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Immune Thrombocytopenic PurpuraPhase 2NCT00621894

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

A Phase 2 study testing LGD-4665 in people with Immune Thrombocytopenic Purpura, run by GlaxoSmithKline at 15 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Immune Thrombocytopenic Purpura
What is being tested?
LGD-4665
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
23 participants
Where
15 sitesUnited States
Study dates
15 Mar 2008to 15 May 2009
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

United States15 sites

  • New Mexico Oncology Hematology Consultants

    Albuquerque, New Mexico, 87109

  • Georgia Cancer Specialists

    Atlanta, Georgia, 30341

  • Case Western Reserve University School of Medicine

    Cleveland, Ohio, 44106-7284

  • Cleveland Clinic Foundation, Univ. of Ohio

    Cleveland, Ohio, 44195

  • Karmanos Cancer Center, Wertz Clinical Cancer Center 4HWCRC

    Detroit, Michigan, 48201

  • Henry Ford Health System

    Detroit, Michigan, 48202

  • Baptist Cancer Institute

    Jacksonville, Florida, 32207

  • Davis, Posteraro and Wasser, MD's LLP

    Manchester, Connecticut, 06040

  • Joan and Sanford I. Weill Medical College, Cornell University

    New York, New York, 10021

  • Mount Sinai School of Medicine

    New York, New York, 10029

  • Cancer Center of Florida

    Orlando, Florida, 32806

  • Hematology Oncology Associates of South Texas

    San Antonio, Texas, 78229

  • University of California San Diego Medical Center

    San Diego, California, 92103-8409

  • University of California, San Francisco

    San Francisco, California, 94143-1270

  • Washington University School of Medicine - St Louis, MO

    St Louis, Missouri, 63110

Other trials in United States

About this study

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

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.

Read the detailed description

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 intervals.
Being tested
LGD-4665, Placebo
Keywords
Immune thrombocytopenic purpura, thrombopoietin mimetic, ITP
Medical subject headings
Purpura, Thrombocytopenic, Idiopathic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LGD-4665Drug

    LGD-4665 Thrombopoietin mimetic

  • PlaceboDrug

    Placebo

Study groups in NCT00621894
Study groupRoleWhat they receive
LGD4665ExperimentalLGD-4665: Experimental Thrombopoietin mimetic
Receives: Drug: LGD-4665
PlaceboPlacebo comparatorPlacebo
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: At Week 6

Secondary outcomes (3)

  1. Number of participants with time to response by Platelet Counts (platelet counts >= 50,000/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.

    Measured: Week 1, 2, 4 and 6 of part 1

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

    ITP Bleeding Severity Scale was used for the analysis of bleeding score. Bleeding scores were, 0=None, 1=minor, and 2=major. Body sites and bleeding grade analysis was as follows: cutaneous (1= 1-5 bruises; scattered petechiae and 2= > 5 bruises, >2 centimeter [cm]; petechiae), oral mucosa (1= 1 blood blister or > 5 petechiae, gum bleeding < 5 minute[min], 2= multiple blood blisters; gum bleeding > 5 min), epistaxis (1= blood on blowing nose or epistaxis < 5 min, 2= bleeding > 5 min), gastrointestinal (1= occult blood, 2= gross blood), gynecological (1= spotting not at time of period, 2= bleeding not at time of period or very heavy period), urinary (1= microscopic (+ by dipstick), 2= macroscopic), pulmonary(1= possible symptoms but mild, 2= yes), subconjunctival (1= yes, 2= both eyes significantly involved), Intracranial (1= possible symptoms, 2= yes, clinically confirmed). Change from Baseline was calculated as Baseline value minus post-randomization value. Baseline was Day 1value.

    Measured: Day 1 (Baseline) and Week 6

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

    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.A Kaplan-Meier projection of time to response by platelet counts was analyzed.

    Measured: Up to Week 6

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Feb 2008 (estimated)
  2. Study start15 Mar 2008
  3. Primary completion1 May 2009
  4. Study completion15 May 2009

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00621894
Sponsor's protocol ID
L4665-03
Status last verified
Dec 2024
First submitted
12 Feb 2008
Last updated
16 Dec 2024
Results posted
No
Data monitoring committee
No
Individual data sharing
No: GSK will assess requests from qualified researchers for anonymized individual patient-level data and related study documents. Data sharing is subject to certain criteria, conditions, and exceptions. For further information, refer to https://www.gsk-studyregister.com/About\_GSK\_Patient\_Level\_Data\_Sharing\_Final\_13July2023.pdf.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing LGD-4665 in people with Immune Thrombocytopenic Purpura, run by GlaxoSmithKline at 15 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of participants with platelet count >= 50000/µL (time frame: At Week 6). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00621894.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Dec 2024.

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