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Nilotinib+ LDE225

Phase 1

Philadelphia Chromosome Positive Chronic Myelogenous Leukemia | Small molecule | Hematology |Novartis AG|Last Updated: Dec 9, 2020

Target and mechanism

Molecular targetABL1, BCR
Target classInhibitor
ModalitySmall molecule

Also known as Nilotinib, Nilotinib 100mg

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment11

FDA Designations

No designations recorded

Clinical trial landscape

Nilotinib+ LDE225 · 1 trial · 1 indication

Phase 1 1
NCT01456676Nilotinib and LDE225 in the Treatment of Chronic or Accelerated Phase Myeloid Leukemia in Patients Who Developed Resistance to Prior TherapyPhiladelphia Chromosome Positive Chronic Myelogenous Leukemia
COMPLETED11 Analytics
PHASE1COMPLETED
Nilotinib and LDE225 in the Treatment of Chronic or Accelerated Phase Myeloid Leukemia in Patients Who Developed Resistance to Prior Therapy
Philadelphia Chromosome Positive Chronic Myelogenous LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence rate and category of dose limiting toxicities (DLTs) during the first two cycles of therapy
56 days (2 treatment cycles at 28 days each)

Determination of the maximum tolerated dose (MTD) and/or recommended Phase II dose (RP2D) of nilotinib in combination with LDE225

Secondary Endpoints

No of participants with Adverse drug reactions and serious adverse drug reactions, changes in hematology and blood chemistry values, assessments of physical examinations, vital signs and electrocardiograms
336 days (12 treatment cycles)
Plasma concentration and basic pharmacokinetics (PK) parameters (as Cmax, Tmax, AUC)
50 days
Major molecular response (MMR) rates at 3, 6 and 12 months
336 days (12 treatment cycles)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Nilotinib + LDE225EXPERIMENTALThe planned dose of nilotinib 400 mg b.i.d (twice a day) was selected for the combination as this is the dose approved for the treatment of the patient population that will be included in the present study. The starting dose for LDE225 chosen for the current study is 400 mg once daily(q.d.). The maximum dose of LDE225 that will be tested in combination with nilotinib is 800 mg once dail.y

Interventions

NameTypeDescription
Nilotinib + LDE225DRUGNilotinib is an aminopyrimidine ATP-competitive inhibitor of the protein tyrosine kinaseactivity of BCR-ABL.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: 1. Philadelphia chromosome positive (Ph+) CML in chronic phase (CP) or accelerated phase (AP)with resistance to at least one prior BCR-ABL targeting TKI 2. Documented chronic phase CML 3. Adequate end organ function 4. Female patients of childbearing potential must have a negati...

Countries:CanadaFranceGermanyItalySpain
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Frequently asked questions about Nilotinib+ LDE225

What is Nilotinib used for?

Nilotinib is used for Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Chronic Myelogenous Leukemia, Myelogenous Leukemia, Chronic, Gastrointestinal Stromal Tumor (GIST), Chronic Myeloid Leukemia, and Glioma. It is an investigational small molecule in Phase 3 clinical development for these oncology indications.

What does Nilotinib target?

Nilotinib is a kinase inhibitor, belonging to the -tinib class of targeted therapies. It works by inhibiting specific kinases involved in cancer cell growth and survival, particularly those implicated in Philadelphia Chromosome Positive leukemias. The drug is being studied for its activity against these molecular targets in various oncology settings.

Who makes Nilotinib?

Nilotinib is developed by Novartis AG, a global pharmaceutical company listed on the stock exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the safety and efficacy of Nilotinib across multiple cancer indications, including chronic myeloid leukemia and gastrointestinal stromal tumors.

What phase is Nilotinib in?

Nilotinib is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The drug is being studied in multiple oncology indications, with a total of 10 clinical trials conducted, of which 9 are completed and 1 is active but not recruiting.

What clinical trials is Nilotinib in?

Nilotinib has been studied in several clinical trials. Notable ones include NCT01220648, a Phase 1 trial in pretreated Ph+ CML-CP patients; NCT01223898, a Phase 1 drug interaction study; NCT02253277, a Phase 1 combination trial with ruxolitinib; and NCT03906292, an active Phase 2 trial of frontline asciminib combination in chronic phase CML.

Is Nilotinib the same as Nilotinib 300 mg or Nilotinib 100mg?

Yes, Nilotinib 300 mg and Nilotinib 100mg refer to different dosage strengths of the same drug, Nilotinib. These are alternative names used to specify the particular dose formulation being administered in clinical studies or prescriptions. The active pharmaceutical ingredient is identical across these dosage forms.