PONTrack = With ponatinib on the track for treatment-free remission in CML [Germany]

What is the purpose of the study

Achieving and maintaining deep molecular response (DMR) to CML treatment is a precondition for successfully discontinuing TKI treatment and achieving treatment-free remission (TFR).

Many patients achieve major molecular response (MMR = BCR-ABL1 transcripts below 0,1% on the International Scale). However, quite a few do not reach DMR or stable DMR (MR4 or better = BCR-ABL below 0.01% IS) even after several years of treatment with TKIs.

The PONTrack study opens the possibility of TFR to these patients as treatment with ponatinib, a highly effective third generation TKI, may help achieve DMR. In Germany, ponatinib is approved for the treatment of CML, but only for patients who no longer benefit from other TKIs (TKI failure) or who do not tolerate other TKIs (intolerance), or for patients with evidence of a T315I mutation.

What will happen during the study

Following inclusion in this study, patients will be treated with ponatinib for two years. Depending on the molecular response to treatment, the initial dose of 30 mg will be reduced to (down to 15 mg) or increased (up to no more than 45 mg). Using the lowest possible dosage and close monitoring are expected to be associated with a clearly lower risk of adverse effects than seen in other studies while treatment efficacy is expected to be the same.

Discontinuation of TKI treatment after achieving DMR will not be attempted within the PONTrack study, but only after patients have completed their participation in this study. Provided patient informed consent, TKI discontinuation data will be collected in a planned registry to gain scientific knowledge.

Key inclusion criteria

This study includes male or female patients who:

Further criteria may apply. Please discuss these with your doctor or study staff.

Key exclusion criteria

This study does not include patients who:

• have not responded to any TKI at any time during CML treatment according to current ELN criteria (including any detection of mutations or additional cytogenetic aberrations)
• were previously diagnosed with accelerated phase (AP) or blast phase (BP) at any time in the history of the disease
• previously underwent or planned allogenic stem cell transplant (SCT)

Further criteria may apply. Please discuss these with your doctor or study staff.

Estimated primary completion date

Where can I find additional information

You can find a study description in the EU Clinical Trials Register. This is a database provided by the U. S. National Institutes of Health.
You can also find study information in German on the website of Deutsche CML Allianz.

Study centers / principal investigators

Germany

Universitätsklinikum der RWTH Aachen
Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation
Dr. M. Crysandt
52074 Aachen

Klinikum Bayreuth
Medizinische Klinik IV
Prof. Dr. Kiani
95445 Bayreuth

Klinikum Chemnitz
Klinik für Innere Medizin III
PD Dr. M. Hänel
09116 Chemnitz

MVZ Onkologische Kooperation Harz
Dr. Mark-Oliver Zahn
38642 Goslar

Universitätsklinikum Halle
Universitätsklinik und Poliklinik für Innere Medizin IV
PD Dr. Haifa Kathrin Al-Ali
06120 Halle

Onkologisches Ambulanzzentrum Hannover
Prof. Dr. M. Koenigsmann
30171 Hannover

Universitätsmedizin Mannheim
III. Medizinische Klinik
Prof. S. Saußele
68167 Mannheim

Klinikum Oldenburg AöR
Universitätsklinik für Innere Medizin – Onkologie und Hämatologie
Studienzentrum
Andreas Voß
26133 Oldenburg

Universitätsmedizin Rostock, ZIM II
Klinik für Hämatologie, Onkologie und Palliativmedizin
Prof. C. Junghanß
18057 Rostock

Universitätsklinikum Ulm
Klinik für Innere Medizin III
Priv.-Doz. Dr. med. Frank Stegelmann
Albert-Einstein-Allee 23
89081 Ulm