Clinical Phase I/II Pilot Trial Will Study New Therapy for Systemic Sclerosis

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SSc study

SSc researchChildren’s Hospital Los Angeles is recruiting Refractory Systemic Sclerosis Patients between 8 to 18 years of age for a Phase I/II pilot trial of the drug Campath-1H ( identifier: NCT01639573) to address whether prolonged administration of the drug can result in immunoablation. Campath-1H is a trade name for a humanized monoclonal that binds to the surface of mature lymphocytes, inducing its cell death. The trial is entitled “Campath-1h as Immunoablative Therapy for Children and Adolescents with Treatment Refractory Systemic Sclerosis” and is led by Andreas O. Reiff.

Systemic sclerosis (SSc), otherwise known as systemic scleroderma, is a rare disorder characterized by immunological abnormalities typical of autoimmunity, vessel vasculopathy, inflammation and fibroblast dysfunction manifested as skin and internal organ fibrosis. SSc has unknown causes, poor prognosis and rapid progression. Immune suppressive drugs are used to treat patients, but no therapy has been shown to reverse the disease. Other trials have shown that Hematopoietic stem cell transplantation (HSCT) reverse skin fibrosis, stabilizing the function of vital organs and improving quality of life. Immunosuppression is one of the beneficial effects of HSCT, however, HSCT is a complicated procedure, requiring harvesting of marrow or stem cells from related siblings or matched unrelated donors that involves risks. In addition, it requires the use of cytotoxic agents with or without body radiation, and it must be followed by post-transplant immunosuppression in order to prevent graft-versus-host disease (GVHD).

This trial seeks to demonstrate that prolonged administration of Campath-1H without prior marrow or stem cell harvesting can result in immunoablation similar to that achieved by HSCT in children and adolescents with severe treatment refractory systemic sclerosis (SSc).

Over the next two years, the study will elucidate why the extended administration of Campath-1H results in immune ablation in some patients and immunosuppression in others. The number of participants with adverse events will be monitored during and after the completion of the Campath-1H administration as a measure of safety and tolerability of the drug. Both the peak Campath-1H levels as well as the duration of circulating Campath-1H will be determined in this trial.

To learn more about this study, visit