The null hypothesis of no difference in CR% at 3 months between the arms will be tested against the alternative of a difference in CR% at an alpha level of .05 by assessing the odds ratio for arm yielded by this model.
This is a superiority trial aiming to increase the 3 month complete response rate. The sample size is calculated on the hypothesis that the experimental treatment will increase the 3 months response rate up to 21% (by 3 folds, based on the 7% reported in Scheinberg et al \[17\]). Under these assumptions, the sample size to reject the null hypothesis is n=96 patients for each treatment arm, increased by 4% for possibly not evaluable patients (total number of 200 patients, 100 each treatment arm). Statistical design for sample size calculation: increase from 7% (control arm) to 21% (investigational arm) in 3 month complete response rate (two-sided binomial test); alpha-error 0.05; power 0.8.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
202
Hopital Jean Minjoz
Besançon, France
CR rate
The primary objective of this trial is to investigate whether Eltrombopag added to standard immunosuppressive treatment increases the rate of early (at three months) complete response in untreated AA patient.
Time frame: 3 months
Time to best heamatological response
Time frame: 2 year
Heamatological Response at 6, 12, 18 and 24 months
Time frame: 2 year
Cumulative incidence of response
Time frame: 2 year
Overall survival
Time frame: 2 year
Event-free survival
Time frame: 2 year
Cumulative incidence of relapse rate
Time frame: 2 year
Cumulative incidences of clonal evolution
Time frame: 2 year
Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence
Time frame: 2 year
Cumulative incidence of discontinuation of immunosuppressive therapy
Time frame: 2 year
Rate of CsA-independent hematological response at 24 months
Time frame: 2 year
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Lille, France
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Lyon, France
St. Louis Hospital
Paris, France
Pontchaillou Hospital
Rennes, France
Hôpital Purpan
Toulouse, France
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
Istituto G. Gaslini children's Hospital
Genova, Italy
San Martino Hospital
Genova, Italy
...and 19 more locations
Need for transfusions and number of transfusions required from treatment
Time frame: 2 year
Need for any supportive care
Time frame: 2 year
Comparison of number of SAEs between the two arms
To look for the safety and tolerability of the investigational treatment
Time frame: 2 year