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

Withdrawn

• Conditions: Scleroderma

• Registration Number: NCT01639573
• Lead Sponsor: Children's Hospital Los Angeles

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 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

Study Timeline

• Study Start: 2011-04
• Study First Submitted: 2012-06-18
• Study First Submitted QC: 2012-07-10
• Study First Posted: 2012-07-12
• Primary Completion: 2018-08-02
• Study Completion: 2018-09-02
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-11
• Last Update Posted: 2023-09-13

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Campath	The site will follow patients for 6 months post adverse event.	Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Primary outcome	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.