NCT00481052: CML0307

Nilotinib as First-line Treatment of Ph+ CML in Early Chronic Phase

Completed Phase 2 Last updated 18 January 2022

What this trial tests

Phase 2 trial testing Nilotinib in Chronic Myeloid Leukemia in 74 participants. Completed in 30 April 2018.

Timeline

23 June 2007

Primary endpoint
30 April 2018

30 April 2018

Quick facts

Lead sponsor	Gruppo Italiano Malattie EMatologiche dell'Adulto
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	74
Start date	23 June 2007
Primary completion	30 April 2018
Estimated completion	30 April 2018
Sites	22 locations across Italy

Drugs / interventions tested

• Nilotinib (NILOTINIB) — full drug profile →

Conditions studied

• Chronic Myeloid Leukemia — all drugs for Chronic Myeloid Leukemia →

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto — full company profile →

Who can join

18 and older, any sex, with Chronic Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Treating Ph pos CML with Imatinib is very effective since the majority of the patients achieve a complete cytogenetic response and a major molecular response and are alive and progression-free after 5 years. However, the great majority of responding patients are not leukemia-free and may be at risk of progression, molecular, cytogenetic and clinical, at any time. In case of disease progression due to Imatinib failure, nilotinib has been found to be very effective, as expected from the preclinical profile of the drug, that is much more potent against BCR-ABL and inhibits nearly all the imatinib-resistant BCR-ABL mutants. For these reasons, nilotinib is going to be registered for the treatment of imatinib-resistant CMl patients. For the same reasons, nilotinib is expected to be more efficient than imatinib also front-line, based on the principle that we should aim at preventing the emergence of resistance better that at treating resistance once it has emerged. This expectation can be tested safely, because the "toxicity profile" of Nilotinib may be even more convenient than that of Imatinib, due to the lower frequency of edema and fluid retention.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Nilotinib for the frontline treatment of Ph(+) chronic myeloid leukemia.
   Rosti G, Palandri F, Castagnetti F, Breccia M, et al · · 2009 · cited 142× · PMID 19822896 · DOI 10.1182/blood-2009-07-232595
2. Differences among young adults, adults and elderly chronic myeloid leukemia patients.
   Castagnetti F, Gugliotta G, Baccarani M, Breccia M, et al · · 2015 · cited 66× · PMID 25361995 · DOI 10.1093/annonc/mdu490
3. Treatment-free remission in chronic myeloid leukemia patients treated front-line with nilotinib: 10-year followup of the GIMEMA CML 0307 study.
   Gugliotta G, Castagnetti F, Breccia M, Levato L, et al · · 2022 · cited 17× · PMID 35385922 · DOI 10.3324/haematol.2021.280175
4. Treatment-free remission after discontinuation of tyrosine kinase inhibitors in patients with chronic myeloid leukemia in the chronic phase: a systematic review and meta-analysis.
   Zheng Z, Tang H, Zhang X, Zheng L, et al · · 2024 · cited 4× · PMID 39441428 · DOI 10.1007/s12672-024-01444-9
5. 46th Congress of the Italian Society of Hematology Rome, Italy, October 15–18, 2017
   · 2017
6. 21st Congress of the European Hematology Association Copenhagen, Denmark, June 9–12, 2016
   · 2016
7. 19th Congress of the European Hematology Association, Milan, Italy, June 12–15, 2014
   · 2014
8. 44° Congress of the Italian Society of Hematology Verona, Italy, October 20–23, 2013
   · 2013