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

NCT00621894 | Completed Phase 2

• 1 other identifier: L4665-03
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 15

Brief 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.

Conditions

Immune Thrombocytopenic Purpura

Keywords

Immune thrombocytopenic purpura; thrombopoietin mimetic; ITP

Outcome Measures

Primary Outcomes (1)

• Percentage of participants with platelet count >= 50000/µL

  Response was defined as platelet count \>= 50 x1000/uL for participants without Baseline steroid uses; or platelet counts \>= 50 x1000/uL and doubling the Baseline platelet counts for participants with baseline steroid uses. Confidence interval of response rate was computed using exact method of binomial proportion.

  At Week 6

Secondary Outcomes (3)

• Number of participants with time to response by Platelet Counts (platelet counts >= 50,000/µL)

  Week 1, 2, 4 and 6 of part 1

• Change From Baseline to Last Bleeding Observation During Double-Blind Treatment

  Day 1 (Baseline) and Week 6

• Duration of platelet counts >= 50,000/µL of LGD4665

  Up to Week 6

Study Arms (2)

LGD4665

EXPERIMENTAL

LGD-4665: Experimental Thrombopoietin mimetic

Drug: LGD-4665

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

LGD-4665 DRUG

LGD-4665 Thrombopoietin mimetic

LGD4665

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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:
• 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
• weeks of the first dose of the study treatment:
• Use of Danazol
• Use of Azathioprine

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (15)

University of California San Diego Medical Center

San Diego, California, 92103-8409, United States

Location

University of California, San Francisco

San Francisco, California, 94143-1270, United States

Location

Davis, Posteraro and Wasser, MD's LLP

Manchester, Connecticut, 06040, United States

Location

Baptist Cancer Institute

Jacksonville, Florida, 32207, United States

Location

Cancer Center of Florida

Orlando, Florida, 32806, United States

Location

Georgia Cancer Specialists

Atlanta, Georgia, 30341, United States

Location

Karmanos Cancer Center, Wertz Clinical Cancer Center 4HWCRC

Detroit, Michigan, 48201, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Washington University School of Medicine - St Louis, MO

St Louis, Missouri, 63110, United States

Location

New Mexico Oncology Hematology Consultants

Albuquerque, New Mexico, 87109, United States

Location

Joan and Sanford I. Weill Medical College, Cornell University

New York, New York, 10021, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

Case Western Reserve University School of Medicine

Cleveland, Ohio, 44106-7284, United States

Location

Cleveland Clinic Foundation, Univ. of Ohio

Cleveland, Ohio, 44195, United States

Location

Hematology Oncology Associates of South Texas

San Antonio, Texas, 78229, United States

Location

Related Links

• Results for study L4665-03 can be found on the GSK Clinical Study Register.

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2008
• First Posted: February 22, 2008
• Study Start: March 15, 2008
• Primary Completion: May 1, 2009
• Study Completion: May 15, 2009
• Last Updated: December 16, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (15)