Oral LGD-4665 Versus Placebo in Adults With Immune Thrombocytopenic Purpura (ITP) for 6 Weeks Plus Open Treatment Continuation

• Status: Completed
• Trial end date: 2009-05-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to assess the ability of LGD-4665 given daily by mouth to increase platelet counts in the treatment of patients with ITP (immune thrombocytopenic purpura). LGD-4665 increased platelet counts safely and tolerably compared to placebo in healthy volunteers. This study will examine the safety, tolerability and efficacy of 7.5 mg capsules of LGD-4665 to increase platelets compared to placebo, randomized 2:1, during blinded treatment for 6 weeks. Evaluation of platelet counts, bleeding scores and safety parameters will be done weekly. All patients are eligible to continue on active, open LGD-4665 treatment for an additional 12 weeks with optimal adjustment of dose for each patient.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: GlaxoSmithKline

Criteria

Inclusion Criteria:

• Adults 18 years or older

• Diagnosis of ITP for at least 3 months consistent with ASH guidelines

• Treated with one or more prior therapies for ITP and platelet counts < 30,000/µL or < 50,000/µL if on a stable oral corticosteroid for ≥ 4 weeks, supported by 2 platelet counts in prior 30 days

• Laboratory results within normal range except for the following analytes

• Hemoglobin ≥ 10 g/dL

• Absolute neutrophil counts > 1000/mL

• ALT ≤ 1.5X ULN

• AST ≤ 1.5X ULN

• Creatinine < 1.5X ULN

• Bilirubin < 1.5X ULN

• BUN < 1.5X ULN

• PT < 1.5X ULN

• aPTT <1.5X ULN

• Women of child-bearing potential must have a negative serum pregnancy test within 4 days prior to the first dose of study treatment and agree to practice an approved method of contraception or abstinence from sexual intercourse.

• Willing to sign a written informed consent

Exclusion criteria:

• History of heart attack or cardiovascular disease

• Known history of arterial or venous thrombosis

• More than 3 risk factors for thromboembolic events (diabetes, smoker, using oral contraception, using estrogen therapy, hypertriglyceridemia, average cholesterol > 240 mg/dL, treatment for hypertension)

• Active cancer or a history of bone marrow disorders

• Women who are pregnant or nursing

• History of alcohol/drug abuse or dependence within one year

• Listed medications dosed within:

• 4 weeks of the first dose of the study treatment:

• Use of Rituximab

• Use of cytotoxic agents

• Use of Cyclosporine and other immunomodulators

• Use of an investigational drug

• 2 weeks of the first dose of the study treatment:

• Use of Danazol

• Use of Azathioprine

• Use of Mycophenolate mofetil and pulsed-dose steroids

• 1 week of the first dose of the study treatment:

• Use of Anti-D (WinRho®)

• Use of IVIG

• Had a platelet transfusion

• Use of herbal/dietary supplements (excluding vitamins and mineral supplements)

• 3 days of the first dose of the study treatment

• Use of aspirin, aspirin containing compounds

• salicylates

• milk of magnesia

• non-steroidal anti-inflammatory drugs (unless prescribed for heart disease)

• History of platelet aggregation that would prevent measurement of platelet counts

• Known active infection with HIV, hepatitis B, or hepatitis C

• In the Investigator's opinion, the patient is not able to comply with requirements of the study