Datum der Studienregistrierung
17.07.2018
Einschluss der ersten teilnehmenden Person
26.09.2018
Rekrutierungsstatus
Authorised-recruitment may be ongoing or finished
Wissenschaftlicher Titel
(Datenquelle: WHO)
A phase II, open-label, prospective, single-arm, study to assess ability of eltrombopag to induce sustained remission in subjects with ITP who are refractory or relapsed after first-line steroids (TAPER)
Studientyp
(Datenquelle: WHO)
Interventional clinical trial of medicinal product
Design der Studie
(Datenquelle: WHO)
Controlled: noRandomised: noOpen: yesSingle blind: noDouble blind: noParallel group: noCross over: noOther: noIf controlled, specify comparator, Other Medicinial Product: noPlacebo: noOther: no
Phase
(Datenquelle: WHO)
Human pharmacology (Phase I): noTherapeutic exploratory (Phase II): yesTherapeutic confirmatory - (Phase III): noTherapeutic use (Phase IV): no
Primäre Endpunkte
(Datenquelle: WHO)
Main Objective: To assess ability of eltrombopag to induce sustained remission by month 12 in ITP subjects who relapsed or failed to respond to first-line steroid treatment;Secondary Objective: assess 1.duration of sustained remission after treatment discontinuation by M12 and 24 2.proportion of patients maintaining a sustained remission after treatment discontinuation until M24 3.ability of eltrombopag to induce early response by month 1 4.ability of eltrombopag to induce a recovery response, in case of loss of response during or after tapering of eltrombopag until M 24 5.platelet count from baseline and every 3M until M24 6.ability of eltrombopag to maintain platelet count = 30×109/L within 12M and every 3M until 24M 7.evaluate patient-Health Related outcome measures for health-related quality of life (fatigue level of the patients through FACIT) & FACT-Th6 and SF-36v2 questionnaires from baseline and every 3M to 24M and end of treatment/ to explore 8.overall impact of side effects on treatment via the GP5 at baseline,M12, and end
of treatment 9.treatment satisfaction with TSQM-9 at baseline, M12, and end of treatment 10.evaluate the safety and tolerability of eltrombopag;Primary end point(s): Proportion of patients with sustained remission (R) by month 12 where
sustained remission is defined as:
? reach platelet count = 100×109/L (complete response [CR]) and then
maintain platelet counts around 100×109/L for 2 months (no counts
below 70×109/L) AND then
? taper off the drug until treatment discontinuation while,
? maintain platelet count = 30×109/L in the absence of bleeding (no
bleeding AEs) or use of any rescue therapy until month 12.
;Timepoint(s) of evaluation of this end point: 12 months
Sekundäre Endpunkte
(Datenquelle: WHO)
Secondary end point(s): 1a. Median duration of sustained remission (weeks) counted from last
dose of eltrombopag to month 12 for patients with sustained remission
(R)
1b. Estimated median duration of sustained remission (weeks) by month
24 for patients who are in sustained remission (R) at month 12 and
enter 12 months follow-up period using Kaplan-Meier method
1c. Estimated median duration of sustained remission (weeks) by month
24 for all patients using Kaplan-Meier method.
2. Proportion of sustained remission (R) patients at months 15, 18, 21,
and 24.
3. Number (%) of patients with platelet count = 50×109/L at least once
by month 1 (first month) without bleeding and no rescue therapy
4. Number (%) of patients with at least one platelet count = 30×109/L
after eltrombopag is re-introduced, in case of loss of response (<
30×109/L and/or bleeding event) without bleeding and no rescue
therapy by month 12 and 24
5. Absolute and relative change in platelet count from baseline to 3, 6,
9,12, 15, 18, 21, and 24 months and end of treatment
6. Number (%) of patients who maintain a platelet count = 30×109/L
from the first time of reaching that level to month 3, 6, 9, 12, 15, 18, 21,
24, and end of treatment without bleeding and no rescue therapy
7. The analysis of HRQoL parameters; fatigue level of the patients
through FACIT & FACT-Th6, SF-36v2 questionnaires. Change in scores
from baseline to month 3, 6, 9, 12, 15, 18, 21, 24, and end of treatment
8. Overall change from baseline to month 12 and end of treatment will
be assessed
9. Overall change from baseline to month 12 and end of treatment will
be assessed
10. Number (%) and severity of patients with AEs, serious AEs (SAEs),
AEs leading to discontinuation, AEs leading to dose adjustments, AEs of
special interest. Change from baseline in vital signs and clinical
laboratory tests;Timepoint(s) of evaluation of this end point: 1. 12 months
1b. 24 months
1c. 24 months
2. at months 15, 18, 21 and 24
3. 1 month
4. 12 and 24 months
5. from baseline to 3, 6, 9,12, 15, 18, 21, and 24 months and end of
treatment
6.from the first time of reaching required platelet level to month 3, 6, 9,
12, 15, 18, 21, 24, and end of treatment
7. from baseline to month 3, 6, 9, 12, 15, 18, 21, 24, and end of
treatment
8. from baseline to month 12 and end of treatment
9. from baseline to month 12 and end of treatment
10. from baseline
Kontakt für Auskünfte
(Datenquelle: WHO)
Novartis Pharma AG
