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A Study of the Safety and Efficacy of Patients With Familial Hypercholesterolaemia Taking Colesevelam as add-on Therapy to Their Existing Medication

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ClinicalTrials.gov Identifier: NCT00655265
Recruitment Status : Completed
First Posted : April 9, 2008
Last Update Posted : February 11, 2014
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
This study is designed to assess whether colesevelam given as third line treatment added to a maximal tolerated and stable dose of a statin and ezetimibe is able to further decrease the level of LDL cholesterol in a safe and efficient manner in difficult to treat Familial Hypercholesterolaemia patients who are not at their target level of LDL cholesterol.

Condition or disease Intervention/treatment Phase
Familial Hypercholesterolemia Drug: Colesevelam hydrochloride film-coated tablets Drug: Placebo Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 86 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 4 Randomised, Double-Blind, Placebo-Controlled, Parallel-Group, MultiCentre Study of Colesevelam as Add-on Therapy in Patients With Familial Hypercholesterolaemia
Study Start Date : August 2007
Actual Primary Completion Date : December 2008
Actual Study Completion Date : October 2009

Arm Intervention/treatment
Experimental: Colesevelam hydrochloride film-coated tablets Drug: Colesevelam hydrochloride film-coated tablets
Other Name: Cholestagel

Placebo Comparator: Placebo Drug: Placebo

Primary Outcome Measures :
  1. Relative reduction in LDL cholesterol [ Time Frame: Between baseline and 6 weeks DB treatment ]

Secondary Outcome Measures :
  1. Relative reduction in HDL, total cholesterol, ApoA1,ApoB,ApoB/ApoA1ratio, triglycerides [ Time Frame: Between Baseline and week 6 and week 12 DB treatment ]
  2. Relative reduction in LDL cholesterol [ Time Frame: Between Baseline and week 12 DB treatment ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients must have a clinical diagnosis of Familial Hypercholesterolaemia defined as EITHER a.Presence of a documented LDL-receptor mutation OR b. History of untreated LDL-cholesterol level above the 95th percentile for sex and age in combination with documentation of at least one of the following: i.Presence of typical tendon xanthomas in the patient or first degree relative. ii. An LDL-cholesterol level above the 95th percentile for age and sex in a first degree relative. iii. Proven coronary artery disease in the patient or in a first degree relative under the age of 60.
  • Patients must have provided and undergone lifestyle changes for more than 6 months at the time of Screening
  • Patients must have been treated for at least 3 consecutive months preceding the Screening visit with a lipid lowering treatment regimen consisting of maximal tolerated combination of a statin with ezetimibe and are still above their target for LDL cholesterol being 2.5 mmol/L (100 mg/dL)
  • Patients must be committed to following the protocol requirements as evidence by written informed consent
  • Patients should be comfortable with swallowing 3 placebo tablets

Exclusion Criteria:

  • Patients with a known allergy to any of the components used in colesevelam or placebo or any other medications like statin or ezetimibe required for participation in this study
  • Patients with a bowel or biliary obstruction
  • Patients with secondary causes of hypercholesterolaemia, e.g., dysproteinaemia, hypothyroidism, nephrotic syndrome (defined as proteinuria >2 g/L), obstructive liver disease, other pharmacological therapies, alcoholism
  • Patients with triglyceride level of > 3.4 mmol/L
  • Patients with dysphagia, swallowing disorders, severe gastrointestinal motility disorders, inflammatory bowel disease, or major gastrointestinal tract surgery
  • Patients have undergone LDL-apheresis within one year prior to the screening visit and/or need to undergo LDL-apheresis
  • Patients with active liver disease or unexplained persistent elevations in transaminases
  • Patients on fenofibrates or on concomitant cholestyramine as this will affect the area under the curve (AUC) of ezetimibe
  • Patients with poorly-controlled diabetes (i.e., HbA1c>9% at Screening)
  • Patients with clinically significant (CS) abnormal haematology, renal, or other laboratory parameters that could be the result of an underlying malignancy or systemic infection as judged by the investigator
  • Patients with a heart transplant, concurrent congestive heart failure (NYHA Class 3 or 4), life-threatening ventricular arrhythmias, unstable angina, recent myocardial infarction within the past 6 months prior to screening, or patients undergoing haemodialysis, or with active disease who may not be healthy enough to successfully complete all protocol requirements
  • Fertile women who are pregnant, nursing or using either no or an inadequate form of contraception taking into account the recommendations for adequate intake of oral contraceptives as outlined in concomitant medication section
  • Patients with a recent history of alcoholism or drug abuse, or sever emotional behavioural or psychiatric problems who may not be able to adequately comply with the requirements of the study or who may be unable to consent
  • Patients receiving experimental medications or participating in another study using an experimental drug or procedure within 30 days prior to signing informed consent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00655265

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Service d'Endocrinologie, Metabolisme Prevention des Maladies Cardio-Vasculaires
Paris Cedex, France
Marburg, Germany
Academic Medical Centre
Amsterdam, Netherlands
University Medical Centre
Nijmegen, Netherlands
University Medical Centre
Utrecht, Netherlands
TweeSteden Ziekenhius
Waalwijk, Netherlands
Centre for Metabolism and Endocrinology
Huddinge, Sweden
United Kingdom
Department of Medicine, University of Manchester
Manchester, United Kingdom
Sponsors and Collaborators
Genzyme, a Sanofi Company
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Study Director: Medical Monitor Genzyme, a Sanofi Company
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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00655265    
Other Study ID Numbers: CHOL00107
EudraCT 2007-000582-37 ( Registry Identifier: EudraCT )
First Posted: April 9, 2008    Key Record Dates
Last Update Posted: February 11, 2014
Last Verified: February 2014
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Colesevelam Hydrochloride
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents