Data updated: May 26, 2026
NILOTINIB
Trial Activity: Declining 13 active trials
Oncology
Approved 2025-12-15
Development Insights
Novartis Pharmaceuticals conducting 40 trials (43%)
133 indications explored (Broad Platform)
→ chronic myeloid leukemia (20 trials)
→ chronic myelogenous leukemia (9 trials)
→ gastrointestinal stromal tumors (5 trials)
1
Indication
--
Phase 3 Trials
0
Years on Market
Details
- Status
- None (Tentative Approval)
- First Approved
- 2025-12-15
- PDUFA Date
- 2026-06-18 (23d)
- Routes
- N/A
- Dosage Forms
- CAPSULE
NILOTINIB Approval History
2026
Original
New Indication
New Form
Label Update
1 FDA actions from 2025 to 2025
Dec 2025 ORIGINAL
Update
What NILOTINIB Treats
1 FDA approvalsOriginally approved for its first indication in 2025 .
- Other (1)
Other
(1 approval)- • Approved indication (Dec 2025) New
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Clinical Trial Registry
92 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04877522 | CABL001A2001B 2021-000602-17, 2023-507557-16-00 | Ph 4 | recruiting | Asciminib Roll-over Study |
| NCT03784014 MULTISARC | C16-40 2024-514873-22-00, 2017-002851-27 | Ph 3 | completed | Molecular Profiling of Advanced Soft-tissue Sarcomas |
| NCT03578367 results posted | CABL001E2201 2018-001594-24, 2024-515040-23-00 | Ph 2 | completed | Study of Efficacy and Safety of Asciminib in Combination With Imatinib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Who Have Been Previously Treated With Imatinib and Have Not Achieved Deep Molecular Response. |
| NCT04971226 results posted | CABL001J12301 2023-508838-33-00 | Ph 3 | active not recruiting | A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP |
| NCT03516279 | EA9171 NCI-2017-02161, EA9171 | Ph 2 | active not recruiting | Pembrolizumab and Dasatinib, Imatinib Mesylate, or Nilotinib in Treating Patients With Chronic Myeloid Leukemia and Persistently Detectable Minimal Residual Disease |
| NCT03874858 DANTE | CAMN107AIT15 2024-516122-66-00 | Ph 2 | completed | A Study of Full Treatment-free Remission in Patients With Chronic Myeloid Leukemia |
| NCT07493408 ASIM-POST Ph+ | ASCPT-01 | Ph 2 | not yet recruiting | Asciminib & Standard-of-Care Integration in Maintenance Therapy for POST Allogeneic Stem Cell Transplant (Allo-HSCT) of Patient With Ph+ B-ALL or Blastic Transformed CML |
| NCT01207492 results posted | 10-179 YUS23T | Ph 2 | completed | Nilotinib in Patients With Relapsed or Metastatic Pigmented Villonodular Synovitis/Tenosynovial Giant Cell Tumor/Diffuse-Type Giant Cell Tumor |
| NCT05456191 ASC4START | CABL001J12302 2024-510947-71-00 | Ph 3 | active not recruiting | A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) |
| NCT00756509 results posted | CAMN107DDE06 2008-000358-11 | Ph 4 | completed | Treatment of Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumors in First Line With Nilotinib |
| NCT01407198 | 11-072 | Ph 1 | completed | Nilotinib With Radiation for High Risk Chordoma |
| NCT03654768 results posted | S1712 NCI-2017-02066, S1712 | Ph 2 | active not recruiting | Testing the Addition of Ruxolitinib to the Usual Treatment (Tyrosine Kinase Inhibitors) for Chronic Myeloid Leukemia |
| NCT02917720 NAUT | ELN-002 2015-004998-33 | Ph 2 | active not recruiting | 2nd or 3rd TKI-stop After 2 Years Nilotinib Pre-treatment in CML-patients |
| NCT01698905 ENESTop results posted | CAMN107A2408 2012-003186-18 | Ph 2 | completed | Treatment-free Remission After Achieving Sustained MR4.5 on Nilotinib (ENESTop) |
| NCT04838041 | PRO00040685 | Ph 2 | recruiting | Protocol Number: HJKC3-0003. Treatment Free Remission After Asciminib Based Therapy in Chronic Phase Chronic Myeloid Leukemia (CP-CML) Patients Who Relapsed After a Prior Attempt at TKI Discontinuation |
| NCT06409936 PEARL | CML1624 | Ph 2 | recruiting | PEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML |
| NCT04205903 results posted | OSU-18317 NCI-2019-03146, R01CA238946 | Ph 1 | completed | Nilotinib in Preventing Paclitaxel-Induced Peripheral Neuropathy in Patients With Stage I-III Breast Cancer |
| NCT01751425 | 2012-0697 NCI-2018-01797, 2012-0697 | Ph 1 | terminated | Ruxolitinib in Treating Participants With Chronic Myeloid Leukemia With Minimal Residual Disease While on Therapy With Tyrosine Kinase Inhibitors |
| NCT01804985 DESTINY | 4203 | Ph 2 | completed | De- Escalation and Stopping Treatment of Imatinib, Nilotinib or sprYcel in Chronic Myeloid Leukaemia |
| NCT05185947 | 10000237 000237-C | Ph 2 | active not recruiting | Study of Intravenous and Intraperitoneal Paclitaxel and Oral Nilotinib for Peritoneal Carcinomatosis From Colorectal, Appendiceal, Small Bowel, Gastric, Cholangiocarcinoma, Breast, Ovarian, or Other Gynecologic Primary Cancer |
| NCT04147533 HALF | HALF2019 | Ph 2 | active not recruiting | Efficacy and Safety of TKIs' Withdrawal After a Two-step Dose Reduction in Patients with Chronic Myeloid Leukemia |
| NCT01863745 results posted | CAMN107A1201 | Ph 2 | completed | Nilotinib Roll-over Protocol for Patients in Novartis-sponsored Nilotinib Study and Benefiting From Nilotinib Treatment |
| NCT02326311 | AOBSTMO-OPTKIMA-2014 | Ph 3 | completed | Optimization of TKIs Treatment and Quality of Life in Ph+ CML Patients ≥60 Years in Deep Molecular Response |
| NCT02081378 | CABL001X2101 2013-004491-36 | Ph 1 | completed | A Phase I Study of Oral Asciminib (ABL001) in Patients With CML or Ph+ ALL |
| NCT01735955 results posted | CAMN107A2409 2012-003902-28 | Ph 4 | completed | Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study |
| NCT02925234 DRUP | M15DRU | Ph 2 | recruiting | The Drug Rediscovery Protocol (DRUP Trial) |
| NCT01806571 results posted | MC1284 NCI-2013-00469, 12-006178 | Ph 2 | completed | Daunorubicin Hydrochloride, Cytarabine, and Nilotinib in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT01657604 TIGER | CML V 2010-024262-22 | Ph 3 | completed | TKI and Interferon Alpha Evaluation Initiated by the German Chronic Myeloid Leukemia Study Group - the TIGER Study |
| NCT01810718 Nilo-cGVHD | GITMO Protocol Nilotinib-cGVHD | Ph 1 | completed | Phase I/II for Safety and Efficacy of Nilotinib in a Population Steroid-refractory/or Steroid-dependent cGVHD |
| NCT01025505 LAL1408 | LAL1408 | Ph 2 | completed | Ph+/Bcr-Abl+ ALL Imatinib and Nilotinib Rotational Study |
| NCT01774630 NiloPost-STIM | CHUBX 2012/18 2012-004048-31 | Ph 2 | completed | 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) |
| NCT02546674 NILOdeepR results posted | CAMN107ADE20 2015-000968-34 | Ph 4 | completed | Study Assessing Deep Molecular Response in Adult Patients With CML in Chronic Phase Treated With Nilotinib Firstline. |
| NCT01884922 VINILO | 2012-003005-10 Phase I 2012/1883, 022 | Ph 1 | completed | Study Of Vinblastine in Combination With Nilotinib in Children, Adolescents and Young Adults |
| NCT02353728 results posted | 1403014950 | Ph 2 | completed | Stem Cell Monitoring for CML Patients Undergoing Nilotinib Therapy |
