Effectiveness of Arginine as a Treatment for Sickle Cell Anemia

Summary

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes. This study will evaluate the effectiveness of the nutritional supplement arginine at improving blood cell function and disease symptoms in people with SCD.

Conditions

• Anemia, Sickle Cell

Interventions

DRUG

Arginine

Depending on the weight of the child or adult, the patients took any where between 4-10 capsules 2 times a day. Patients weighing less than 45 kilograms were on the low dose active (or placebo) so the capsules were smaller. Patients greater than or equal to 45 kgs were on the high dose active or placebo, so these capsules were larger.

DRUG

Placebo

Depending on the weight of the child or adult, the patients took any where between 4-10 capsules 2 times a day. Patients weighing less than 45 kilograms were on the low dose active (or placebo) so the capsules were smaller. Patients greater than or equal to 45 kgs were on the high dose active or placebo, so these capsules were larger.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• UCSF Benioff Children's Hospital Oakland

  lead OTHER

Principal Investigators

• Lillian McMahon, MD · Boston Medical Center

• Rathi Iyer, MD · University of Mississippi Medical Center (Pediatric)

• Carolyn Bigelow, MD · University of Mississippi Medical Center (Adult)

• Lennette Benjamin, MD · Montefiore Medical Center

• Mary Fabry, MD · Albert Einstein College of Medicine

• Thomas Moulton, MD · Children's Hospital of Montefiore

• Kim Smith-Whitley, MD · Children's Hospital of Philadelphia

• Laura DeCastro, MD · Duke University

• Kenneth Ataga, MD · University of North Carolina, Chapel Hill

• Samir K. Ballas, MD · Thomas Jefferson University

• Sal Bertalone, MD · Norton Healthcare

• Carlton Dampier, MD · St. Christopher's Childrens Hospital

• William Mentzer, MD · University of California, San Francisco

• Winfred Wang, MD · St. Jude's Childrens Research Hospital

• Ulrike Reiss, MD · St. Jude Children's Research Hospital

• Cynthia Rutherford, MD · Children's Medical Center Dallas

• Kathryn Hassell, MD · University of Colorado, Denver

• Joan Parkhurst Cain, MD · Children's Hospital of Oklahoma

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

5 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2004-06-30

Primary Completion

2007-09-30

Completion

2008-01-31

Countries

• United States

Study Locations