CLINICAL TRIALS PROFILE FOR CERITINIB

All Clinical Trials for CERITINIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01685060 ↗	LDK378 in Adult Patients With ALK-activated NSCLC Previously Treated With Chemotherapy and Crizotinib	Completed	Novartis Pharmaceuticals	Phase 2	2012-11-26	A single-arm, open-label, multicenter, phase II study. Treatment with LDK378 750 mg qd continued until the patient experienced unacceptable toxicity that precluded further treatment, discontinued treatment at the discretion of the investigator or patient, started a new anti-cancer therapy and/or died. LDK378 could be continued beyond RECIST-defined progressive disease (PD) as assessed by the investigator if, in the judgment of the investigator, there was evidence of clinical benefit. In these patients tumor assessment would continue as per the schedule of assessments until treatment with LDK378 was permanently discontinued. Patients who discontinued the study medication in the absence of progression continued to be followed for tumor assessment until the time of PD as assessed by the investigator
NCT01685138 ↗	LDK378 in Crizotinib naïve Adult Patients With ALK-activated Non-small Cell Lung Cancer	Completed	Novartis Pharmaceuticals	Phase 2	2012-12-20	A single-arm, open-label, two-stage multicenter, phase II study. Patients were pre-screened for ALK positive status. Treatment with LDK378 at 750 mg qd was continued until the patient experienced unacceptable toxicity that precluded further treatment, discontinued treatment at the discretion of the investigator or patient, started a new anticancer therapy and/or died. LDK378 was continued beyond RECIST defined progressive disease (PD) as assessed by the investigator, if in the judgment of the investigator, there was evidence of clinical benefit. Patients who discontinued the study medication in the absence of progression continued to be followed for tumor assessment until the time of PD as assessed by the investigator. Male and female patients aged 18 or over with ALK-rearranged non-small cell cancer (NSCLC) were screened for eligibility. Patients had to have received no prior crizotinib, and had to be chemotherapy-naïve or been pretreated with cytotoxic chemotherapy (up to three prior lines).
NCT01742286 ↗	Phase I Study of LDK378 in Pediatric, Malignancies With a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK)	Completed	Novartis Pharmaceuticals	Phase 1	2013-08-28	The purpose of this study was to estimate the maximum tolerated dose and/or recommended dose for expansion of LDK378 as a single agent, assess safety, tolerability and anti-tumor activity and characterize single and multiple-dose pharmacokinetics when administered orally to pediatric patients with ALK-activated tumors, with and without food.
NCT01828099 ↗	LDK378 Versus Chemotherapy in Previously Untreated Patients With ALK Rearranged Non-small Cell Lung Cancer	Active, not recruiting	Novartis Pharmaceuticals	Phase 3	2013-07-09	The primary purpose of the study was to compare the antitumor activity of LDK378 versus reference chemotherapy. Patients in the chemotherapy arm were allowed to cross-over to LDK378 after confirmed progressive disease (PD).
NCT01828112 ↗	LDK378 Versus Chemotherapy in ALK Rearranged (ALK Positive) Patients Previously Treated With Chemotherapy (Platinum Doublet) and Crizotinib	Active, not recruiting	Novartis Pharmaceuticals	Phase 3	2013-06-28	The primary purpose of the study was to compare the antitumor activity of LDK378 vs. chemotherapy in patients previously treated with chemotherapy (platinum doublet) and crizotinib.
NCT02186821 ↗	Ceritinib (LDK378) for Patients Whose Tumors Have Aberrations in ALK or ROS1 (SIGNATURE)	Terminated	Novartis Pharmaceuticals	Phase 2	2014-09-17	The purpose of this signal seeking study was to determine whether treatment with ceritinib demonstrated sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study.
NCT02227940 ↗	Ceritinib and Combination Chemotherapy in Treating Patients With Advanced Solid Tumors or Locally Advanced or Metastatic Pancreatic Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2015-01-08	This phase I trial studies the side effects and best dose of ceritinib and combination chemotherapy in treating patients with solid tumors that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced) or pancreatic cancer that has spread from where it started to nearby tissue or lymph nodes (locally advanced) or has spread to other places in the body (metastatic). Ceritinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as gemcitabine hydrochloride, paclitaxel albumin-stabilized nanoparticle formulation, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ceritinib and more than one drug (combination chemotherapy) may be a better treatment for solid tumors or pancreatic cancer.

Clinical Trial Conditions for CERITINIB

Condition Name

Condition Name for CERITINIB
Intervention	Trials
Non-Small Cell Lung Cancer	9
ALK Positive	3
Glioblastoma	2
Melanoma	2

Condition MeSH

Condition MeSH for CERITINIB
Intervention	Trials
Carcinoma, Non-Small-Cell Lung	18
Lung Neoplasms	14
Neoplasms	9
Lymphoma	5

Clinical Trial Locations for CERITINIB

Trials by Country

Trials by Country for CERITINIB
Location	Trials
United States	186
Italy	89
Spain	52
Japan	44
Korea, Republic of	28

Trials by US State

Trials by US State for CERITINIB
Location	Trials
California	14
Texas	12
Massachusetts	9
Colorado	8
Florida	8

Clinical Trial Progress for CERITINIB

Clinical Trial Phase

Clinical Trial Phase for CERITINIB
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	3
Phase 4	2

Clinical Trial Status

Clinical Trial Status for CERITINIB
Clinical Trial Phase	Trials
Recruiting	15
Completed	9
Active, not recruiting	6

Clinical Trial Sponsors for CERITINIB

Sponsor Name

Sponsor Name for CERITINIB
Sponsor	Trials
Novartis Pharmaceuticals	17
Novartis	5
National Cancer Institute (NCI)	4

Sponsor Type

Sponsor Type for CERITINIB
Sponsor	Trials
Other	55
Industry	35
NIH	4

Ceritinib: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 30, 2025

Introduction

Ceritinib (brand name: Zykadia) is a targeted oncology therapy developed by Novartis, primarily indicated for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC). Since its approval in 2014, Ceritinib has established itself as a critical option in the treatment of ALK+ NSCLC, especially in cases resistant to prior therapies. This report provides a detailed update on the latest clinical trials, a comprehensive market analysis, and future projections for Ceritinib.

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Clinical Trials Update

Recent Developments and Ongoing Studies

Since the FDA approval, numerous clinical evaluations have sought to expand Ceritinib's therapeutic profile, optimize its efficacy, and mitigate adverse effects.

Key Clinical Trials and Outcomes

1. ASCEND-8 Trial (NCT03155518):

   • Objective: To compare the safety and efficacy of lower-dose Ceritinib (450 mg daily with food) versus the standard 750 mg fasted dose.
   • Outcome: Results showcased comparable efficacy with an improved safety profile, notably reduced gastrointestinal adverse events, reinforcing the current dosing recommendation (450 mg with food) and influencing prescribing practices.

2. ASCEND-5 Trial (NCT02027978):

   • Focus: Ceritinib’s efficacy in patients previously treated with crizotinib.
   • Results: Demonstrated significant objective response rates (~56%) and progression-free survival (PFS) improvements, consolidating Ceritinib’s role as a second-line therapy.

3. ASCEND-4 Trial (NCT02315966):

   • Aim: Comparison of Ceritinib versus chemotherapy in treatment-naïve ALK+ NSCLC patients.
   • Findings: Ceritinib significantly improved PFS, with a median of 16.6 months versus 8.1 months for chemotherapy, leading to its recommendation as first-line treatment in this setting.

4. Combination Trials:

   • Novartis is investigating Ceritinib combined with other agents such as anti-PD-1/PD-L1 immune checkpoint inhibitors (NCT03632535), aiming to enhance therapeutic outcomes for resistant cases.

Safety and Efficacy Optimization

Recent trials have focused on dose adjustments to balance efficacy and tolerability:

• The shift to a 450 mg dose taken with food (via ASCEND-8) is supported by pharmacokinetic studies showing increased bioavailability, reduced gastrointestinal toxicity, and maintained therapeutic efficacy.
• Ongoing research aims to develop next-generation ALK inhibitors with reduced CNS penetration issues, considering Ceritinib’s limited activity regarding brain metastases.

Regulatory and Market Impact

The FDA’s approval of Ceritinib’s new dosing regimen has enhanced its clinical utility, while ongoing phase 3 trials explore broader indications such as ROS1-positive NSCLC and other tumor types.

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Market Analysis

Market Overview and Drivers

The global ALK inhibitor market, valued at approximately USD 1.2 billion in 2022, is projected to grow at a CAGR of 7-8% through 2030 [1]. Ceritinib holds a significant share within this niche, driven by:

• Increasing Incidence of ALK+ NSCLC:
  ALK rearrangements are found in approximately 3–5% of NSCLC cases. With lung cancer accounting for the highest cancer mortality worldwide, the rising prevalence underscores sustained demand for targeted therapies.

• Expanded Indications:
  Beyond second-line treatment, Ceritinib’s potential application in first-line settings and other tumors (e.g., ROS1-positive cancers) complements market growth.

