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Leukemia, Myelogenous, Chronic, BCR-ABL PositivePhase 2NiloPost-STIMNCT01774630

Multicenter Single-arm Pilot Study Evaluating Efficacy of Nilotinib in CML Patients With Molecular Relapse After Glivec Discontinuation Within the Context of the STIM Trials (STIM and STIM2)

A Phase 2 study testing Nilotinib in people with Leukemia, Myelogenous, Chronic, BCR-ABL Positive, run by University Hospital, Bordeaux at 10 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Chronic myeloid leukemia (CML) is a hematopoietic neoplasm characterized by the reciprocal translocation t(9;22). The resulting oncoprotein, bcr-abl is an essential trigger for growth and survival of leukemic cells. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Nilotinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
31 participants
Where
10 sitesFrance
Study dates
10 Apr 2013to 21 Dec 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male and female patients
  • Patient participating to the STIM trials (including STIM, STIM2 et EURO-SKI) and with confirmed molecular relapse on two consecutive RQ PCR, after imatinib discontinuation
  • Still in chronic phase
  • Not yet treated for this relapse
  • At least 18 years old (no upper age limit)
  • SGOT and SGPT < 2.5 UNL
Show all 10Show fewer
  • Serum creatinin < 2 UNL
  • No planned allogeneic stem cell transplantation
  • Signed informed consent
  • ECOG score 0 to 2

Who cannot join

  • Pregnancy, lactation
  • Prior or concurrent malignancy other than CML (exceptions to be mentioned)
  • Serious uncontrolled cardiovascular disease
  • Severe psychiatric/neurological disease (previous or ongoing)
  • Ongoing treatment at risk for inducing "torsades de pointe"
  • QTcF > 450ms despite correction of predisposing factors (i.e electrolytes…)
  • Congenital long QTcF
  • No health insurance coverage

Trial locations and contacts

10 sites in 1 country.

10 sites in 1 country

France10 sites

  • CHU Angers

    Angers, 49033

  • Institut Bergonié

    Bordeaux, 33076

  • Centre Hospitalier de Versailles - Hôpital André Mignot - Service de Médecine B

    Le Chesnay, 78157

  • CHU de Nice, Service Hématologie Clinique

    Nice, 06202

  • Hôpital Haut Lévêque, Service Hématologie

    Pessac, 33604

  • Centre Hospitalier Lyon Sud, Service Hématologie

    Pierre-Bénite, 69495

  • CH d'Annecy

    Pringy, 74374

  • Hôpital Pontchaillou

    Rennes, 35033

  • CHU de Toulouse, Service d'Hématologie

    Toulouse, 31059

  • CH Valence

    Valence, 26953

Other trials in France

About this study

Chronic myeloid leukemia (CML) is a hematopoietic neoplasm characterized by the reciprocal translocation t(9;22). The resulting oncoprotein, bcr-abl is an essential trigger for growth and survival of leukemic cells. In the past decade, the bcr-abl tyrosine kinase inhibitor (TKI) imatinib (IM or Glivec©) has been the standard of care for patients with CML, inducing durable responses. However, requiring continuing IM indefinitely and the ability of IM to eradicate the CML clone was uncertain.

In a small proportion of patients, IM can induce complete molecular response (CMR) defined by the disappearance of the bcr-abl transcript in conventional quantitative RT-PCR. The question whether or not these patients are cured and can discontinue drug therapy has been assessed by Mahon and coll, in the STIM study. He demonstrates that IM can be safely discontinued in patient with a CMR of at least 2 year duration and all patients who relapsed after IM discontinuation mainly did it in the first 6 months and responded to reintroduction of imatinib.

Read more

Nilotinib is a rationally designed second generation tyrosine kinase inhibitor with improved target specificity over imatinib. Its efficacy and safety in the treatment of patients who are resistant or intolerant to imatinib as well as patients with newly diagnosed CML-CP led to the registration in second and first line treatment of CML-CP patients. Nilotinib produces even faster and deeper responses with more occurrence of CMR than does Imatinib. Consequently, one can assume that a more potent drug such nilotinib could induce deeper and sustained CMR allowing longer period off treatment than IM.

The objective of this pilot trial is to assess if Nilotinib can rescue STIM patients in molecular relapse after IM discontinuation and to provide an estimation about duration of CMR after nilotinib discontinuation in 2nd line therapy among patients experiencing 2 years of stable CMR with nilotinib.

Read the detailed description

Patients with CML included in STIM trials, stopped their treatment by imatinib because the signal was not detectable. In case of reappearance of this transcript Bcr-Abl, the patient relapses. The trial Nilo Post STIM is suggested to the patient to assess if Nilotinib can rescue STIM patients in molecular relapse after IM discontinuation and to provide an estimation about duration of CMR after nilotinib discontinuation in 2nd line therapy among patients experiencing 2 years of stable CMR with nilotinib.

