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

NCT01702246 | Completed Phase 1 Results DMC

• 1 other identifier: DDCF-ICRA-2011
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether simvastatin is effective in reducing the frequency and intensity of vaso-occlusive pain episodes in patients with sickle cell disease.

Conditions

Sickle Cell Disease

Keywords

sickle cell disease; statins; vaso-occlusive pain; inflammation; biomarkers

Outcome Measures

Primary Outcomes (1)

• Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin

  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.

  Baseline and 3 months

Secondary Outcomes (2)

• Change in Plasma High Sensitivity C-reactive Protein

  Baseline and 3 months

• Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin

  Baseline and 3 months

Study Arms (1)

simvastatin

EXPERIMENTAL

Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months

Drug: Simvastatin

Interventions

Simvastatin DRUG

40 mg, orally, once daily for 3 months

Also known as: Zocor

simvastatin

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Sickle cell disease (HbSS or S/β0 thalassemia)
• ≥ 3 vaso-occlusive pain episodes in the year prior to enrollment
• Age ≥ 10 years
• Weight \> 30 kg

You may not qualify if:

• Creatine kinase (CK) \> UNL
• Total cholesterol \< 90 mg/dL, or triglycerides \<30mg/dL
• Renal dysfunction (Creatinine \> 1.5-fold UNL)
• Hepatic dysfunction (ALT \> 2-fold UNL)
• Treatment with drugs having known metabolic interactions with statins within the past 30 days
• Vaso-occlusive pain requiring hospitalization within past 30 days
• Red blood cell transfusion within the past 30 days
• Pregnancy/lactation
• Musculoskeletal disorder associated with an elevated CK level
• Past or present history of substance abuse (alcohol, cocaine, amphetamines, heroin)
• Chronic pain caused by avascular necrosis of the bone (AVN) or leg ulcers, and pain due to trauma or causes other than SCD.
• Major cognitive or neurological impairments that may hamper the ability to use the smartphone or complete the electronic pain diary in this study

Sponsors & Collaborators

• UCSF Benioff Children's Hospital Oakland: lead
• University of California, Los Angeles: collaborator

Study Sites (1)

Children's Hospital and Research Center Oakland

Oakland, California, 94609, United States

Location

Related Publications (1)

• Hoppe C, Kuypers F, Larkin S, Hagar W, Vichinsky E, Styles L. A pilot study of the short-term use of simvastatin in sickle cell disease: effects on markers of vascular dysfunction. Br J Haematol. 2011 Jun;153(5):655-63. doi: 10.1111/j.1365-2141.2010.08480.x. Epub 2011 Apr 8.

  PMID: 21477202 BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle Cell; Inflammation

Interventions

Simvastatin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Lovastatin; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Results Point of Contact

• Title: Dr. Carolyn Hoppe
• Organization: UCSF Benioff Children's Hospital Oakland

choppe@mail.cho.org

(510)428-3193

Study Officials

• Carolyn Hoppe, MD

  UCSF Benioff Children's Hospital Oakland

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Hematologist-Oncologist

Study Record Dates

• First Submitted: October 4, 2012
• First Posted: October 8, 2012
• Study Start: February 1, 2012
• Primary Completion: May 1, 2015
• Study Completion: June 1, 2015
• Last Updated: December 19, 2016
• Results First Posted: December 19, 2016

Record last verified: 2016-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)