The Efficacy and Safety of CC-10004 in Chronic Cutaneous Sarcoidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00794274
• Date of registration: 19/11/2008
• Prospective Registration: No
• Primary sponsor: University of Cincinnati
• Public title: The Efficacy and Safety of CC-10004 in Chronic Cutaneous Sarcoidosis
• Scientific title: The Efficacy and Safety of CC-10004 in Chronic Cutaneous Sarcoidosis
• Date of first enrolment: November 2008
• Target sample size: 15
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00794274
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: Robert P Baughman
• Affiliation: University of Cincinnati

Key inclusion & exclusion criteria

Inclusion Criteria:

- Must understand and voluntarily sign an informed consent form

- Must be male or female and aged = 18 years at time of consent

- Must be able to adhere to the study visit schedule and other protocol requirements

- Patients with sarcoidosis as defined by the ATS/ERS/WASOG statement on sarcoidosis as
defined by the clinical presentation consistent with sarcoidosis, biopsy finding
granulomas, and no alternative for the cause of the granulomas, such as tuberculosis

- Patients must have chronic cutaneous skin lesions while taking chronic therapy
(corticosteroids, methotrexate (max 10mg/week), azathioprine, hydroxychloroquine,
cyclophosphamide, minocycline, doxycycline and chloroquine), in which the dose has
not been altered in the three months prior to starting the study.

- Must have two visits within the previous 1-6 months (at least one month apart) with
stable skin lesions, such as a SASI score was within one point for each of the
features of the lesion.

- Must meet the following laboratory criteria:

- Hemoglobin > 9 g/dL

- Hematocrit = 27%

- White blood cell (WBC) count = 3000 (= 3.0 X 109/L) and < 20,000
(< 20 X 109/L)

- Neutrophils = 1500 (= 1.5 X 109/L)

- Platelets = 100,000 (= 100 X 109/L)

- Serum creatinine = 1.5 mg/dL (= 132.6 µmol/L)

- Total bilirubin < 2.0 mg/dL

- Aspartate transaminase (AST [serum glutamic oxaloacetic transaminase, SGOT]) and
alanine transaminase (ALT [serum glutamate pyruvic transaminase, SGPT]) < 1.5x
upper limit of normal (ULN)

- Females of childbearing potential (FCBP)‡ must have a negative urine pregnancy test
at screening (Visit 1). In addition, sexually active FCBP must agree to use TWO of
the following adequate forms of contraception while on study medication: oral,
injectable, or implantable hormonal contraceptives; tubal ligation; intrauterine
device; barrier contraceptive or vasectomized partner. A FCBP must agree to have
pregnancy tests every 28 days while on study medication.

- Males (including those who have had a vasectomy) must agree to use barrier
contraception (latex condoms) when engaging in sexual activity with FCBP while on
study medication and for 84 days after taking the last dose of study medication.

Exclusion Criteria:

- History of any clinically significant cardiac, endocrinologic, pulmonary, neurologic,
psychiatric, hepatic, renal, hematologic, immunologic, or other major diseases (not
including pulmonary sarcoidosis)

- Any condition, including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study or confounds
the ability to interpret data from the study

- Pregnant or lactating female

- History of active Mycobacterium tuberculosis infection (any subspecies) within 3
years prior to the screening visit. Infections that occurred > 3 years prior to
entry must have been effectively treated.

- History of incompletely treated latent Mycobacterium tuberculosis infection (as
indicated by a positive Purified Protein Derivative [PPD] skin test or in vitro test
[T-SPOT®.TB, QuantiFERON Gold®].

- Clinically significant abnormality on the chest x-ray (CXR) at screening not due to
sarcoidosis

- Use of any investigational medication within 28 days.

- Any clinically significant abnormality on 12-lead ECG at screening

- Positive human immunodeficiency virus (HIV), hepatitis B, or hepatitis C laboratory
test result indicating active infection at screening

- History of malignancy within previous 5 years (except for treated basal-cell skin
carcinoma(s) and/or fewer than 3 treated squamous-cell skin carcinomas)

- Use of infliximab, etanercept, adalimumab, pentoxifylline, or thalidomide in the
prior three months.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Cutaneous Sarcoidosis

Sarcoidosis

Intervention(s)

Drug: CC-100004

Primary Outcome(s)

Improvement in skin lesions as assessed by a Sarcoidosis Skin Activity and Severity Index (SASI) . [Time Frame: 6 months]

Secondary Outcome(s)

Improvement of global score of sarcoidosis using a visual analogue scale (VAS) by both the patient and a separate VAS by the physician. [Time Frame: 6 months]

Change in genomic and proteomic expression of cytokines in paired skin biopsies (non-facial lesions). [Time Frame: 6 months]

To determine the safety (type, frequency, severity, and relationship of adverse events to study treatment) of CC-10004 in patients with chronic cutaneous sarcoidosis. [Time Frame: 6 months]

Change in the quality of life using the Sarcoidosis Health Questionnaire and Short Form-36 (SF-36). [Time Frame: 6 months]

Improvement in pulmonary status, specifically the six-minute walk test, dyspnea score, and forced vital capacity (FVC). [Time Frame: 6 months]

Secondary ID(s)

AP0005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Medical University of South Carolina

Celgene Corporation