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

NCT00794274 | Completed Phase 2 DMC

• 1 other identifier: AP0005
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

To determine whether CC-10004, a phosphodiesterase inhibitor, is useful in treating chronic cutaneous sarcoidosis.

Conditions

Sarcoidosis; Cutaneous Sarcoidosis

Keywords

Sarcodiosis; tumor necrosis factor

Outcome Measures

Primary Outcomes (1)

• Improvement in skin lesions as assessed by a Sarcoidosis Skin Activity and Severity Index (SASI) .

  6 months

Secondary Outcomes (5)

• To determine the safety (type, frequency, severity, and relationship of adverse events to study treatment) of CC-10004 in patients with chronic cutaneous sarcoidosis.

  6 months

• Improvement of global score of sarcoidosis using a visual analogue scale (VAS) by both the patient and a separate VAS by the physician.

  6 months

• Change in the quality of life using the Sarcoidosis Health Questionnaire and Short Form-36 (SF-36).

  6 months

• Change in genomic and proteomic expression of cytokines in paired skin biopsies (non-facial lesions).

  6 months

• Improvement in pulmonary status, specifically the six-minute walk test, dyspnea score, and forced vital capacity (FVC).

  6 months

Study Arms (1)

Open label drug

EXPERIMENTAL

Drug: CC-100004 After the screening period, subjects will receive CC-10004 20mg by mouth BID for 84 days. The 84-day duration of treatment is expected to provide adequate time to assess the short-term efficacy and safety of CC-10004 in a population of subjects with chronic cutaneous sarcoidosis

Drug: CC-100004

Interventions

CC-100004 DRUG

After the screening period, subjects will receive CC-10004 20mg by mouth BID for 84 days. The 84-day duration of treatment is expected to provide adequate time to assess the short-term efficacy and safety of CC-10004 in a population of subjects with chronic cutaneous sarcoidosis.

Also known as: Aprelimast

Open label drug

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Cincinnati: lead
• Celgene Corporation: collaborator
• Medical University of South Carolina: collaborator

Study Sites (1)

University of Cincinnati

Cincinnati, Ohio, 45220, United States

Location

Related Publications (1)

• Baughman RP, Judson MA, Ingledue R, Craft NL, Lower EE. Efficacy and safety of apremilast in chronic cutaneous sarcoidosis. Arch Dermatol. 2012 Feb;148(2):262-4. doi: 10.1001/archdermatol.2011.301. Epub 2011 Oct 17. No abstract available.

  PMID: 22004880 DERIVED

MeSH Terms

Conditions

Sarcoidosis

Condition Hierarchy (Ancestors)

Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases

Study Officials

• Robert P Baughman

  University of Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Medicine

Study Record Dates

• First Submitted: November 19, 2008
• First Posted: November 20, 2008
• Study Start: November 1, 2008
• Primary Completion: December 1, 2011
• Study Completion: December 1, 2011
• Last Updated: April 12, 2013

Record last verified: 2013-04

Locations

US (1)