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Trial record 1 of 1 for:    10597756 [PUBMED-IDS]
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Simvastatin (Zocor) Therapy in Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT00508027
Recruitment Status : Completed
First Posted : July 27, 2007
Results First Posted : August 16, 2013
Last Update Posted : September 17, 2013
Sponsor:
Collaborators:
Department of Health and Human Services
FDA Office of Orphan Products Development
Information provided by (Responsible Party):
Carolyn Hoppe, Children's Hospital & Research Center Oakland

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Sickle Cell Disease
Intervention: Drug: Simvastatin

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
During the study period (05/2006-09/2010), eligible adult and adolescent SCD subjects followed at the CHRCO Sickle Cell Center were approached about participation in this trial. Subjects were enrolled at “steady-state” (i.e., no acute illness or acute SCD-related complications) during a routine clinic visit.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
There were no significant events following enrollment after inclusion and exclusion criteria were met.

Reporting Groups
  Description
Simvastatin, 3 Escalating Dose Groups

Simvastatin was given in a dose-escalating fashion to 3 sequential dose groups:

dose level 1= 20 mg/day, dose level 2= 40 mg/day, dose level 3= 80 mg/day The number of subjects starting each dose level are new cohorts of subjects.

Determination of clinical safety in the first dose level group was required in order to begin enrollment in the second dose level group and ultimately the third dose group. Enrollment in the third dose level was discontinued early due to newly reported FDA warnings regarding high dose (80mg/day) simvastatin.


Participant Flow for 3 periods

Period 1:   Dose Level 1
    Simvastatin, 3 Escalating Dose Groups
STARTED   20 [1] 
COMPLETED   12 
NOT COMPLETED   8 
Lost to Follow-up                4 
Withdrawal by Subject                2 
Physician Decision                2 
[1] The participants starting each dose period is a new cohort of subjects. 20 subjects started dose 1

Period 2:   Dose Level 2
    Simvastatin, 3 Escalating Dose Groups
STARTED   20 [1] 
COMPLETED   16 
NOT COMPLETED   4 
Lost to Follow-up                2 
Withdrawal by Subject                2 
[1] The participants starting each dose period is a new cohort of subjects. 20 new subjects in dose 2

Period 3:   Dose Level 3
    Simvastatin, 3 Escalating Dose Groups
STARTED   2 [1] 
COMPLETED   2 
NOT COMPLETED   0 
[1] enrollment discontinued due to FDA recommendations regarding high dose (80mg/day) simvastatin



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
42 subjects started the study; of these,12 were withdrawn from the study and 30 completed the study: dose level 1 (n=16); dose level 2 (n=12), dose level 3 (n=2). Only those participants who completed the study were included in the final analyses (n=30).

Reporting Groups
  Description
Simvastatin, Dose Escalation

There are no arms in this study. Simvastatin will be given in a dose-escalating fashion to 3 sequential dosage groups (20 mg/day, 40 mg/day, 80 mg/day).

Simvastatin : Comparison of 3 dosages of simvastatin given in a dose-escalating fashion.

20 mg, 40 mg, or 80 mg PO QD x 21 days followed by a drug taper x 4 days.


Baseline Measures
   Simvastatin, Dose Escalation 
Overall Participants Analyzed 
[Units: Participants]
 30 
Age 
[Units: Years]
Mean (Standard Deviation)
 25  (11.5) 
Gender 
[Units: Participants]
 
Female   9 
Male   21 
Region of Enrollment 
[Units: Participants]
 
United States   30 
Sickle cell genotype 
[Units: Participants]
 
Homozygous Hb S (SS or S/beta0 thalassemia)   20 
Compound heterozygous Hb S and Hb C (SC)   10 


  Outcome Measures

1.  Primary:   Change in Total Cholesterol Level   [ Time Frame: Baseline, 21 days ]

2.  Primary:   Change in Hemoglobin Level   [ Time Frame: Baseline, 21 days ]

3.  Primary:   Change in Serum Creatine Kinase Levels   [ Time Frame: Baseline, 21 days ]

4.  Primary:   Change in Serum Alanine Transaminase (ALT) Levels   [ Time Frame: Baseline, 21 days ]

5.  Primary:   Change in Serum Creatinine Levels   [ Time Frame: Baseline, 21 days ]

6.  Other Pre-specified:   Change in Plasma NOx Levels   [ Time Frame: Baseline, 21 days ]

7.  Other Pre-specified:   Change in Plasma Hs-CRP Levels   [ Time Frame: Baseline, 21 days ]

8.  Other Pre-specified:   Change in Plasma IL-6 Levels   [ Time Frame: Baseline, 21 days ]

9.  Other Pre-specified:   Change in Plasma VEGF Levels   [ Time Frame: Baseline, 21 days ]

10.  Other Pre-specified:   Change in Plasma VCAM1 Levels   [ Time Frame: Baseline, 21 days ]

11.  Other Pre-specified:   Change in Plasma TF Levels   [ Time Frame: Baseline, 21 days ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Carolyn Hoppe, Principal Investigator
Organization: Children's Hospital & Research Center Oakland
phone: (510)428-3193
e-mail: choppe@mail.cho.org


Publications of Results:
Other Publications:


Responsible Party: Carolyn Hoppe, Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier: NCT00508027     History of Changes
Other Study ID Numbers: 1R01FD003080-01A1 ( U.S. FDA Grant/Contract )
First Submitted: July 26, 2007
First Posted: July 27, 2007
Results First Submitted: February 11, 2013
Results First Posted: August 16, 2013
Last Update Posted: September 17, 2013