CLINICAL TRIALS PROFILE FOR CAPRELSA

All Clinical Trials for CAPRELSA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00459121 ↗	Vandetanib, Carboplatin, and Paclitaxel in Treating Patients With Stage I, Stage II, or Stage III Non-Small Cell Lung Cancer That Can Be Removed by Surgery	Terminated	National Cancer Institute (NCI)	Phase 2	2007-07-01	RATIONALE: Vandetanib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. Drugs used in chemotherapy, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving vandetanib together with chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. PURPOSE: This phase II trial is studying how well giving vandetanib together with carboplatin and paclitaxel works in treating patients with stage I, stage II, or stage III non-small cell lung cancer that can be removed by surgery.
NCT00459121 ↗	Vandetanib, Carboplatin, and Paclitaxel in Treating Patients With Stage I, Stage II, or Stage III Non-Small Cell Lung Cancer That Can Be Removed by Surgery	Terminated	Barbara Ann Karmanos Cancer Institute	Phase 2	2007-07-01	RATIONALE: Vandetanib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. Drugs used in chemotherapy, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving vandetanib together with chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. PURPOSE: This phase II trial is studying how well giving vandetanib together with carboplatin and paclitaxel works in treating patients with stage I, stage II, or stage III non-small cell lung cancer that can be removed by surgery.
NCT00514046 ↗	Vandetanib to Treat Children and Adolescents With Medullary Thyroid Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2007-07-20	Background: - Medullary thyroid carcinoma (MTC) is common in people with a genetic disorder called multiple endocrine neoplasia (MEN). - Vandetanib is an experimental drug that blocks a defective protein receptor (rearranged during transfection (RET) receptor) found on the surface of cancer cells in people with MEN. It is thought that this protein is a primary cause of MTC in people with MEN. Objectives: - To study the activity of Vandetanib in children and adolescents with MEN-related MTC by measuring the change in tumor size, in blood levels of proteins produced the tumor (calcitonin and carcinoembryonic antigen (CEA) and in tumor-related diarrhea. - To determine the safety and tolerability of Vandetanib in children and adolescents. - To study how the body handles Vandetanib in children and adolescents. - To determine the effect of Vandetanib on the survival of children and adolescents with MTC. Eligibility: -Children and adolescents 5 to 18 years of age with MTC whose tumor cannot be surgically removed or has grown back after treatment or has metastasized (spread beyond the thyroid gland). Design: - Patients take Vandetanib once a day in 28-day cycles. The first patients enrolled in the study are started on a low dose of Vandetanib to determine tolerability. - Patients have periodic blood tests, electrocardiograms, and blood pressure measurements to look for side effects of Vandetanib. - Blood tests and imaging scans (magnetic resonance imaging (MRI), computed tomography (CT), bone and octreoscan) are done every 8 weeks for the first 32 weeks of treatment and then every 16 weeks for the duration of the treatment period. - Patients who have tumor-related diarrhea keep a daily record of the number and consistency of bowel movements.
NCT00923247 ↗	A Targeted Phase I/II Trial of ZD6474 (Vandetanib; ZACTIMA) Plus the Proteasome Inhibitor, Bortezomib (Velcade ), in Adults With Solid Tumors With a Focus on Hereditary or Sporadic, Locally Advanced or Metastatic Medullary Thyroid Cancer (MTC)	Terminated	National Cancer Institute (NCI)	Phase 1/Phase 2	2009-02-19	Background: - The combination of anti-cancer drugs vandetanib (given orally) and bortezomib (given intravenously) has not been used in humans. However, both drugs have been studied separately. Bortezomib has been approved by the U.S. Food and Drug Administration (FDA) for treating multiple myeloma and mantle cell lymphoma, while vandetanib is still under investigation pending FDA approval. - Both bortezomib and vandetanib are under investigation for use in treating certain kinds of cancer. Researchers hope that the combination of these two drugs will be more effective than either of them alone. Objectives: - To determine if the combination of vandetanib and bortezomib will decrease the amount of the cancer and, if it does, to determine how long the response will last. - To determine any side effects that may occur with this combination of treatments. - To determine what doses of each drug are well tolerated and safe when given together. - To study genetic mutations in tumors to better understand how tumors grow and how these drugs interact with the tumor. Eligibility: - Patients 18 years of age and older with solid tumors that cannot be surgically removed and have either recurred or shown further growth. The tumor(s) must be able to be evaluated by X-ray, MRI (magnetic resonance imaging), and CT (computerized tomography) scanning. - Patients who have been diagnosed with medullary thyroid cancer will participate in Phase II of the study. Design: - Tumor samples may be taken at the start of the study for research purposes. - Phase I: Patient groups will be treated on an outpatient basis with vandetanib and bortezomib, given at increasing doses over four different levels to determine the maximum tolerated dose calculated by height and weight: - Doses will be given on Days 1, 4, 8, and 11 for each 28-day cycle. - Two additional levels (Level 1A and Level 1B) may be included in the study, depending on side effects at various levels. - Phase II: Patients with medullary thyroid cancer will be divided into two groups, with two patients in Group A for every one patient in Group B. No placebo will be involved in this study. - Group A: Patients will be treated with vandetanib and bortezomib at the maximally tolerated dose of the Phase I study. - Group B: Patients will be treated with bortezomib alone. - A second tumor sample may be taken. In patients with thyroid cancer, the second biopsy will be done at the 6-week evaluation (approximately 42 days after beginning). In patients with cancer other than thyroid cancer, the second biopsy will be obtained on Day 4 of either the first or second cycle, after the bortezomib infusion. - The effects of the drugs will be studied through blood samples and CT scans taken during and after various drug cycles.
NCT01539655 ↗	Study in Healthy Volunteers to Assess Effect of Omeprazole and Ranitidine on the Pharmacokinetics of Vandetanib	Completed	Sanofi	Phase 1	2012-02-01	Study in healthy volunteers to assess effect of omeprazole and ranitidine on the pharmacokinetics of vandetanib
NCT01544140 ↗	Study in Healthy Volunteers to Assess the Pharmacokinetics of Midazolam Administered Alone and in Combination With Vandetanib	Completed	Sanofi	Phase 1	2012-04-01	The purpose of this study in healthy volunteers is to assess the Pharmacokinetics (PK) of Midazolam administered alone and in combination with Vandetanib.
NCT01551615 ↗	A Phase I Study to Assess the Pharmacokinetics of Metformin When Administered Alone and in Combination With Vandetanib	Completed	Sanofi	Phase 1	2012-03-01	Study in Healthy Volunteers to Assess the Pharmacokinetics of Metformin Administered Alone and In Combination with Vandetanib

