ENDURE - Efficacy and Safety of AOP2014 With CML Patients in Remission (ENDURE-CML-IX)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03117816|
Recruitment Status : Recruiting
First Posted : April 18, 2017
Last Update Posted : September 16, 2020
|Condition or disease||Intervention/treatment||Phase|
|Chronic Myeloid Leukemia in Remission||Drug: AOP2014 / Pegylated-Proline-interferon alpha-2b Other: Surveillance||Phase 2|
Four hypotheses are tested in hierarchical order. To avoid inflation of type 1 error (false rejection of a null hypothesis), further confirmatory testing has to be stopped as soon as a null hypothesis could not be rejected.
All four hypotheses are tested at significance level 0.05. Null hypotheses 1, 2, and 4 deal with probabilities of molecular relapse-free survival 7, 13, and 25 months after randomisation, respectively; arms A and B are compared with the uncorrected chi-square test. Null hypothesis 3 investigates molecular relapse-free survival as a time-to-event variable; the two arms are compared with the log-rank test
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||214 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Single (Outcomes Assessor)|
|Masking Description:||A randomized, open-label assessor blinded, multi-center, controlled phase II trial|
|Official Title:||Efficacy and Safety of Pegylated Proline Interferon Alpha 2b (AOP2014) in Maintaining Deep Molecular Remissions in Patients With Chronic Myeloid Leukemia (CML) Who Discontinue ABL-Kinase Inhibitor Therapy - a Randomized Phase II, Multicenter Trial With Post-study Follow-up|
|Actual Study Start Date :||May 4, 2017|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||December 2024|
Experimental: investigational arm A
There will be an overlapping treatment with AOP2014 and TKI for one month. After one month, the TKI therapy will be stopped and patient will receive only AOP2014 treatment for the next 14 months.
Drug: AOP2014 / Pegylated-Proline-interferon alpha-2b
AOP2014 as pre-filled auto-injection pen for subcutaneous injection, containing 250 µg AOP2014 / 0.5 ml. The solution also contains inactive ingredients (sodium chloride, polysorbate 80, benzyl alcohol, sodium acetate, and acetic acid). The solution is colorless to light yellow.
surveillance arm B
This is an open-label study with a "surveillance" group as comparator arm. Similar as in the arm A, patient will discontinue TKI therapy one month after randomization. From then on patient will receive no further CML treatment.
For patients randomized into this treatment arm stopp their standard treatment and will just be under observation.
- RFS 7 [ Time Frame: 7 months after randomization ]The primary efficacy endpoint is molecular relapse free survival, RFS 7 months after randomization. Relapse is defined as loss of major molecular remission, MMR, which is any increase of the BCR-ABL ratio to > 0.1% according to the international scale (IS). Time to relapse is defined as the time from randomization to relapse
- RFS 13 [ Time Frame: 13 months after randomization ]The relapse free survival, RFS 13 months after randomization
- RFS 25 [ Time Frame: 25 months after randomization ]The relapse free survival, RFS 25 months after randomization
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Day 0 - Month 15 (Arm B) or Month 16 (additional safety visit one month after last application for Arm A) ]Adverse events, serious adverse events (AEs, SAEs)• Safety, tolerability and toxicity based on incidences of adverse events, serious adverse events
- Quality of life measured by EORTC QLQ-C30 [ Time Frame: Day 0 - Month 25 ]The data will be compared between the treatment groups and to QoL of normal population.
- Quality of life measured by EORTC-QLQ-CML24 [ Time Frame: Day 0 - Month 25 ]The data will be compared between the treatment groups and to QoL of normal population. Furthermore, results of the CML24 module should be shared with the EORTC group to complete the validation of this questionnaire
- detection of blood parameter 95 CD86+pDC as RFS predictor [ Time Frame: Day 0 - Month 15 ]To validate the value of 95 CD86+pDC / 105 lymphocytes at baseline (before TKI stop) as a predictor of RFS
- OS (overall survival) [ Time Frame: Day 0 - Month 25 (plus annual post study follow up Months 36,48,60) ]Overall survival (OS), defined as the time between the date of randomization and the date of death from any cause.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03117816
|Contact: Andreas Burchert, Prof. Dr.||0049 6421 586 ext email@example.com|
|Contact: Kerstin Balthasar||0049 6421 286 ext firstname.lastname@example.org|
|Study Director:||Andreas Burchert, Prof. Dr.||Philipps University Marburg Medical Center|
|Principal Investigator:||Franck E Nicoloni, MD, PhD||Centre Léon Bérard Lyon|