Nilotinib ± Peg-IFN for First Line Chronic Phase CML Patients

Study at a glance

From the registry record, last updated by the sponsor on 2 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

Randomized Multicenter Phase III Study Comparing the Rate of Molecular Response 4.5 at 12 Months in Newly Diagnosed Philadelphia Positive Chronic Phase Chronic Myelogenous Leukemia Patients Receiving Either Frontline Nilotinib 600 mg Daily or Nilotinib 600 mg Daily Combined to Pegylated Interferon-alfa 2a (Peg-IFN)

ClinicalTrials.gov ID

NCT02201459

Acronym / sponsor ID

PETALs / 2013.837

Conditions

Chronic Myeloid Leukemia

Keywords

Peg-IFN, nilotinib, MR4.5, CML, chronic phase, first-line

Phase

Phase 3

Study type

Interventional

Recruitment status

Completed

Enrolment

200 participants (estimated)

Lead sponsor

Hospices Civils de LyonUniversity, hospital or other

Collaborators

Novartis

Study start

Aug 2014

Primary completion

Oct 2022 (actual)

Study completion

Oct 2022 (actual)

First posted

28 Jul 2014 (estimated)

Results

Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Randomised

Intervention model

Parallel groups

Masking

None (open label)

Number of arms

2

Who may qualify

Ages

18 to 65 years

Sex

All

Healthy volunteers

Not accepted

Age groups

Adult, Older adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Molecular response (MR) 4.5 at 12 months of nilotinib 300 mg twice a day versus a combination of low-dose Peg-Interferon (Peg-IFN) to nilotinib 300 mg twice a day in newly diagnosed CP-CML Chronic Phase Chronic Myelogenous Leukemia patients.

   Time frame: 12 months

   Centralised assessment of the BCR-ABL transcripts at 12 months since nilotinib initiation

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2025.
• Results publicationAbstract We evaluated whether adding pegylated interferon-α2a (Peg-IFNα2a) to nilotinib affected dose intensity, molecular response kinetics, and long-term outcomes in newly diagnosed chronic myeloid leukemia. Delivered nilotinib doses remained comparable between treatment arms up to 72 months, indicating no dose reduction from Peg-IFNα2a-related toxicity. At diagnosis, 8.5 % of 199 patients had additional cytogenetic abnormalities (ACAs). At 3 months, complete and partial cytogenetic response (CCyR/PCyR) rates did not differ between nilotinib alone and the combination (CCyR 72.5 % vs 76 %; PCyR 16.5 % vs 11.5 %). Molecular kinetics showed faster early BCR::ABL1 transcript decline with the combination, but cumulative incidence (CI) curves for major molecular response (MMR) converged by 36 months. Two-year CI of MMR was 80.5 % with nilotinib and 91 % with the combination; five-year CI 93 % vs 97 % (global p = 0.155). The primary endpoint, MR4.5 at 12 months, was reached in 15 % vs 24 % (p = 0.048), but long-term deep molecular response rates (MR4/MR4.5) were ultimately similar at 5 years. In exploratory analyses, female sex (HR 3.06) and higher cumulative Peg-IFNα2a dose in the first 9 months (HR 2.89) predicted early MR4.5, whereas high Sokal or ELTS scores and elevated BCR::ABL1 at month 3 were adverse. ABL1 kinase domain mutations emerged in 10 patients overall (8 nilotinib, 2 combination). Conclusion Peg-IFNα2a with nilotinib accelerated early molecular responses and increased 12-month MR4.5 rates without impairing nilotinib exposure or long-term outcomes. Female sex and Peg-IFNα2a dose intensity correlated with deep early response, supporting potential personalization of combination strategies.
• Derived from this study: PubMed 42069411Nicolini FE, Etienne G, Huguet F, Charbonnier A, Roth-Guepin G, Escoffre-Barbe M, Dubruille V, Johnson-Ansah H, Rousselot P, Legros L, Parry A, Roy L, Coiteux V, Lenain P, Ianotto JC, Doublet C, Orvain C, Simonet-Boissard M, Chretien ML, Penot A, Meunier M, Ame S, Hermet E, Quittet P, Lapusan S, Schwiertz V, Cayuela JM, Maute C, Rea D, Morisset S, Mahon FX, Dulucq S. Final results of nilotinib versus nilotinib combined with pegylated interferon alfa-2a as first-line therapy in chronic phase chronic myeloid leukaemia in France (PETALs): an open-label, multicentre, randomised phase 3 trial. Lancet Haematol. 2026 May;13(5):e315-e326. doi: 10.1016/S2352-3026(26)00043-8.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

1 site in 1 country: see every site and its status.