The treatment/strategy for this study:

  • Screening
  • Inclusion/exclusion criteria
  • CML history
  • Confirm molecular relapse after discontinuation of imatinib (quantitative RT-PCR on two consecutive assessments from peripheral blood samples)
  • Treatment
  • Nilotinib 300mg BID for 2 years
  • Premature treatment discontinuation while on study: primary or secondary resistance progression to accelerated phase or blast crisis, AE (to be defined later).
  • In case of unsatisfactory response: transcript stability or increase on two consecutive PCR: nilotinib blood monitoring, and nilotinib dose escalation up to 400mg BID will be proposed
  • Discontinuation at 2 years for patients who resumed confirmed CMR
  • Follow-up while on treatment with nilotinib:
  • Physical exam, basic laboratory parameters, monthly during the first 3 months then every 3 months.
  • Centralized quantitative RT-PCR for Bcr-Abl monthly for 6 months then every 3 months for 24 months
  • Follow AE management guidelines for nilotinib reduction/interruptions
  • Follow-up after nilotinib discontinuation
  • Patients in confirmed molecular relapse
  • Physical exam, event collection, basic laboratory parameters (including glycemic and lipid profile) every 2 months during the first year then every 3 months
  • Hematology and centralized quantitative RT-PCR monthly the first year then every 3 months for 12 months
  • Patients without confirmed molecular relapse will take another treatment (dasatinib for example) and will stop their follow-up in the trial
Being tested
Nilotinib
Keywords
Treatment, relapse, complete molecular response
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NilotinibDrug

    300 mg/twice a day

Study groups in NCT01774630
Study groupRoleWhat they receive
NilotinibExperimental300 mg/twice a day
Receives: Drug: Nilotinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Estimated survival rate of patients without molecular relapse 3 years after enrollment

    CMR is defined as >5 log reduction in Bcr-Abl and Abl levels and undetectable transcripts on quantitative RTq-PCR

    Measured: Evaluation by RTq-PCR monthly the first year of treatment with nilotinib then every 3 months until 24 months, date of discontinuation of Nilotinib for patients in sustained complete molecular response (CMR). After discontinuation of Nilotinib patie

Secondary outcomes (7)

  1. Rate and kinetics of CMR while on treatment with nilotinib

    Same definition of CMR as above

    Measured: at 6 and 12 months of treatment with nilotinib

  2. Duration of CMR while on treatment with nilotinib

    Defined as the time from the date of first documented CMR to the date of first confirmed molecular relapse defined as positivity of Bcr-Abl transcripts in quantitative RT-PCR with a ratio of bcr-abl to Abl ≥ 10-5, as confirmed by a second analysis point at two successive assessments

    Measured: Any time

  3. Event free survival (EFS)

    Events include loss of major molecular response (MMR) , loss of complete cytogenetic response (CCyR) loss of complete hematologic response (CHR), progression to accelerated phase and blst crisis (AP-BC), death whatever the cause, adverse-event leading to premature discontinuation of nilotinib

    Measured: Any time

  4. Safety tolerability of nilotinib and compliance

    Haematological and non-haematological adverse events (AE) graded will be according to the NCI CTC AE v4. Compliance will be estimated using the 4 items Morisky scale

    Measured: Any time

  5. Duration of CMR after nilotinib discontinuation

    Measured: Measured from the start of nilotinib discontinuation to the date of first confirmed molecular relapse as defined above

  6. Event free survival (EFS)

    Same events as for EFS described above

    Measured: Overall and after nilotinib discontinuation

  7. Predictive factors of maintained CMR after nilotinib discontinuation: sex, Sokal risk score at diagnosis, duration of previous treatment with imatinib, CMR duration before and after discontinuation of imatinib

    Parameters will be recorded before and after both sequences of treatment imatinib and nilotinib

    Measured: After discontinuation of nilotinib

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Jan 2013 (estimated)
  2. Study start10 Apr 2013
  3. Primary completion21 Dec 2020
  4. Study completion21 Dec 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
31 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01774630
Sponsor's protocol ID
CHUBX 2012/18
Other IDs
2012-004048-31
Acronym
NiloPost-STIM
Status last verified
Aug 2022
First submitted
22 Jan 2013
Last updated
26 May 2026
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Dulucq S, Rigal-Huguet F, Nicolini FE, Cony-Makhoul P, Escoffre-Barbe M, Gardembas M, Legros L, Rousselot P, Liu J, Rea D, De Mas V, Hayette S, Raynaud S, Lacoste-Roussillon C, Robbesyn F, Klein E, Morisset S, Mahon FX, Etienne G. Efficacy and safety of nilotinib in chronic myeloid leukaemia patients who failed to achieve a treatment-free remission period after imatinib discontinuation: Results of the French Nilo post-STIM study. Br J Haematol. 2023 Jun;201(6):1116-1124. doi: 10.1111/bjh.18796. Epub 2023 Apr 2.

    PubMed 37004981

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Nilotinib in people with Leukemia, Myelogenous, Chronic, BCR-ABL Positive, run by University Hospital, Bordeaux at 10 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

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

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Estimated survival rate of patients without molecular relapse 3 years after enrollment (time frame: Evaluation by RTq-PCR monthly the first year of treatment with nilotinib then every 3 months until 24 months, date of discontinuation of Nilotinib for patients in sustained complete molecular response (CMR). After discontinuation of Nilotinib patie). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01774630, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 May 2026.

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