Trial Outcomes & Findings for Smith-Lemli-Opitz Syndrome and Cholic Acid (NCT NCT03720990)
NCT ID: NCT03720990
Last Updated: 2024-04-30
Results Overview
Plasma cholesterol will be measured. Response to cholic acid treatment will consist of a change in plasma cholesterol.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
12 participants
Primary outcome timeframe
8 weeks
Results posted on
2024-04-30
Participant Flow
Recruitment conducted at Children's Hospital and Medical Center (CHMC), Omaha, NE and Childrens Hospital Colorado (CHCO).
Participant milestones
| Measure |
Cholic Acid
Participants will be treated with cholic acid 10 mg/kg body weight.
Cholic Acid: Participants will be treated with cholic acid for 8 weeks
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Smith-Lemli-Opitz Syndrome and Cholic Acid
Baseline characteristics by cohort
| Measure |
Cholic Acid
n=12 plasma
Participants will be treated with cholic acid 10 mg/kg body weight.
Cholic Acid: Participants will be treated with cholic acid for 8 weeks
|
|---|---|
|
Age, Categorical
<=18 years
|
10 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
9.5 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
12 Participants
n=99 Participants
|
|
Cholesterol
|
12 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 8 weeksPlasma cholesterol will be measured. Response to cholic acid treatment will consist of a change in plasma cholesterol.
Outcome measures
| Measure |
Cholic Acid
n=12 Participants
Participants will be treated with cholic acid 10 mg/kg body weight.
Cholic Acid: Participants will be treated with cholic acid for 8 weeks
|
|---|---|
|
Change in Plasma Cholesterol
|
37.8 change (%) compared to baseline
Standard Deviation 22.4
|
Adverse Events
Cholic Acid
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place