Trial Outcomes & Findings for Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease (NCT NCT01702246)

Last Updated: 2016-12-19

Results Overview

The effect of simvastatin treatment will be assessed by measuring the difference from baseline in the mean frequency (and intensity) of vaso-occlusive pain events, after treatment with simvastatin. Pain rate (proportion of pain days) was defined as the number of days reported with sickle cell disease-related pain divided by the number of daily pain diaries completed.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

24 participants

Primary outcome timeframe

Baseline and 3 months

Results posted on

2016-12-19

Participant Flow

Participant milestones

Participant milestones
Measure	Simvastatin Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months
Overall Study STARTED	24
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Simvastatin Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months
Overall Study Lost to Follow-up	2
Overall Study Withdrawal by Subject	2
Overall Study Physician Decision	1

Baseline Characteristics

Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Simvastatin n=19 Participants Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months
Age, Categorical <=18 years	11 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	18.5 years STANDARD_DEVIATION 6.8 • n=5 Participants
Gender Female	13 Participants n=5 Participants
Gender Male	6 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 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	18 Participants n=5 Participants
Race (NIH/OMB) White	1 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
Receiving hydroxyurea (HU) therapy	10 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 3 months

Outcome measures

Outcome measures
Measure	Baseline n=19 Participants prior to treatment with simvastatin	Simvastatin n=19 Participants after treatment with simvastatin: 40 mg, orally, once daily for 3 months
Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin	0.2365 proportion of pain days Standard Deviation 0.21	0.1311 proportion of pain days Standard Deviation 0.15

SECONDARY outcome

Timeframe: Baseline and 3 months

Mean difference in plasma high sensitivity C-reactive protein level, before and after treatment with simvastatin

Outcome measures

Outcome measures
Measure	Baseline n=19 Participants prior to treatment with simvastatin	Simvastatin n=19 Participants after treatment with simvastatin: 40 mg, orally, once daily for 3 months
Change in Plasma High Sensitivity C-reactive Protein	7.2 mg/mL Standard Deviation 11	2.989 mg/mL Standard Deviation 4.169

SECONDARY outcome

Timeframe: Baseline and 3 months

Population: change in baseline cholesterol level from baseline, after treatment with simvastatin

Outcome measures

Outcome measures
Measure	Baseline n=19 Participants prior to treatment with simvastatin	Simvastatin n=19 Participants after treatment with simvastatin: 40 mg, orally, once daily for 3 months
Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin	124 mmol/L Standard Deviation 27	101 mmol/L Standard Deviation 17

Adverse Events

Simvastatin

Serious events: 5 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Simvastatin n=19 participants at risk Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months
Blood and lymphatic system disorders sickle cell vaso-occlusive pain crisis	21.1% 4/19 • Safety data were collected on each participant during the treatment period (3 months) and at 1 month follow up
Blood and lymphatic system disorders hyperhemolysis	5.3% 1/19 • Safety data were collected on each participant during the treatment period (3 months) and at 1 month follow up

Other adverse events

Other adverse events
Measure	Simvastatin n=19 participants at risk Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months
Skin and subcutaneous tissue disorders facial rash	5.3% 1/19 • Number of events 1 • Safety data were collected on each participant during the treatment period (3 months) and at 1 month follow up

Additional Information

Dr. Carolyn Hoppe

UCSF Benioff Children's Hospital Oakland

Phone: (510)428-3193

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place