Thalidomide for Decreasing Collagen Biosynthesis in People With Progressive Systemic Sclerosis

NCT00418132 | Terminated Phase 1 DMC

• 1 other identifier: K23AR002187
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

Progressive systemic sclerosis (SSc) is an immune-based disease that causes abnormal connective tissue growth of the skin and internal organs. At this point, there are no effective therapies for treating SSc. Thalidomide is a medication that has been shown to stimulate an immune response that reduces the body's synthesis of collagen, the main component of connective tissue. This study will determine the effectiveness of thalidomide in treating adults with SSc.

Conditions

Scleroderma, Systemic

Keywords

SSc; Systemic Sclerosis; Progressive Systemic Sclerosis

Outcome Measures

Primary Outcomes (2)

• Collagen mRNA levels in the skin

  Measured at Weeks 16 and 48

• In vivo collagen production

  Measured at Weeks 16 and 48

Secondary Outcomes (4)

• Immune function

  Measured at Weeks 4, 16, and 48

• Clinical disease measures

  Measured at Weeks 16 and 48

• Hypothalamic-Pituitary-Adrenal (HPA) axis measures

  Measured at Weeks 16 and 48

• Safety measures

  Measured at Weeks 4, 16, and 48

Study Arms (2)

1

EXPERIMENTAL

Participants will receive thalidomide.

Drug: Thalidomide

2

PLACEBO COMPARATOR

Participants will receive placebo thalidomide.

Drug: Placebo thalidomide

Interventions

Thalidomide DRUG

Thalidomide at a dose of 50 mg/day. The dose will be increased to 100 mg/day at Week 2, then to 200 mg/day at Week 4, and finally to 300 mg/day at Week 6.

1

Placebo thalidomide DRUG

Participants will receive placebo thalidomide. The placebo dose will be increased through to Week 6.

2

Eligibility Criteria

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

You may qualify if:

• Diagnosis of scleroderma
• Agrees to use an effective form of contraception for 1 month prior to study entry, throughout the study, and for 60 days after completing the study
• Positive serum anti-nuclear antibody titer

You may not qualify if:

• Systemic sclerosis-like illnesses associated with environmental, ingested, or injected agents or with other connective tissue diseases
• Significant existing damage to any of the following internal organs:
• Kidneys, defined as a serum creatinine level greater than 2 mg/dl or renal crisis
• Lungs, defined as needing supplemental oxygen
• Heart, defined as left ventricular ejection fraction less than or equal to 40%
• Gut, defined as pseudo-obstruction or malabsorption requiring total parental nutrition
• Concurrent interventional therapy that might independently influence the outcome of this trial (e.g., D-penicillamine, cyclosporine, interferon-γ, methotrexate, or photophorosis)
• Clinically significant and inadequately medically treated concurrent endocrine, blood, liver, lung, or kidney diseases
• Pregnant
• Recent drug or alcohol abuse
• Documented noncompliance
• Significant psychiatric history
• Therapy with another investigational drug within 4 weeks prior to study entry
• Screening laboratory results exceeding the following limits: hemoglobin level less than 7 gm/dl; white blood cell level less than 3,000/nl; platelet count less than 50/nl; alanine aminotransferase (ALT) level greater than 65 U/L; creatinine level greater than 2 mg/dl

Sponsors & Collaborators

• NYU Langone Health: lead
• National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS): collaborator

Study Sites (1)

New York University School of Medicine General Clinical Research Center, Bellevue Hospital

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Scleroderma, Systemic; Scleroderma, Diffuse

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Stephen J. Oliver, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: January 3, 2007
• First Posted: January 4, 2007
• Study Start: August 1, 2000
• Study Completion: October 1, 2007
• Last Updated: March 11, 2016

Record last verified: 2016-03

Locations

US (1)