NCT00532883

Brief Summary

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells. The purpose of this study is to evaluate the safety and effectiveness of hydroxyurea and magnesium pidolate, alone and combined, at reducing red blood cell density and the frequency of pain episodes in people with HbSC.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
44

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jan 2007

Geographic Reach
1 country

19 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2007

Completed
9 months until next milestone

First Submitted

Initial submission to the registry

September 20, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 21, 2007

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2009

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2009

Completed
9 months until next milestone

Results Posted

Study results publicly available

May 11, 2010

Completed
Last Updated

January 18, 2013

Status Verified

January 1, 2010

Enrollment Period

2.2 years

First QC Date

September 20, 2007

Results QC Date

April 1, 2010

Last Update Submit

January 14, 2013

Conditions

Keywords

Sickle Cell DiseaseVaso-occlusive EventPainful CrisesAcute Chest Syndrome

Outcome Measures

Primary Outcomes (1)

  • Distribution of the Density of Hemoglobin SC Red Cells

    An individuals' percentage of red blood cells with density greater than 41 g/dL as measured by Advia.

    measured 2 months after initiation of treatment

Study Arms (4)

Placebo Pills and Placebo Liquid

PLACEBO COMPARATOR
Other: Placebo Pills and Placebo Liquid

Hydroxyurea Pills and Placebo Liquid

ACTIVE COMPARATOR
Drug: Hydroxyurea

Placebo Pills and Magnesium Pidolate Liquid

ACTIVE COMPARATOR
Drug: Magnesium Pidolate

Hydroxyurea Pills and Magnesium Pidolate Liquid

ACTIVE COMPARATOR
Drug: HydroxyureaDrug: Magnesium Pidolate

Interventions

HU capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months)

Hydroxyurea Pills and Magnesium Pidolate LiquidHydroxyurea Pills and Placebo Liquid

HU/Placebo capsules (20 mg/kg/day for 11 months) Mg liquid (0.6 mEq/kg/day for 11 months)

Hydroxyurea Pills and Magnesium Pidolate LiquidPlacebo Pills and Magnesium Pidolate Liquid

HU/Placebo capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months)

Placebo Pills and Placebo Liquid

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of HbSC disease
  • Hemoglobin level between 8 and 12.5 g/dL
  • At least one vaso-occlusive event (e.g., pain, acute chest syndrome) in the 12 months prior to study entry. An episode of pain is defined as the occurrence of pain in the extremities, back, abdomen, chest, or head that lasts at least 2 hours; requires a visit to a hospital, emergency room, clinic, or provider's office; and is not explained except by SCD. Acute chest syndrome is defined as a new pulmonary infiltrate on a chest x-ray associated with a fever (greater than 38.5° C), tachypnea, wheezing, cough, or chest pain.
  • Regular compliance with comprehensive care
  • In a steady disease state and not experiencing an acute complication of SCD (i.e., no hospitalization, pain event, or episode of acute chest syndrome within the 1 month prior to study entry)

You may not qualify if:

  • Previous transfusion with remaining hemoglobin A greater than 10%
  • Previous treatment with hydroxyurea within the last 3 months
  • Previous treatment with magnesium within the 3 months prior to study entry (including vitamins containing magnesium)
  • Poor compliance with previous treatment regimens
  • Liver dysfunction (SGPT greater than twice the upper limit of normal) within the 1 month prior to study entry
  • Kidney dysfunction (creatinine greater than or equal to 1.0 mg/dL for participants less than 18 years of age; greater than or equal to 1.2 mg/dL for participants 18 years of age or older) within the 1 month prior to study entry
  • Pregnant
  • Ten or more hospital admissions for pain in the 12 months prior to study entry
  • Daily use of narcotics
  • Treatment with any investigational drug in the 3 months prior to study entry
  • Less than 3% red blood cells with density greater than 41 g/dL (as measured by the ADVIA 120 system)
  • Positive HIV test
  • Other long-term illness or disorder other than SCD that could adversely affect performance in the study (e.g., tuberculosis)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Children's Hospital and Research Center

Oakland, California, 94609-1809, United States

Location

University of California Davis

Sacramento, California, 95817, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342-1600, United States

Location

University of Louisville

Louisville, Kentucky, 40202, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02118, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Montefiore Medical Center

The Bronx, New York, 10463, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

University of Oklahoma

Oklahoma City, Oklahoma, 73104, United States

Location

Saint Christopher's Hospital

Philadelphia, Pennsylvania, 19134-1095, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19444, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Children's Medical Center

Dallas, Texas, 75235, United States

Location

Related Publications (1)

  • Wang W, Brugnara C, Snyder C, Wynn L, Rogers Z, Kalinyak K, Brown C, Qureshi A, Bigelow C, Neumayr L, Smith-Whitley K, Chui DH, Delahunty M, Woolson R, Steinberg M, Telen M, Kesler K. The effects of hydroxycarbamide and magnesium on haemoglobin SC disease: results of the multi-centre CHAMPS trial. Br J Haematol. 2011 Mar;152(6):771-6. doi: 10.1111/j.1365-2141.2010.08523.x. Epub 2011 Jan 31.

MeSH Terms

Conditions

Hemoglobin SC DiseaseAnemia, Sickle CellVaso-Occlusive CrisesAcute Chest Syndrome

Interventions

HydroxyureaPyrrolidonecarboxylic Acid

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLung DiseasesRespiratory Tract DiseasesRespiration Disorders

Intervention Hierarchy (Ancestors)

UreaAmidesOrganic ChemicalsPyrrolidinonesPyrrolidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsGlutamatesAmino Acids, AcidicAmino AcidsAmino Acids, Peptides, and ProteinsImino AcidsAmino Acids, Cyclic

Limitations and Caveats

This study was stopped early due to slow enrollment. It should therefore be viewed as a pilot study.

Results Point of Contact

Title
Harvey Luksenburg
Organization
NHLBI

Study Officials

  • Winfred C. Wang, MD

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 20, 2007

First Posted

September 21, 2007

Study Start

January 1, 2007

Primary Completion

March 1, 2009

Study Completion

August 1, 2009

Last Updated

January 18, 2013

Results First Posted

May 11, 2010

Record last verified: 2010-01

Locations