| NCT04835584 | KRT-232-117 | Ph 1, Ph 2 | recruiting | KRT-232 and TKI Study in Chronic Myeloid Leukemia |
| NCT00769327 CML0408 | CML0408 GIMEMA-CML0408, EUDRACT-2008-004384-19 | Ph 2 | completed | Nilotinib and Imatinib Mesylate in Treating Patients With Early Chronic Phase Chronic Myelogenous Leukemia |
| NCT03932669 | 1811-074-985 | Ph 2 | completed | Effect of Nilotinib in Cerebellar Ataxia Patients |
| NCT00644878 MACS0254 results posted | CAMN107AUS09 | Ph 2 | terminated | Study of Molecular Response in Adult Patients on Nilotinib With Philadelphia Chromosome Positive Chronic Myelogenous Leukemia (Ph+ CML) in Chronic Phase and a Suboptimal Molecular Response to Imatinib |
| NCT01743989 ENESTPath results posted | CAMN107AIC05 2012-005124-15 | Ph 3 | completed | A Randomized Phase III Study to Assess the Effect of a Longer Duration of Consolidation Treatment With Nilotinib on TFR in CP CML. |
| NCT00980018 MACS0999 results posted | CAMN107AUS17 | Ph 4 | completed | An Exploratory Trial to Assess the Improvement of Adverse Events in Chronic Myelogenous Leukemia Patients Treated With Imatinib When Switched to Nilotinib Treatment |
| NCT01131325 MACS1148 results posted | CAMN107AUS20 | Ph 4 | terminated | Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations |
| NCT01844765 DIALOG results posted | CAMN107A2203 2013-000200-41 | Ph 2 | completed | Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients. |
| NCT02973711 | UMCC 2015.103 HUM00122063 | Ph 1, Ph 2 | withdrawn | A Study of Ruxolitinib in Combination With Nilotinib in Patients With Chronic Phase CML |
| NCT01702064 | MCC-17114 | Ph 1 | completed | Ruxolitinib in Combination With Nilotinib in Chronic Myeloid Leukemia (CML) Patients |
| NCT01077544 results posted | CAMN107A2120 2010-018419-14 | Ph 1 | completed | A Pharmacokinetic (PK) Study of Nilotinib in Pediatric Patients With Philadelphia Chromosome-positive (Ph+) Chronic Myelogenous Leukemia (CML) or Acute Lymphoblastic Leukemia (ALL) |
| NCT01914484 | CAMN107ACA06T OZM-051 | Ph 1, Ph 2 | completed | Phase I/II Study of Nilotinib/Ruxolitinb Therapy for TKI Resistant Ph-Leukemia |
| NCT01274351 results posted | CAMN107ETR02 | Ph 2 | completed | Study of Nilotinib as First Line Treatment in Philadelphia Chromosome Positive(Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) |
| NCT01528085 | EWALL-PH-02 2010-022855-46 | Ph 2 | completed | Evaluation of Efficacy and Safety of Nilotinib in Combination With Chemotherapy in Elderly Patients With Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia |
| NCT01140568 results posted | 091728 | Ph 2 | completed | Study of Platelet Derived Growth Factor Receptor (PDGFR) in Recurrent Malignant Gliomas |
| NCT01155817 | IRB-18743 SU-06112010-6317, IRB-18743 | Ph 1 | completed | Phase 1 Nilotinib in Steroid Dependent/Refractory Chronic Graft Versus Host Disease |
Showing 50 of 92 trials
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Pro Active Pipeline
Ongoing clinical trials by development phase
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Pro Key Completed Trials
Completed studies with published results, ranked by significance
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Trial Timeline
Full development history with FDA approval milestones
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Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
NILOTINIB FDA Label Details
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment