End of EXTEND: Discontinuation of Medication for Patients With Immune Thrombocytopenia

End of EXTEND: Observing for Sustained Response Despite Discontinuation of Medication in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP) Treated With Eltrombopag

This study proposes to observe whether a stable platelet count would be maintained without additional treatment in the long term in at least a proportion of patients who have discontinued eltrombopag taken for at least 4 months. This requires that if patients stop treatment with eltrombopag, they are not immediately transitioned to further treatment unless it is necessary.

The objective of the study is to assess how frequently patients who have discontinued eltrombopag attain a stable, treatment-free, unmaintained adequate platelet count 4 to 8 weeks after discontinuing eltrombopag and how long such a response lasts (if it occurs).

Study Overview

• Status: Terminated
• Conditions: Immune Thrombocytopenia
• Intervention / Treatment: Drug: Discontinuation of eltrombopag

• Study Type: Observational
• Enrollment (Actual): 35

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10065
      • Weill Cornell Medical College/New York Presbyterian Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

ITP patients discontinuing the use of eltrombopag

Description

Inclusion Criteria:

• A subject is eligible for study entry if all of the following criteria apply:

  • Subject or their guardian has signed and dated a written informed consent
  • Male or female adults (≥ 18 years) diagnosed with ITP according to the new consensus guidelines
  • No indication of a disease which may cause thrombocytopenia other than ITP.
  • Having taken eltrombopag for at least 4 months prior to beginning of study.
  • Subject experienced no toxicity other than transient eltrombopag-related toxicity or other drug intolerance.
  • Subjects treated with concomitant ITP medications (e.g. corticosteroids or azathioprine) must be receiving a dose that has been stable for at least four weeks prior to start of this study.
  • A subject is practicing an acceptable method of contraception (documented in chart). female subjects or female partners of male subjects must either be of non-child bearing potential (hysterectomy, bilateral ovariectomy, bilateral tubal ligation or post menopausal for more than one year) OR of child bearing potential using one of the following highly effective methods of contraception.
• complete abstinence from intercourse
• Intrauterine device (IUD)
• Two forms of barrier contraception. diaphragm plus spermicide, or for males condoms plus spermicide.
• Male partner is sterile and is the only partner of the female.
• Systemic contraceptives (combined oral progesterone only)

Exclusion Criteria:

A subject is at least temporarily ineligible for the study if any of the following criteria apply:

• Any clinically relevant abnormality, other than chronic ITP, identified on the screening examination, or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study such as:

  • an active malignancy
  • an arterial or venous thrombosis
  • development of grade III-IV cardiovascular disease (including congestive heart failure, New York Heart Association (NYHA Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation), or subjects with a QTc >480 msec.
• Recent history of alcohol/drug abuse.
• Subjects recently treated with drugs that affect platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDs) or anti-coagulants for > 3 consecutive days within 2 weeks of the study start and until the end of the study.
• development of platelet agglutination abnormality that prevents reliable measurement of platelet counts.

Rescreening is possible if the condition resulting in the failed screening has been resolved. There will be no limit on the number of screening tests and the screening will be valid for 30 days.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Discontinuation of eltrombopag; ITP subjects who have discontinued the use of eltrombopag	Drug: Discontinuation of eltrombopag; Observation of subjects as they discontinue the use of eltrombopag; Other Names: eltrombopag, promacta

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet Response	To determine the frequency of a sustained response of at least 6 months in subjects with persistent or chronic ITP who have been receiving eltrombopag for a minimum of four months and are able to discontinue it within two years of initiating the tapering	6 months

Collaborators and Investigators

• Sponsor: Weill Medical College of Cornell University
• Collaborators: Novartis
• Investigators: Principal Investigator: James B. Bussel, M.D., Weill Medical College of Cornell University

Study record dates

Study Major Dates

• Study Start: June 1, 2011
• Primary Completion (Actual): January 31, 2017

Study Registration Dates

• First Submitted: June 13, 2011
• First Submitted That Met QC Criteria: June 30, 2011
• First Posted (Estimate): July 1, 2011

Study Record Updates

• Last Update Posted (Actual): July 17, 2018
• Last Update Submitted That Met QC Criteria: July 13, 2018
• Last Verified: July 1, 2018

More Information

Terms related to this study

• Keywords: eltrombopag; promacta; immune thrombocytopenia (ITP)
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: 1003010950

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No