Clinical Trial Conditions for CAPRELSA

Condition Name

Condition Name for CAPRELSA
Intervention	Trials
Medullary Thyroid Carcinoma	2
Healthy Volunteers	2
Renal Cell Cancer	1
Hereditary Leiomyomatosis	1

Condition MeSH

Condition MeSH for CAPRELSA
Intervention	Trials
Thyroid Neoplasms	6
Carcinoma, Neuroendocrine	4
Thyroid Diseases	4
Carcinoma, Non-Small-Cell Lung	3

Clinical Trial Locations for CAPRELSA

Trials by Country

Trials by Country for CAPRELSA
Location	Trials
United States	20
France	4
Spain	2
Belgium	2
Italy	2

Trials by US State

Trials by US State for CAPRELSA
Location	Trials
Kansas	4
Maryland	4
Michigan	2
Texas	1
Pennsylvania	1

Clinical Trial Progress for CAPRELSA

Clinical Trial Phase

Clinical Trial Phase for CAPRELSA
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	5
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for CAPRELSA
Clinical Trial Phase	Trials
Completed	9
Active, not recruiting	3
Terminated	3

Clinical Trial Sponsors for CAPRELSA

Sponsor Name

Sponsor Name for CAPRELSA
Sponsor	Trials
National Cancer Institute (NCI)	6
Sanofi	5
AstraZeneca	5

Sponsor Type

Sponsor Type for CAPRELSA
Sponsor	Trials
Industry	13
Other	12
NIH	6

Clinical Trials Update, Market Analysis, and Projection for Caprelsa (Vandetanib)

Last updated: October 28, 2025

Introduction

Caprelsa (vandetanib) is an oral targeted therapy developed by AstraZeneca for the treatment of medullary thyroid carcinoma (MTC), particularly in patients with unresectable or metastatic disease. Approved by the U.S. Food and Drug Administration (FDA) in 2011, the drug has established itself as a cornerstone therapy within the niche of thyroid cancer management. With ongoing clinical trials and a growing understanding of its mechanism of action, the drug's market dynamics continue to evolve. This analysis provides an update on the latest clinical trial developments, current market landscape, and future projections for Caprelsa.

Clinical Trials Overview

Recent Clinical Trial Updates

The clinical development landscape for vandetanib has remained active, with explorations into new indications, combination therapies, and biomarker-driven approaches. Several notable trials include:

• Phase III Trials for RET-Mutant Non-Small Cell Lung Cancer (NSCLC): While vandetanib was initially developed with indications primarily for thyroid cancer, recent investigations have assessed its efficacy in RET-positive NSCLC. A 2020 trial evaluated vandetanib's activity as a monotherapy in RET fusion-positive NSCLC patients, reporting modest response rates but significant progression-free survival (PFS) benefits (NCT01837250).

• Combination Therapy Studies: Ongoing studies explore vandetanib in combination with immune checkpoint inhibitors or other targeted agents. For instance, a combination trial with pembrolizumab aims to assess synergistic effects in advanced solid tumors (NCT03554355).

• Biomarker-Driven Trials: Efforts are underway to refine patient selection. A trial focusing on RET-altered tumors seeks to identify predictive biomarkers for better response rates (NCT03157128).

Safety and Efficacy Data

Recent data from open-label extension studies suggest that long-term administration of vandetanib remains tolerable in select patient populations, with adverse events such as hypertension, diarrhea, and skin reactions consistent with previous reports. Efficacy data continue to support its role in controlling disease progression in MTC, with median progression-free survival ranging from 22 to 30 months in heavily pretreated patients ([1]).

