The Statistical and Clinical Coordinating Center (SACCC)

The Statistical and Clinical Coordinating Center (SACCC) at Rho Federal Systems Division, Inc provides operational support, statistical analysis, data management, and regulatory support for the Autoimmune Disease Clinical Trials sponsored by the Division of Allergy, Immunology, and Transplant (DAIT), within the National Institute of Allergy and Infectious Disease (NIAID), within the National Institutes of Health (NIH), an agency of the Department of Health and Human Services (DHHS).

• Operational Support activities include:
  • Facilitating communication between study sites, study collaborators, and the NIH
  • Supporting the study's conduct, logistics, and compliance
  • Overseeing the collection and maintenance of study documentation
  • Developing study documents, such as the protocol and Manual of Operations
  • Preparing reports for the NIH, the Data & Safety Monitoring Board (DSMB), and the Food and Drug Administration (FDA)
• Statistical Analysis activities include:
  • Participating as collaborative scientists in all the study's stages, from concept development to study design to manuscript development
  • Providing routine analyses of study progress
  • Conducting interim analyses in support of the DSMB's activities
  • Conducting final analyses and preparing final study reports
• Data Management activities include:
  • Developing Case Report Forms (CRFs) for the accurate and efficient collection of study data
  • Developing, maintaining, and validating study databases
• Regulatory Support activities include:
  • Ensuring that all regulatory documentation is properly collected, reviewed, and maintained
  • Assisting in the development and submission of Investigational New Drug applications and other FDA reports and applications
  • Monitoring sites to ensure compliance with the protocol and with Good Clinical Practice (GCP), NIH, and FDA requirements
  • Facilitating safety communication between sites and the NIH

Autoimmune Diseases

Autoimmune diseases are caused by the misdirection of an immune response toward the body's own tissues. The principal role of the immune system is to defend against infection. The body has safeguards to prevent the immune system from attacking its own tissues, but when these safeguards are breached, an autoimmune disease can result.

Collectively, autoimmune diseases afflict between 14 and 22 million Americans. Medical science has identified more than 80 clinically distinct autoimmune diseases, including systemic lupus erythematosus, type 1 diabetes, severe lupus nephritis, rheumatoid arthritis, Sjogren's syndrome, scleroderma, and multiple sclerosis. Those suffering with autoimmune diseases often endure loss of function, disability, hospitalizations, outpatient visits, decreased productivity, and impaired quality of life.

Current Autoimmune Projects Supported by the SACCC

As the Statistical and Clinical Coordinating Center at Rho, Inc supports the two components of the Autoimmune Disease Clinical Trials, all studies from both the Autoimmunity Centers of Excellence and the Stem Cell Transplantation Consortium are coordinated from the SACCC.

The AUTOIMMUNITY CENTERS of EXCELLENCE (ACE)

The Autoimmunity Centers of Excellence (ACEs) program was created to encourage and enable collaborative research – across scientific disciplines, across medical specialties, and between basic and clinical scientists – in the search for effective treatments for autoimmune diseases. The nine ACEs support an integrated basic and clinical research program focusing on treatment or prevention approaches that induce immune tolerance or modulate the immune system. The program brings together basic scientists and clinicians from leading research institutions to evaluate the safety and efficacy of treatment strategies for autoimmune diseases. ACEs investigators also explore the immune mechanisms underlying the agents evaluated in these trials – research that commonly is not included in other clinical trial programs. The ACEs program will enhance interactions between scientists and clinicians in order to accelerate the translation of research findings into medical applications.

The ACE program is a cooperative effort of NIAID, the NIH Office of Research on Women's Health, and the National Institute of Diabetes and Digestive and Kidney Diseases, all of which are components of the NIH, an agency of the Department of Health and Human Services.

Current ACE studies include:

• APV01: Use of Infliximab for the Treatment of Pemphigus Vulgaris
• ARA02: Lovastatin for the Treatment of Mildly Active Rheumatoid Arthritis
• ASJ01: Anti-CD20 Antibody Therapy for Sjogren's Syndrome

APV01: Use of Infliximab for the Treatment of Pemphigus Vulgaris

Pemphigus Vulgaris (PV) is a rare autoimmune skin disorder that causes blistering of the skin and mucous membranes. Infliximab is a man made antibody directed against a chemical messenger that activates an immune response and has been used to treat other autoimmune diseases (rheumatoid arthritis, Crohn's disease). APV01 is a randomized, placebo controlled, double blind study that is designed to evaluate the safety and efficacy of infliximab (in combination with prednisone) for the treatment of adults with PV. Participants will be seen by study clinicians a total of 9 times and will be followed for 26 weeks. More information on the APV01 trial...

ARA02: Lovastatin for the Treatment of Mildly Active Rheumatoid Arthritis

Rheumatoid Arthritis (RA) is characterized by persistent inflammation of peripheral joints, causing pain, stiffness, swelling, warmth, and joint damage resulting in deformity and loss of function. Current RA therapy with antirheumatic drugs is expensive and long-term safety remains unknown. Lovastatin is currently used to lower levels of cholesterol and other fats in the blood. ARA02 is a randomized, placebo controlled, double blind study designed to study the safety and efficacy of lovastatin in controlling inflammation in individuals who have mildly active RA despite treatment. Participants will see study clinicans 4 times over the 12 week study period. More information on the ARA02 trial...

