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

NCT01639573 | Withdrawn DMC

• 1 other identifier: CCI-11-00077
• Study Type: observational
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Scleroderma

Keywords

Patients with Scleroderma

Outcome Measures

Primary Outcomes (2)

• Primary outcome

  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.

  2 years

• Campath

  Number of Participants with Adverse Events as a Measure of Safety and Tolerability

  The site will follow patients for 6 months post adverse event.

Interventions

Campath DRUG

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.

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

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

You may not qualify if:

• to 21 years of age, inclusive
• Diffuse, cutaneous dcSSc as defined by the ACR criteria with evidence of active inflammatory disease.
• Plus at least 1 of the following:
• dcSSc-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
• o History of SSc-related renal crisis or disease, not active at the time of screening
• Moderate to severe upper and/or lower gastrointestinal involvement AND
• Unacceptable toxicity or steroid dependence \> 0.3 mg/kg/d
• Failure to respond to, or unacceptable toxicity of MTX \> 1mg/kg in combination with cyclosporine or azathioprine or cyclophosphamide or Rituximab 375 mg/m2 x 4 doses or Imatinib 800 mg/d or tocilizumab 8 mg/kg for at least 3 doses.
• Disease recurrence after tapering medication above (in #4)
• Pulmonary, cardiac, hepatic, or renal impairment that would limit therapy and compromise survival includes, but is not restricted to, any of the following:
• Severe pulmonary dysfunction: hemoglobin-corrected DLCO \< 45%, DLCO \<4 mL/mmHg/min/L or pO2 \< 70 mm Hg or pCO2, ≥ 45 mm Hg without supplemental O2 sat 92% at rest without supplemental O2
• Significant pulmonary hypertension
• Uncontrolled clinically significant arrhythmias
• NYHA heart failure class IV
• LVEF \< 50% by echo or prior insertion of a pacemaker or cardioverter-defibrillator

Sponsors & Collaborators

• Children's Hospital Los Angeles: lead

MeSH Terms

Conditions

Scleroderma, Diffuse

Interventions

Alemtuzumab

Condition Hierarchy (Ancestors)

Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Katherine Marzan, MD

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Co-Principal Investigator

Study Record Dates

• First Submitted: June 18, 2012
• First Posted: July 12, 2012
• Study Start: April 1, 2011
• Primary Completion: August 2, 2018
• Study Completion: September 2, 2018
• Last Updated: September 13, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share