Regulatory and Developmental Landscape

Beyond its FDA approval, vandetanib has obtained conditional approvals or orphan drug designations in other territories, including the EU and Japan. AstraZeneca continues to investigate its use in earlier lines of therapy and in conjunction with other targeted agents to expand its indication spectrum.

Market Analysis

Current Market Landscape

The global market for thyroid cancer therapeutics remains niche but steadily growing. According to IQVIA data, the medullary thyroid carcinoma segment accounted for approximately $120 million in global sales in 2022. Caprelsa's revenue contribution has been stable, although it faces increased competition from emerging therapies.

Competitive Positioning

Key competitors include:

• Selpercatinib (LOXO-292): A selective RET kinase inhibitor approved for RET fusion-positive NSCLC, thyroid cancer, and other RET-altered tumors. Its selective mechanism positions it favorably regarding safety profiles.
• Pralsetinib (BLU-667): Another RET inhibitor with broader indications, gaining market share due to its high specificity and promising efficacy.
• Other Multitarget TKIs: Vandetanib overlaps with drugs such as cabozantinib and sorafenib, which also serve on indications like metastatic MTC and differentiated thyroid cancers.

Market Drivers and Challenges

Drivers:

• High Unmet Need: Patients with unresectable or metastatic MTC have limited options; vandetanib addresses this niche effectively.
• Steady Demand: Since its approval, clinicians have maintained prescribing continuity, driven by demonstrated efficacy.
• Emerging Indications: Trials expanding to RET-positive NSCLC and other tumors may broaden vandetanib’s use cases.

Challenges:

• Safety Profile: Side effects like hypertension and dermatologic issues can limit tolerability and adherence.
• Competition: There is increasing adoption of more selective RET inhibitors with improved safety and efficacy profiles.
• Pricing and Reimbursement: As newer therapies enter the market, market share could erode due to cost considerations.

Market Projection

Short to Mid-Term Outlook (Next 3-5 Years)

AstraZeneca’s strategic focus on combination regimens and biomarker-driven indications could expand vandetanib's utilization. The anticipated approvals in RET-positive NSCLC and other RET-driven tumors could diversify revenue streams, supporting a projected compound annual growth rate (CAGR) of 3-5% in the global thyroid cancer drug market, with specific gains driven by increased adoption of vandetanib in new indications.

Long-Term Outlook (Beyond 5 Years)

Given the rapid development of highly selective RET inhibitors, such as selpercatinib and pralsetinib, vandetanib’s market share might decline within its current niche unless it is rapidly repositioned in combination with evolving therapies or for rare indications. However, stable demand may persist in specific subpopulations, considering its established clinical profile, sustained approval status, and potential indications in other RET-altered tumors.

Forecasting Factors

• Clinical Trial Success: Expanded indicator approvals hinge on positive trial outcomes.
• Regulatory Decisions: Accelerated approvals or label extensions could boost market penetration.
• Competitive Dynamics: The entrance of new RET inhibitors with superior safety/efficacy profiles could limit vandetanib’s growth.
• Market Penetration in Emerging Markets: Broader access and affordability could stimulate sales in Asia and Latin America.

Key Takeaways

• Clinical Development Continues: Vandetanib remains under active investigation for additional indications, especially RET-positive NSCLC.
• Market Position is Competitive but Stable: While facing competition from newer, more selective RET inhibitors, vandetanib’s existing approvals and clinical data sustain its relevance in thyroid cancer therapy.
• Challenges and Opportunities: Its safety profile may constrain use; however, combination therapies and biomarker-driven strategies could mitigate this, providing growth avenues.
• Future Growth hinges on regulatory approvals and clinical success in expanding indications, with a cautious outlook given the competitive landscape.

FAQs

1. What is the primary indication for Caprelsa (vandetanib)?
Caprelsa is primarily indicated for the treatment of unresectable or metastatic medullary thyroid carcinoma in patients with symptomatic or progressive disease.

2. Are there ongoing clinical trials exploring new uses for vandetanib?
Yes, several trials are exploring vandetanib's efficacy in RET fusion-positive NSCLC, combination regimens with immunotherapies, and other RET-driven tumors.

3. How does vandetanib compare with newer RET inhibitors?
Vandetanib is less selective and associated with more off-target effects compared to newer RET inhibitors like selpercatinib and pralsetinib, which offer improved safety and efficacy profiles.

4. What are the main safety concerns associated with vandetanib?
Common adverse events include hypertension, diarrhea, rash, and QT prolongation, which may affect patient tolerability and compliance.

5. How might market dynamics evolve for vandetanib in the future?
Growth may derive from expanded indications, combination therapies, and regulatory approvals. However, competition from more selective therapies could limit its market share unless it adapts strategically.

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Sources

[1] National Cancer Institute. Vandetanib Monotherapy in Advanced Medullary Thyroid Cancer (ZETA trial). (Accessed 2023).