Clinical trial NCT01386723: End of EXTEND: Discontinuation of Medication for Patients With Immune Thrombocytopenia
Official title: End of EXTEND: Observing for Sustained Response Despite Discontinuation of Medication in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP) Treated With Eltrombopag
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Immune Thrombocytopenia
Keywords: eltrombopag
promacta
immune thrombocytopenia (ITP)
Lead sponsor: Weill Medical College of Cornell University
Sponsor class: OTHER
Collaborators: name: Novartis
First posted: 2011-07-01
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: ITP patients discontinuing the use of eltrombopag

Brief summary: 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Platelet Response
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Weill Cornell Medical College/New York Presbyterian Hospital
state: New York
Study officials:
affiliation: Weill Medical College of Cornell University
name: James B. Bussel, M.D.
role: PRINCIPAL_INVESTIGATOR
References: