NCT01639573

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

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

• Status: Withdrawn
• Phase: —
• Study type: Observational
• Enrollment: 0 (actual)
• Sponsor: Children's Hospital Los Angeles · Academic / Other
• Sex: All
• Age: 8 Years – 18 Years
• Healthy volunteers: Not accepted

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 description

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

Conditions

• Scleroderma

Interventions

Type	Name	Description
DRUG	Campath	Pediatric patients with dcSSc are eligible for the clinical trial if they fulfill the inclusion and exclusion criteria of the trial. The inclusion and exclusion criteria are based upon those of the SCOT trial for adult dcSSc patients, which is the Phase 3 clinical trial in the United States comparing autologous HSCT to monthly high dose cyclophosphamide (CY) alone.

Timeline

• Start date: 2011-04-01
• Primary completion: 2018-08-02
• Completion: 2018-09-02
• First posted: 2012-07-12
• Last updated: 2023-09-13