Know Cancer

forgot password

A Phase IIA Randomized, Double-Blind, Placebo-Controlled Study of LGD-4665 in Patients With Immune Thrombocytopenic Purpura (ITP) With an Open Label Extension

Phase 2
18 Years
Not Enrolling
Immune Thrombocytopenic Purpura

Thank you

Trial Information

A Phase IIA Randomized, Double-Blind, Placebo-Controlled Study of LGD-4665 in Patients With Immune Thrombocytopenic Purpura (ITP) With an Open Label Extension

This is a Phase IIA study with two parts to the design.

- Part 1 is a randomized, double-blinded, placebo-controlled treatment of 7.5 mg/day
LGD-4665 versus placebo in approximately 24 patients with ITP who have been treated
with at least one prior therapy for ITP. Patients will be randomized in a ratio of 1:2
(placebo: 7.5 mg/day LGD-4665) for 6 weeks of treatment. Platelet counts, bleeding
scores, vital signs, physical exams and laboratory tests will be assessed weekly.
Treatment groups will be analyzed for efficacy by the percentage of patients with
platelet counts two times baseline and ≥ 50,000/uL at 6 weeks of treatment, and for
safety by adverse events, vital signs, physical exams, laboratory tests and use of ITP
rescue medications or transfusions.

- Part 2 is an extension of study treatment with open label LGD-4665. All patients who
participate in the Part 1 randomized double-blind treatment of this Ph IIA trial are
eligible to continue open label treatment with LGD-4665 for up to 3 months at an
appropriate dose for the safe maintenance of platelet counts (≥ 50,000/uL to ≤
200,000/uL). Assessments of effectiveness and safety will be made at 2 and 4 week

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

- 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

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Platelet counts

Outcome Time Frame:

Week 6

Safety Issue:


Principal Investigator

GSK Clinical Trials

Investigator Role:

Study Director

Investigator Affiliation:



United States: Food and Drug Administration

Study ID:




Start Date:

March 2008

Completion Date:

May 2009

Related Keywords:

  • Immune Thrombocytopenic Purpura
  • Immune thrombocytopenic purpura
  • thrombopoietin mimetic
  • ITP
  • Purpura
  • Purpura, Thrombocytopenic
  • Purpura, Thrombocytopenic, Idiopathic



Mount Sinai School of Medicine New York, New York  10029
Georgia Cancer Specialists Decatur, Georgia  30033
University of California, San Francisco San Francisco, California  94143
Henry Ford Health System Detroit, Michigan  48202
New Mexico Oncology Hematology Consultants Albuquerque, New Mexico  87102
Cancer Center of Florida Ocoee, Florida  34761
Hematology Oncology Associates of South Texas San Antonio, Texas  78229
Baptist Cancer Institute Jacksonville, Florida  32207
University of California San Diego Medical Center San Diego, California  92103-8409
Davis, Posteraro and Wasser, MD's LLP Manchester, Connecticut  06040
Karmanos Cancer Center, Wertz Clinical Cancer Center 4HWCRC Detroit, Michigan  48201
Washington University School of Medicine - St Louis, MO St. Louis, Missouri  63110
Joan and Sanford I. Weill Medical College, Cornell University New York, New York  10021
Cleveland Clinic Foundation, Univ. of Ohio Cleveland, Ohio  44195
Case Western Reserve University School of Medicine Cleveland, Ohio  44106-7284