Trial Outcomes & Findings for Orlistat for the Treatment of Type I Hyperlipoproteinemia (NCT NCT02767531)

Last Updated: 2023-02-08

Results Overview

Fasting blood samples were collected on three consequetive days at the end of each three month period. Mean values of the three days were calculated.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

3 days

Results posted on

2023-02-08

Participant Flow

A randomized,open-label, crossover trial with four periods and two sequences ("orlistat" and "off" for 3 months each) was conducted.

Participant milestones

Participant milestones
Measure	Orlistat Then Off Therapy Randomized to Orlistat for first 3 months, then cross over to no therapy for 3 months, followed by repeat sequence of Orlistat for additional 3 months, and no therapy for next 3 months	Off Orlistat Then Orlistat Randomized to no therapy for first 3 months and then cross over to Orlistat for 3 months and then repeat sequence of no therapy for additional 3 months and then Orlistat for next 3 months
Period 1 STARTED	1	1
Period 1 COMPLETED	1	1
Period 1 NOT COMPLETED	0	0
Period 2 STARTED	1	1
Period 2 COMPLETED	1	1
Period 2 NOT COMPLETED	0	0
Period 3 STARTED	1	1
Period 3 COMPLETED	1	1
Period 3 NOT COMPLETED	0	0
Period 4 STARTED	1	1
Period 4 COMPLETED	1	1
Period 4 NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Orlistat for the Treatment of Type I Hyperlipoproteinemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Baseline Features of Patients Enrolled n=2 Participants 120 mg of Orlistat will be given 3 times to patients weighing greater than 50 kg and patients weighing less than 40 kg will be given 60 mg of Orlistat 3 times a day for 3 months. The patients were randomized to receive 3 months of orlistat or no therapy (off), then crossed over to the other arm, and this sequence was then repeated for an additional 6 months
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race/Ethnicity, Customized Asian	2 Participants n=5 Participants
Triglyceride	1674 mg/dL n=5 Participants

PRIMARY outcome

Timeframe: 3 days

Population: The patients were randomized to receive 3 months of orlistat or no therapy (off), then crossed over to the other arm, and this sequence was then repeated. Because only 2 patients participated, the results are provided per sequence and not per intervention.

Fasting blood samples were collected on three consequetive days at the end of each three month period. Mean values of the three days were calculated.

Outcome measures

Outcome measures
Measure	Orlistat and Then Off Therapy n=1 Participants Randomized to Orlistat for first 3 months, then cross over to no therapy for 3 months, followed by repeat sequence of Orlistat for additional 3 months, and no therapy for next 3 months 120 mg of Orlistat will be given 3 times to patients weighing greater than 50 kg and patients weighing less than 40 kg will be given 60 mg of Orlistat 3 times a day for 3 months.	Off Therapy and Then Orlistat n=1 Participants Randomized to no therapy for first 3 months and then cross over to Orlistat for 3 months and then repeat sequence of no therapy for additional 3 months and then Orlistat for next 3 months
Fasting Serum Triglycerides Period 1	705 mg/dL Interval 656.0 to 730.0	1827 mg/dL Interval 1266.0 to 2320.0
Fasting Serum Triglycerides Period 2	1511 mg/dL Interval 1226.0 to 1750.0	1007 mg/dL Interval 964.0 to 1040.0
Fasting Serum Triglycerides Period 3	758 mg/dL Interval 617.0 to 876.0	3121 mg/dL Interval 2780.0 to 3490.0
Fasting Serum Triglycerides Period 4	1614 mg/dL Interval 1540.0 to 1680.0	1181 mg/dL Interval 1122.0 to 1248.0

Adverse Events

Orlistat

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Off Therapy

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Orlistat n=2 participants at risk Each of the two patients, were randomized to receive Orlistat for 3 months in two periods. Patient 1 took Orlistat 60 mg with the first and second meal and 120 mg with the third meal, whereas Patient 2 took 60 mg of Orlistat 3 times daily with meals.	Off Therapy n=2 participants at risk Each of the two patients, were randomized to "Off therapy" for 3 months in two periods.
Gastrointestinal disorders Bloating	100.0% 2/2 • Number of events 2 • 1 year	0.00% 0/2 • 1 year

Additional Information

Abhimanyu Garg, MD

UT Southwestern Medical Center

Phone: 2146482895

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place