Clinical trial NCT01466725: A Research Study to Assess if CC-930 is Safe in Treating Subjects With Discoid Lupus Erythematosus
Official title: A Phase 2, Pilot, Multicenter, Randomized, Placebo-Controlled Sequential, Ascending Dose Study to Characterize the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Activity of CC-930 in Subjects With Recalcitrant Discoid Lupus Erythematosus (DLE)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Discoid Lupus
Keywords: Lupus
Discoid Lupus
DLE
Cutaneous Lupus
Lead sponsor: Celgene
Sponsor class: INDUSTRY
First posted: 2011-11-08
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to assess if CC-930 is safe and tolerable in treating subjects with Discoid Lupus Erythematosus. Pharmacokinetic and pharmacodynamics will also be evaluated.

Detailed description: Enrollment will occur in sequential, ascending, dose-sequence design, where a higher CC-930 dose level and longer duration of dosing (cohort) will not be initiated until supportive safety profile is demonstrated in the preceding cohort. There will be 4 cohorts as described here;

Cohort 1: 25 mg once daily for 4 weeks Cohort 2: 50 mg once daily for 4 weeks Cohort 3: 100 mg once daily for 6 weeks Cohort 4: 100 mg twice daily for 6 weeks

Eligibility criteria: Inclusion Criteria:

1. Male or female with clinical diagnosis of Discoid Lupus Erythematosus (DLE) aged 18 to 64 years
2. Good health as assessed by Investigator
3. DLE for at least 16 weeks prior to screening and consistent histological findings.
4. Considered a candidate for systemic therapy. May be naïve to systemic therapy or experiencing incomplete or refractory disease on systemic therapy.
5. Cutaneous Lupus Area and Severity Index (CLASI) activity score of at least 10, as determined by investigator.
6. Subjects using hydroxychloroquine, chloroquine, or quinacrine must have documented ophthalmologic exam within 24 weeks of baseline visit.
7. Must meet laboratory criteria for white blood cells, absolute neutrophils, platelets, serum creatinine, aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin and hemoglobin.
8. Subjects, male and female, must agree to strict pregnancy prevention and testing requirements.

Exclusion Criteria:

1. Significant illnesses as determined by physician.
2. History of significant cardiac conditions or interventions within prior 6 months including abnormal electrocardiogram (ECG) findings.
3. Systolic blood pressure \< 95 or \> 150 mm Hg
4. Diastolic blood pressure \> 90 mm Hg.
5. Pregnancy or breast feeding.
6. Other dermatological conditions that would interfere with CLASI Activity Score assessments.
7. History of or active; malignancy, human immunodeficiency virus (HIV), tuberculosis infection, other mycobacterial infection, congenital or acquired immunodeficiency, Hepatitis B and C.
8. Clinically significant abnormality on chest X-ray.
9. Participation in multiple CC-930 cohorts.
10. History of thrombolytic event.
11. Positive tests for lupus anticoagulant, anti-cardiolipin antibodies, antibodies to beta-2 glycoprotein 1 or phosphatidylserine at screening.
12. Positive antineutrophilic cytoplasmic antibody (ANCA) at screening.
13. Diagnosis of SLE.
14. Presence or history of medically significant Systemic Lupus Erythematosis (SLE) or Lupus Erythematosis (LE) comorbidities.
15. History of seizures, chorea or psychosis.
16. Presence or history of persistent proteinuria or urinary cellular casts.
17. Prohibited prior or concomitant medications.

Primary outcomes:
measure: Number of subjects with adverse events
timeFrame: 4 to 6 weeks
Secondary outcomes:
measure: To describe the pharmacokinetics of CC-930 in subjects with recalcitrant DLE.
timeFrame: 18 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: UAB Dermatology
state: Alabama
city: Irvine
country: United States
facility: The Regents of the University of California
state: California
city: Washington
country: United States
facility: Medical Faculty Associates
state: District of Columbia
city: Miami
country: United States
facility: Advanced Pharma, CR, LLC
state: Florida
city: Miami
country: United States
facility: University of Miami - Department of Dermatology
state: Florida
city: Chicago
country: United States
facility: Rush Medical Center
state: Illinois
city: Skokie
country: United States
facility: North Shore University Health System
state: Illinois
city: Springfield
country: United States
facility: SIU School of Medicine
state: Illinois
city: New Orleans
country: United States
facility: Tulane University School of Medicine
state: Louisiana
city: Baltimore
country: United States
facility: John Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston Cancer Center
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota-Department of Dermatology
state: Minnesota
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Columbus
country: United States
facility: Ohio State Univ Medical Center, Division of Dermatology
state: Ohio
city: Providence
country: United States
facility: Rhode Island Hospital University Dermatology, Inc.
state: Rhode Island
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Dallas
country: United States
facility: Dermatology and Research
state: Texas
city: Houston
country: United States
facility: University of Texas Dermatology
state: Texas
city: San Antonio
country: United States
facility: Sun Research Institute
state: Texas
Study officials:
affiliation: Celgene
name: William Smith, MD
role: STUDY_DIRECTOR
References: