Clinical Trial: Campath-1h Phase I/II Pilot Trial as Immunoablative Therapy for Refractory Systemic Sclerosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Campath-1h as Immunoablative Therapy for Children and Adolescents With Treatment Refractory Systemic Sclerosis

Brief Summary: This phase I/II pilot 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 hematopoietic stem cell transplantation (HSCT) from either bone marrow or peripheral blood stem cell sources in children and adolescents with severe treatment refractory systemic sclerosis (SSc).

Detailed Summary:

Patients, 8 to18 years of age, will be included if they have a proven diagnosis of diffuse cutaneous or systemic SSc as defined by the ACR criteria with evidence of active inflammatory disease Plus at least 1 of the following:SSc-related pulmonary disease with forced vital capacity (FVC) or hemoglobin-adjusted DLCO < 70% and evidence of alveolitis by high-resolution CT scan or bronchoalveolar lavage.

OR:History of SSc-related renal crisis or disease, not active at the time of screening

OR:Moderate to severe upper and/or lower gastrointestinal involvement

AND:Unacceptable toxicity or steroid dependence > 0.3 mg/kg/d,

OR:Failure to respond to, or unacceptable toxicity of MTX > 1mg/kg in combination with cyclosporine or azathioprine or cyclophosphamide 2 kg/d or Rituximab 375 mg/m2 x 4 doses or Imatinib 800 mg/

OR:Disease recurrence after tapering medication above

Current Primary Outcome:

• Primary outcome [ Time Frame: 2 years ]
  To determine why the extended administration of Campath-1H results in immune ablation in some patients and immunosuppression in others, Number of Participants with Adverse Events as a Measure of Safety and Tolerability Campath-1H antibody levels during and after the completion of the Campath administration. (47) Thus, both the peak Campath-1H levels as well as the duration of circulating Campath will be determined.
• Campath [ Time Frame: The site will follow patients for 6 months post adverse event. ]
  Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Children's Hospital Los Angeles

Dates:
Date Received: June 18, 2012
Date Started: April 2011
Date Completion: April 2018
Last Updated: August 5, 2016
Last Verified: August 2016