Trial Outcomes & Findings for Evaluation of Oral Alpha-Cyclodextrin for Decreasing Serum Cholesterol (NCT NCT01131299)

NCT ID: NCT01131299

Last Updated: 2016-10-21

Results Overview

Total cholesterol levels were measured at the end of each study arm (a-CD or placebo). a-CD (2g) was taken orally three times a day for 12-14 weeks. Placebo (2 tablets) was taken orally for 12-14 weeks. There was a one-week washout period between each arm.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 103 participants
• Primary outcome timeframe: 24-28 weeks
• Results posted on: 2016-10-21

Participant Flow

The recruitment occurred at the Clinical Center, NIH.

103 screened participants, 75 completed the study, 14 subject withdrawals and 14 subjects who did not meet inclusion criteria

Participant milestones

Measure	Placebo First, Then Alpha Cyclodextrin Randomized subjects will receive placebo 2 tablets orally (three times a day) for 12-14 weeks. After a one-week washout, the subjects will receive alpha cyclodextrin. Alpha cyclodextrin: 2 tablets PO 3 times a day for 12-14 weeks	Alpha Cyclodextrin First, Then Placebo Subjects will receive alpha cyclodextrin: 2 tablets PO 3 times a day for 12-14 weeks. After a one-week washout, the subjects will receive placebo. Subjects will receive placebo 2 tablets orally (three times a day) for 12-14 weeks
Overall Study STARTED	42	44
Overall Study COMPLETED	38	37
Overall Study NOT COMPLETED	4	7

Reasons for withdrawal

Measure	Placebo First, Then Alpha Cyclodextrin Randomized subjects will receive placebo 2 tablets orally (three times a day) for 12-14 weeks. After a one-week washout, the subjects will receive alpha cyclodextrin. Alpha cyclodextrin: 2 tablets PO 3 times a day for 12-14 weeks	Alpha Cyclodextrin First, Then Placebo Subjects will receive alpha cyclodextrin: 2 tablets PO 3 times a day for 12-14 weeks. After a one-week washout, the subjects will receive placebo. Subjects will receive placebo 2 tablets orally (three times a day) for 12-14 weeks
Overall Study Withdrawal by Subject	4	7

Baseline Characteristics

Evaluation of Oral Alpha-Cyclodextrin for Decreasing Serum Cholesterol

Baseline characteristics by cohort

Measure	Alpha Cyclodextrin & Placebo Participants n=103 Participants Randomized subjects receiving alpha cyclodextrin Alpha cyclodextrin: 2 g PO 3 times a day for 12- 14 weeks Randomized subjects receiving placebo comparator Placebo: 2 tablets PO 3 times a day for 12-14 weeks
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	99 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Sex: Female, Male Female	60 Participants n=5 Participants
Sex: Female, Male Male	43 Participants n=5 Participants
Region of Enrollment United States	103 participants n=5 Participants

PRIMARY outcome

Timeframe: 24-28 weeks

Total cholesterol levels were measured at the end of each study arm (a-CD or placebo). a-CD (2g) was taken orally three times a day for 12-14 weeks. Placebo (2 tablets) was taken orally for 12-14 weeks. There was a one-week washout period between each arm.

Outcome measures

Measure	a-CD n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline	Placebo n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline
Total Serum Cholesterol Levels for Alpha-cyclodextrin (a-CD) and Placebo Groups After 12-14 Weeks, Compared to Baseline	180 mg/dL Standard Error 4	180 mg/dL Standard Error 4

SECONDARY outcome

Timeframe: 24-28 weeks

Small LDL particle numbers were measured at the end of each study arm (a-CD or placebo). a-CD (2g) was taken orally three times a day for 12-14 weeks. Placebo (2 tablets) was taken orally for 12-14 weeks. There was a one-week washout period between each arm.

Outcome measures

Measure	a-CD n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline	Placebo n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline
Small LDL Particle Number (by NMR Spectrometry of Lipoproteins) After 12-14 Weeks Intervention, Compared to Baseline for Alpha-cyclodextrin (a-CD) and Placebo Groups After 12-14 Weeks, Compared to Baseline	365 nmol/L Standard Error 35	405 nmol/L Standard Error 38

SECONDARY outcome

Timeframe: 24-28 weeks

Serum glucose levels was measured at the end of each study arm (a-CD or placebo). a-CD (2g) was taken orally three times a day for 12-14 weeks. Placebo (2 tablets) was taken orally for 12-14 weeks. There was a one-week washout period between each arm.

Outcome measures

Measure	a-CD n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline	Placebo n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline
Serum Glucose Levels After 12-14 Weeks Intervention, Compared to Baseline for Alpha-cyclodextrin (a-CD) and Placebo Groups After 12-14 Weeks, Compared to Baseline	87 mg/dL Standard Error 0.7	88 mg/dL Standard Error 0.9

SECONDARY outcome

Timeframe: 24-28 weeks

Population: The LIRI score is a composite of six lipoprotein parameters (VLDL, HDL and LDL, and concentrations of large VLDL, large HDL and small LDL subclasses) measured by NMR spectroscopy, which may be apparent years before the onset of overt hyperglycemia. LIRI scores range from zero, the most insulin sensitive, to 100, the most insulin resistant.

LIRI was measured at the end of each study arm (a-CD or placebo). a-CD (2g) was taken orally three times a day for 12-14 weeks. Placebo (2 tablets) was taken orally for 12-14 weeks. There was a one-week washout period between each arm.

Outcome measures

Measure	a-CD n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline	Placebo n=75 Participants Change in total serum cholesterol after 12-14 weeks intervention, compared to baseline
Lipoprotein Insulin Resistance Index (LIRI) After 12-14 Weeks Intervention, Compared to Baseline for Alpha-cyclodextrin (a-CD) and Placebo Groups After 12-14 Weeks, Compared to Baseline	1.4 percentage Standard Error 0.1	1.6 percentage Standard Error 0.1

Adverse Events

Entire Study Population

Serious events: 2 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Measure	Entire Study Population n=75 participants at risk Randomized subjects receiving active comparator Alpha cyclodextrin: 2g PO 3 times a day for 12-14 weeks Randomized subjects receiving placebo comparator Placebo: 2 tablets PO 3 times a day for 12-14 weeks
Gastrointestinal disorders appendectomy	1.3% 1/75
Social circumstances enrollment in conflicting clinical trial	1.3% 1/75

Other adverse events

Measure	Entire Study Population n=75 participants at risk Randomized subjects receiving active comparator Alpha cyclodextrin: 2g PO 3 times a day for 12-14 weeks Randomized subjects receiving placebo comparator Placebo: 2 tablets PO 3 times a day for 12-14 weeks
Gastrointestinal disorders abdominal pain	2.7% 2/75 • Number of events 2
Gastrointestinal disorders intestinal gas	4.0% 3/75 • Number of events 3
Gastrointestinal disorders nausea	1.3% 1/75 • Number of events 1
Gastrointestinal disorders diarrhea	4.0% 3/75 • Number of events 3
Renal and urinary disorders urinary urgency	1.3% 1/75 • Number of events 1
Gastrointestinal disorders dyspepsia (indigestion)	2.7% 2/75 • Number of events 2
Gastrointestinal disorders abdominal cramps	1.3% 1/75 • Number of events 1
Renal and urinary disorders urinary frequency	1.3% 1/75 • Number of events 1

Additional Information

Dr. Marcelo Amar

NIH NHLBI

Phone: 301-402-0521

Email: mamar@mail.nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place