ASJ01: Anti-CD20 Antibody Therapy for Sjogren's Syndrome

Sjogren's Syndrome (SS), which attacks and destroys the glands that produce tears and saliva, is the second most common autoimmune disease. Traditional treatment strategies are aimed at addressing the dryness symptoms because there are no established disease modifying treatments currently available. Rituximab is a laboratory made antibody that targets a specific antigen on the surface of B cells (an active participant in immune responses) and is currently approved to treat B cell non-Hodgkin's lymphoma and has been shown to relieve RA symptoms in a small study. Since Sjogren's is associated with the development of B cell-related cancers, rituximab provides a possible option for alleviating Sjogren's symptoms. ASJ01 is a one arm study designed to evaluate the safety of Rituximab in Sjogren's patients. Participants will receive two injections of Rituximab two weeks apart and then will be seen 6 times for follow-up visits within the following year. More information on the ASJ01 trial...

The STEM CELL TRANSPLANTATION CONSORTIUM

The Stem Cell Transplantation Consortium is researching a therapy aimed at "resetting" an autoimmune patient's immune system so that it only attacks foreign entities, thus stopping the progression of the autoimmune disease. The therapy is high dose immunosuppressive therapy (HDIT) followed by a autologous hematopoeitic stem cell transplant (HSCT). The high dose immunosuppressive therapy suppresses the patient's malfunctioning immune system. While this stops the immune system from attacking the patient's own tissues, this also leaves the immune system unable to protect the patient from foreign entities (infections). The second step in the therapy involves "rescue" by a specific type of stem cell transplant. The patient's own stem cells are collected. The hematopoietic stem cells are then selected as they are the immature blood cells that can develop into all of the different immune cells needed for a properly functioning immune system. Those selected immature cells are then reinfused into the patient, where they will hopefully mature into an immune system that only attacks foreign entities, thus keeping the patient safe from his/her own immune system.

Current Stem Cell Transplantation Consortium studies include:

• HALT MS: High Dose Immunosuppression and AutoLogous Transplantation for Multiple Sclerosis
• LIST: Lupus Immunosuppressive/Immunomodulatory Therapy or Stem Cell Transplant
• SCOT: Scleroderma Cyclophosphamide or Transplantation

HALT MS: High Dose Immunosuppression and AutoLogous Transplantation for Multiple Sclerosis

Multiple Sclerosis (MS) is a chronic autoimmune disease of the central nervous system in which myelin (the protective coat surrounding nerve cells) is damaged or destroyed by autoimmune T cells and macrophages which leads to an eventual loss of neurologic function. The HALT MS study intends to confirm results from a pilot study and offer a therapeutic option to patients with early MS and a poor prognosis. All study participants of this one-arm study will be given high dose chemotherapy followed by an autologous transplant of hematopoietic stem cells. Study participants will be seen by study clinicians eight times over a sixty month post-transplant follow-up period. More information on the HALT MS trial...

LIST: Lupus Immunosuppressive/Immunomodulatory Therapy or Stem Cell Transplant

Systemic lupus erythematosus (SLE) is a chronic, multisystem autoimmune disease in which the body's internal system of defense attacks its own tissues (skin, joints, lungs, heart, brain, intestine, and kidneys). While the disease may go through a cycle of remissions and relapses, lupus is associated with progressive morbidity. Currently, there is not one accepted course of treatment for SLE patients; clinicians prescribe an aggressive regimen of one or more immunosuppressive/immunomodulatory treatments. Unfortunately, such a treatment course is not effective in all subjects and even if it is effective it is also associated with drug-induced morbidities. LIST will compare a control arm of currently available therapies and a treatment arm of high dose immunosuppressive therapy followed by an autologous transplant of hematopoietic stem cells. Study participants will be seen by study clinicians every 4 weeks for 30 months.More information on the LIST trial...

SCOT: Scleroderma Cyclophosphamide or Transplantation

Scleroderma is a disabling autoimmune disease that is characterized by both diffuse inflammation and subsequent fibrosis of skin and internal organs. Immune based therapies have been shown to be beneficial but no single treatment has been effective in preventing disease progression or reversing fibrosis. SCOT will compare two Scleroderma treatments in an effort to treat a subset of scleroderma patients with a high death rate. One therapy is thought to "reset" the immune system, by initially suppressing the patient's immune system via high dose immunosuppressive therapy and then rescuing the immune system via an autologous hematopoietic stem cell transplant. The second therapy is high dose pulse IV cyclophosphamide, an immunosuppressive drug which has anecdotal support for its ability to slow down scleroderma induced lung disease. Study participants will be seen by study clinicians every 4 weeks for the first 46 weeks and then at selected monthly timepoints for the remainder of their 44 month follow-up period.More information on the SCOT trial...

Please contact the Statistical and Clinical Coordinating Center for questions or comments.
Last modified: 1June2006