• Regulatory Approvals and Label Expansion:
  The adoption of lower-dose, food-based administration broadens patient tolerability, enhancing adherence and market penetration.

Competitive Landscape

Ceritinib faces competition from several ALK inhibitors, chiefly:

• Alectinib:
  Marketed by Roche, it exhibits superior CNS activity and safety profile and has supplanted Ceritinib as the first-line treatment in many regions [2].

• Brigatinib:
  Approved for ALK+ NSCLC post-crizotinib, noted for effective CNS penetration.

• Lorlatinib:
  The most recent entrant with high CNS activity, suitable for resistant and metastatic cases.

While Ceritinib remains effective, its position is somewhat challenged by these newer agents, especially in frontline therapy.

Market Challenges

Despite its efficacy, Ceritinib’s adverse event profile, notably gastrointestinal toxicity and hepatotoxicity, limits its broader application. The shift towards dose optimization aims to address these challenges and sustain market relevance.

Sales and Revenue Projection

Projections indicate Ceritinib’s global sales could reach USD 500–700 million by 2030, driven by:

• Brand Loyalty and Clinical Efficacy:
  Established as a second-line agent and ongoing trials expanding its indications.

• Competitive Dynamics:
  Market share will depend on pricing, tolerability improvements, and regulatory approvals.

Emerging Opportunities

The evolution of personalized medicine and combination therapies creates new avenues:

• Combination Regimens:
  Synergies with immunotherapies may enhance efficacy, particularly in resistant cases.

• Biomarker-driven Approvals:
  Stratification based on molecular profiles could unlock niche markets and improve trial success.

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Future Projections

Market Growth and Strategic Positioning

The longer-term outlook for Ceritinib hinges on its ability to maintain competitive edge through:

• Ongoing Clinical Trials:
  Positive phase 3 outcomes in first-line settings and broader tumor types will bolster its positioning.

• Formulation Innovations:
  Supporting safer, more tolerable dosing creates opportunities for increased patient acceptance and adherence.

• Expanded Indications:
  Substantially, trials exploring its efficacy in ROS1+ NSCLC and other solid tumors could diversify revenue streams.

Regulatory and Patent Outlook

Patent expirations are anticipated around 2027–2029; however, formulation patents or combination patent protections could extend exclusivity. Regulatory approvals for new indications will likely leverage existing safety data, expediting market access.

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Key Takeaways

• Ceritinib remains a vital second-line treatment for ALK+ NSCLC, with ongoing research solidifying its role in first-line therapy.
• The dosing regimen shift to lower-dose, food-based administration enhances safety, promoting broader acceptance.
• Market competition, especially from alectinib and lorlatinib, challenges Ceritinib’s dominance but also incentivizes innovation.
• Future growth opportunities depend on expanding indications, combination therapies, and new formulation strategies.
• Strategic engagement in clinical trials and regulatory pathways will be critical for maintaining and growing Ceritinib’s market share.

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FAQs

1. What led to the adoption of Ceritinib’s new dosing regimen?
Pharmacokinetic studies demonstrated that 450 mg taken with food achieves plasma concentrations similar to the original 750 mg fasted dose, significantly reducing gastrointestinal toxicity and improving tolerability [3].

2. How does Ceritinib compare with other ALK inhibitors in CNS penetration?
Ceritinib has limited central nervous system (CNS) activity compared to newer agents like alectinib and lorlatinib, which exhibit superior CNS penetration, influencing their preference in treating brain metastases [4].

3. What are the primary adverse effects of Ceritinib?
Common adverse events include gastrointestinal symptoms (nausea, diarrhea, vomiting), hepatotoxicity, and elevated blood glucose. Dose adjustments and supportive care mitigate these effects.

4. Are there ongoing trials investigating Ceritinib’s use in other cancers?
Yes, trials are exploring Ceritinib’s efficacy in ROS1-positive NSCLC and other solid tumors, expanding its therapeutic scope.

5. What will determine Ceritinib’s future market share?
Its success hinges on ongoing clinical trial outcomes, regulatory approvals for new indications, patient tolerability, and competition from next-generation ALK inhibitors.

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References

[1] Global Market Insights. “ALK Inhibitors Market Size & Forecast 2022-2030.” (2022).
[2] Shaw AT, et al. “Alectinib versus Crizotinib in Treatment-Naive ALK-Positive Non-Small-Cell Lung Cancer.” N Engl J Med. 2017; 377(9):829–38.
[3] Novartis. “ASCEND-8 Trial Results.” 2020.
[4] Camidge DR, et al. “The Emerging Role of Next-Generation ALK Inhibitors in Lung Cancer.” Oncologist. 2018; 23(8):974–82.