Chronic Myelogenous Leukemia (CML)NCT04518644
Nilotinib, for Patients With CML-CP or CML-AP
A study testing Nilotinib in people with Chronic Myelogenous Leukemia (CML), run by Novartis Pharmaceuticals.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Chronic Myelogenous Leukemia (CML)
- What is being tested?
- Nilotinib
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years
- One of the diagnoses listed below
- Imatinib resistant Philadelphia chromosome positive CML in chronic phase and the presence of the following criteria:
- < 15% blasts in peripheral blood or bone marrow
- < 30% blasts plus promyelocytes in peripheral blood and bone marrow
- < 20 % basophils in the peripheral blood
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- ≥ 100 x 10^9 / L (≥ 100,000/mm3) platelets
- No evidence of extramedullary leukemic involvement, with the exception of liver or spleen
- Imatinib resistant Philadelphia chromosome positive CML in accelerated phase defined as never in blast crisis before starting treatment with one or more of the following criteria present within 4 weeks prior to beginning treatment:
- ≥ 15% but < 30% blasts in blood or bone marrow
- ≥ 30% blasts plus promyelocytes in peripheral blood or bone marrow (providing that <30% blasts present in bone marrow)
- peripheral basophils ≥ 20%
- thrombocytopenia < 100 x 10^9 / L unrelated to therapy
- WHO Performance status of 0, 1, or 2
- Imatinib must be discontinued at least 5 days prior to beginning nilotinib therapy
- Normal organ, electrolyte and marrow functions as described below:
- Potassium ≥ LLN (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of investigational medication Total calcium (corrected for serum albumin) ≥ LLN or correctable with supplements Magnesium ≥ LLN or corrected to within normal limits with supplements prior to the first dose of investigational medication AST and ALT ≤ 2.5 x ULN or ≤ 5.0 x ULN if considered due to tumor Alkaline phosphatase ≤ 2.5 x ULN unless considered due to tumor Serum bilirubin ≤ 1.5 x ULN Serum amylase and lipase ≤ 1.5 x ULN Serum creatinine ≤ 1.5 x ULN or 24-hour creatinine clearance ≥50 ml/min (calculated creatinine clearance using Cockcroft formula is acceptable) Serum phosphorous ≥ LLN or corrected to within normal limits with supplements prior to the first dose of nilotinib medication Written patient informed consent must be obtained prior to start of treatment. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness. An acceptable alternative (e.g., an assent form, or consent from a parent or guardian) should be signed for legally incompetent patients (e.g., children).
Who cannot join
- History of hypersensitivity to any drugs or metabolites of similar chemical classes as nilotinib.
- Previous treatment with any cytotoxic investigational drug ≤4 weeks prior to beginning nilotinib. At least 2 weeks must have elapsed since the last dose of hormonal therapy or any approved or investigational "targeted" kinase inhibitor agent with the exception of imatinib which must be discontinued at least 5 days prior to beginning therapy with nilotinib.
- Impaired cardiac function, including any one of the following:
- Inability to determine the QT interval on ECG Complete left bundle branch block Long QT syndrome or family history of long QT syndrome History of or presence of significant ventricular or atrial tachyarrhythmias Clinically significant resting brachycardia (<50 beats per minute) QTc > 450 msec on baseline ECG (using the QTcF formula). If QTc >450 and electrolytes are not within normal ranges, electrolytes should be corrected and then the patient rescreened for QTc Myocardial infarction within 12 months prior to starting nilotinib Other clinically significant heart disease (e.g. unstable angina, congestive heart failure or uncontrolled hypertension).
- any medications that prolong the QT interval and CYP3A4 inhibitors if the treatment cannot Use of be either safely discontinued or switched to a different medication prior to starting treatment. Please see http://crediblemeds.org/index.php for a comprehensive list of agents that prolong the QT interval
- Severe and/ or uncontrolled concurrent medical disease that in the opinion of the treating physicians could cause unacceptable safety risks or compromise compliance with the compassionate use treatment (e.g. impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of nilotinib, ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection, uncontrolled diabetes, )
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- Patients who have undergone major surgery ≤ 2 weeks prior to Visit 1 or who have not recovered from side effects of such surgery
- Known Cytopathologically confirmed Central Nervous System infiltration.
- Use of therapeutic warfarin.
- Acute or chronic liver or renal disease considered unrelated to tumor.
- Treatment with any hematopoietic colony-stimulating growth factors ≤ 1 week prior to starting Nilotinib. Erythropoietin is allowed.
- Patient who has not recovered from side effects of prior chemotherapy, immunotherapy, other investigational drugs, wide field radiotherapy, or major surgery. Patient who has received imatinib < 5 days prior to starting nilotinib or has not recovered from side effects of therapy. Hydroxyurea is permitted during the first 28 days of treatment (up to 5 g/day) for a maximum of 7 days.
- Patient with a history of another primary malignancy that is currently clinically significant or requires active intervention.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL).
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods. The two methods can be a double barrier method or a barrier method plus a hormonal method.
- Adequate barrier methods of contraception include: diaphragm, condom (by the partner), intrauterine device (copper or hormonal), sponge or spermicide. Hormonal contraceptives include any marketed contraceptive agent that includes an estrogen and/or a progestational agent.
About this study
Official title: 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
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.
- Conditions
- Chronic Myelogenous Leukemia (CML)
- Being tested
- Nilotinib
- Keywords
- Nilotinib, Imatinib-intolerant, Imatinib resistant, Philadelphia Chromosome positive, Ph+, Chronic Myelogenous Leukemia in Chronic Phase, CML-CP, Chronic Myelogenous Leukemia in Accelerated Phase, CML-AP
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NilotinibDrug
The recommended dosing of nilotinib is 400 mg orally twice daily
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myelogenous Leukemia (CML)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04518644
- Sponsor's protocol ID
- CAMN107A2411
- Status last verified
- Apr 2023
- First submitted
- 14 Aug 2020
- Last updated
- 28 Apr 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04518644
- Acronym / sponsor ID
- CAMN107A2411
- Conditions
- Chronic Myelogenous Leukemia (CML)
- Keywords
- Nilotinib, Imatinib-intolerant, Imatinib resistant, Philadelphia Chromosome positive, Ph+, Chronic Myelogenous Leukemia in Chronic Phase, CML-CP, Chronic Myelogenous Leukemia in Accelerated Phase
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 19 Aug 2020 (actual)
- 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.
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.
Study design
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Age ≥ 18 years
- One of the diagnoses listed below
- Potassium ≥ LLN (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of investigational medication Total calcium (corrected for serum albumin) ≥ LLN or correctable with supplements Magnesium ≥ LLN or corrected to within normal limits with supplements prior to the first dose of investigational medication AST and ALT ≤ 2.5 x ULN or ≤ 5.0 x ULN if considered due to tumor Alkaline phosphatase ≤ 2.5 x ULN unless considered due to tumor Serum bilirubin ≤ 1.5 x ULN Serum amylase and lipase ≤ 1.5 x ULN Serum creatinine ≤ 1.5 x ULN or 24-hour creatinine clearance ≥50 ml/min (calculated creatinine clearance using Cockcroft formula is acceptable) Serum phosphorous ≥ LLN or corrected to within normal limits with supplements prior to the first dose of nilotinib medication Written patient informed consent must be obtained prior to start of treatment. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness. An acceptable alternative (e.g., an assent form, or consent from a parent or guardian) should be signed for legally incompetent patients (e.g., children).
Key exclusion criteria (16 listed)
- History of hypersensitivity to any drugs or metabolites of similar chemical classes as nilotinib.
- Previous treatment with any cytotoxic investigational drug ≤4 weeks prior to beginning nilotinib. At least 2 weeks must have elapsed since the last dose of hormonal therapy or any approved or investigational "targeted" kinase inhibitor agent with the exception of imatinib which must be discontinued at least 5 days prior to beginning therapy with nilotinib.
- Impaired cardiac function, including any one of the following:
- Inability to determine the QT interval on ECG Complete left bundle branch block Long QT syndrome or family history of long QT syndrome History of or presence of significant ventricular or atrial tachyarrhythmias Clinically significant resting brachycardia (<50 beats per minute) QTc > 450 msec on baseline ECG (using the QTcF formula). If QTc >450 and electrolytes are not within normal ranges, electrolytes should be corrected and then the patient rescreened for QTc Myocardial infarction within 12 months prior to starting nilotinib Other clinically significant heart disease (e.g. unstable angina, congestive heart failure or uncontrolled hypertension).
- any medications that prolong the QT interval and CYP3A4 inhibitors if the treatment cannot Use of be either safely discontinued or switched to a different medication prior to starting treatment. Please see http://crediblemeds.org/index.php for a comprehensive list of agents that prolong the QT interval
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NilotinibThe recommended dosing of nilotinib is 400 mg orally twice daily | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Nilotinib in people with Chronic Myelogenous Leukemia (CML), run by Novartis Pharmaceuticals.
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 17 inclusion and 16 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: status not confirmed, as of the sponsor's last update on 28 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
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.
Where can I read the original study information?
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 28 Apr 2023.