Neoplasms

Blood and Lymph Conditions

All Conditions

Symptoms and General Pathology

Rare Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Neoplasms by Histologic Type

Hematologic Diseases

Myeloproliferative Disorders

Bone Marrow Diseases

Chronic Disease

Disease Attributes

Myeloid Leukemia

Chronic Graft Versus Host Disease

Chronic Myeloid Leukemia

Chronic Myeloproliferative Disorders

Antineoplastic Agents

All Drugs and Chemicals

Amino Acids

Ponatinib

Imatinib Mesylate

Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Tyrosine

Ponatinib is given orally 30 mg daily, with dose adjustment to 15 mg daily once a BCR-ABL1 level minor or equal to 0.1% (MMR) has been achieved and confirmed in the next test, 4 weeks apart. A return to prior, 30 mg, dose is due in case of return of BCR-ABL1 transcripts level to \> 1%. Dose adjustments for toxicity are detailed in the protocol.

Fausto Castagnetti

This study aims at evaluating the efficacy of treatment with ponatinib in patients with chronic myeloid leukemia who are in a chronic phase and who previously received treatment with imatinib but resulted to be resistant to it.

Phase 2, single-arm, multicentre, open label. No interim analysis is planned, but a monitoring committee will evaluate the data every 6 months. Ponatinib is given orally 30 mg daily, with dose adjustment to 15 mg daily once a BCR-ABL1 level smaller or equal to 0.1% (MMR) has been achieved and confirmed in the next test, 4 weeks apart. A return to prior, 30 mg, dose is due in case of return of BCR-ABL1 transcripts level to \> 1%. Dose adjustments for toxicity are detailed in the protocol. Treatment time will be 52 weeks, during which study drug will be provided free-of-charge by ARIAD Pharmaceuticals, upon approval of the protocol. Treatment is discontinued at any time in case of failure or treatment-related SAEs. After one year of treatment, upon request of the local investigator and upon confirmation of the Treatment Advisory Committee (TAC, see section 23), ARIAD Pharmaceutics, Inc. will continue to provide ponatinib for the study patients who will benefit from treatment continuation, for at least 2 years, until the drug will be approved with that indication.

Activity and Risk Profile of Ponatinib in Chronic Phase Patients With Chronic Myeloid Leukemia Resistant to Imatinib

Optimizing Ponatinib USe (OPUS). A GIMEMA Phase 2 Study of the Activity and Risk Profile of Ponatinib, 30 mg Once Daily, in Chronic Myeloid Leukemia (CML) Chronic Phase (CP) Patients Resistant to Imatinib

Cytogenetic response (CyR) is defined based on the percentage of Ph pos metaphases, as evaluated by chromosome banding analysis (CBA) of at least 20 marrow cell metaphases:

1. Major Cytogenetic Response if Ph pos metaphases \< 35%
2. Complete (CCyR) if Ph pos metaphases 0 or FISH BCR-ABL1 nuclei minor or equal to 1%
3. Partial (PCyR) if Ph pos metaphases 1-34%
4. Minor (mCyR) if Ph pos metaphases 35-65%
5. Minimal or none (min/none CyR) if Ph pos metaphases \> 65% If marrow cell metaphases cannot be obtained or analysed, interphase fluorescence-in-situ-hybridization (FISH) can be used, but only to distinguish a CCyR (minor or equal to 1% positive nuclei out of at least 200 nuclei) from a non CCyR. FISH data cannot be used to classify a response as minimal, minor, or partial.

Gruppo Italiano Malattie EMatologiche dell'Adulto

Philadelphia Chromosome

Nilotinib

The recommended dosing of nilotinib is 400 mg orally twice daily

The purpose of this Cohort Treatment Plan is to allow access to Nilotinib for eligible patients diagnosed with imatinib-intolerant and/or resistant Philadelphia Chromosome positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) or Chronic Myelogenous Leukemia in Accelerated Phase (CML-AP). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Nilotinib, for Patients With CML-CP or CML-AP

Managed Access Program to Provide Access to Nilotinib, for Patients With Imatinib-intolerant and/or Resistant Ph+ Chronic Myelogenous Leukemia (CML) in Chronic Phase or CML in Accelerated Phase

Novartis Pharmaceuticals

Dasatinib

Adult patients with Imatinib resistant (failure + suboptimal) or intolerant chronic myeloid leukaemia in all phases, who started treatment with Nilotinib between January 2005 and December 2012 in Italy.

Adult pts treated with Nilotinib as second line therapy after Dasatinib.

Observation of Imatinib resistant patients treated with Nilotinib.

Imatinib

Giuseppe SAGLIO, Pr.

The GIMEMA CML Working Party promotes an observational (retrospective and perspective) study of Imatinib-resistant or intolerant CML patients treated with Nilotinib in Italy. Enrollment will include all patients who started Nilotinib between January 2005 and December 2012. Patients will be followed for 4 years since treatment start. After this time, survival data, disease status and treatment will be recorded at 6-months-interval. This study will help the definition of guidelines for a proper management of Nilotinib in any-phase CML patients.

Follow-up is required until the end of treatment for the purposes of the study for all patients by standard hematologic, cytogenetic and molecular criteria. This study will not contemplate any additional expense beyond what is expected for a regular follow-up, according to the international guidelines for CML.

Sample Size: target accrual was not defined, all eligible patients observed between January 2005 and December 2012 will be included.

Observational Study in Adults With Imatinib-resistant/Intolerant Chronic Myeloid Leukemia Treated With Nilotinib

Observational Study in Adult Patients With Imatinib-resistant or Intolerant Chronic Myeloid Leukemia (CML) Treated With Nilotinib: Follow-up of the Italian Patients. GIMEMA Study CML0609

Rate of complete haematologic remission with Nilotinib treatment and the duration of the responses.

Rate of major cytogenetic response with Nilotinib treatment and the duration of the responses.

Rate of complete cytogenetic response with Nilotinib treatment and the duration of the responses.

Rate of major molecular remission with Nilotinib treatment and the duration of the responses.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Activity and Risk Profile of Ponatinib in Chronic Phase Patients With Chronic Myeloid Leukemia Resistant to Imatinib	NCT02398825	Chronic Myeloid... Chronic Phase Adults	Ponatinib	18 Years -	Gruppo Italiano Malattie EMatologiche dell'Adulto
Nilotinib, for Patients With CML-CP or CML-AP	NCT04518644	Chronic Myeloge...	Nilotinib	18 Years - 99 Years	Novartis
Activity and Risk Profile of Ponatinib in Chronic Phase Patients With Chronic Myeloid Leukemia Resistant to Imatinib	NCT02398825	Chronic Myeloid... Chronic Phase Adults	Ponatinib	18 Years -	Gruppo Italiano Malattie EMatologiche dell'Adulto
Observational Study in Adults With Imatinib-resistant/Intolerant Chronic Myeloid Leukemia Treated With Nilotinib	NCT01475110	Chronic Myeloid...	Imatinib	18 Years -	Gruppo Italiano Malattie EMatologiche dell'Adulto
Nilotinib, for Patients With CML-CP or CML-AP	NCT04518644	Chronic Myeloge...	Nilotinib	18 Years - 99 Years	Novartis
Activity and Risk Profile of Ponatinib in Chronic Phase Patients With Chronic Myeloid Leukemia Resistant to Imatinib	NCT02398825	Chronic Myeloid... Chronic Phase Adults	Ponatinib	18 Years -	Gruppo Italiano Malattie EMatologiche dell'Adulto

Spots Global Cancer Trial Database for imatinib resistant