 48 weeks
|
5.3%
1/19 • Number of events 1 • 48 weeks
|
|
General disorders
Localized Edema
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/118 • 48 weeks
|
8.8%
3/34 • Number of events 3 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/118 • 48 weeks
|
17.6%
6/34 • Number of events 6 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Nervous system disorders
Neuralgia
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Platelet Count Decreased
|
0.00%
0/118 • 48 weeks
|
5.9%
2/34 • Number of events 2 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Skin and subcutaneous tissue disorders
Rash Maculo-Papular
|
0.00%
0/118 • 48 weeks
|
5.9%
2/34 • Number of events 2 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Investigations
Serum Amylase Increased
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/118 • 48 weeks
|
2.9%
1/34 • Number of events 1 • 48 weeks
|
0.00%
0/19 • 48 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place