- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01639573
Campath-1h Phase I/II Pilot Trial as Immunoablative Therapy for Refractory Systemic Sclerosis (CAMPATH-1H)
Campath-1h as Immunoablative Therapy for Children and Adolescents With Treatment Refractory Systemic Sclerosis
Study Overview
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 Type
Contacts and Locations
Study Locations
-
-
California
-
Los Angeles, California, United States, 90027
- Childrens Hospitla Los Angeles
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
4.2 Inclusion/Exclusion criteria 4.2.1 Inclusion criteria
- 8 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
OR
o 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
- 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)
4.2.2 Exclusion Criteria
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
- End-stage renal disease (GFR<50 ml/min/1.73 m2 or creatinine . 2 mg/dl; estimated CrCl < 40 mL/min or active, untreated dcSSc renal crisis at time of enrollment
- Active hepatitis (ALT, AST, or bilirubin > 2x ULN)
- Active gastric antral vascular ectasia (GAVE, "watermelon stomach")
- 2 mg/kg/day prednisone or equivalent within 30 days of treatment
- Unwilling or unable to discontinue DMARDs for treatment of dcSSc
- Co-morbid illnesses with an estimated median life expectancy < 5 Years
- Active uncontrolled infection
- Positive serology for hepatitis B or C, HIV
- ANC < 1500 cells/µL, platelets < 120,000 cells/µL, Hct < 27% or Hgb < 9.0 g/dL
- Malignancy within the previous 2 years, excluding treated skin cancer
- Myelodysplasia
- Uncontrolled hypertension
- History of hypersensitivity to murine or E. coli proteins
- Pregnancy or unwilling to use contraceptive methods for at least 15 months
- Steroid dependence: > 2mg/kg/day, prednisone or equivalent within 30 days prior to treatment
- History of substance abuse within the last 5 years
- History or presence of 2nd autoimmune disease requiring immunosuppressive therapy that has a substantial risk of recurrence
- Demonstrated lack of compliance with prior medical care
- Lack of rehabilitation potential
4.3 SSc patients, who fulfill the inclusion criteria, will then be assessed for residual thymic function since our previous study of pediatric dcSSc patients demonstrated that the dcSSc patients had decreased thymic function as compared to age matched controls as measured by the proportion of naïve CD4+ T lymphocytes (CD4+, CD31+), recent thymic emigrants (RTE). (5) Patients with less than 40% RTE will be excluded because of concerns about their ability to reconstitute their immune system after immune ablation.
4.4 dcSSc patients, who fulfill the screening criteria, will be consented for entry into the clinical trial.
Study Plan
How is the study designed?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
2 years
|
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
|
The site will follow patients for 6 months post adverse event.
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Katherine Marzan, MD, Children's Hospital Los Angeles
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CCI-11-00077
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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