CLINICAL TRIALS PROFILE FOR LUTATHERA

All Clinical Trials for Lutathera

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01578239 ↗	A Study Comparing Treatment With 177Lu-DOTA0-Tyr3-Octreotate to Octreotide LAR in Patients With Inoperable, Progressive, Somatostatin Receptor Positive Midgut Carcinoid Tumours	Completed	Pierrel Research Europe GmbH	Phase 3	2012-09-06	This was a multicenter, stratified, open, randomized, comparator-controlled, parallel-group phase III study comparing treatment with Lutathera plus best supportive care (30 mg Octreotide LAR) to treatment with high dose (60 mg) Octreotide LAR in participants with metastasized or locally advanced, inoperable, somatostatin receptor positive, histologically proven midgut carcinoid tumours with progression despite LAR treatment.
NCT01578239 ↗	A Study Comparing Treatment With 177Lu-DOTA0-Tyr3-Octreotate to Octreotide LAR in Patients With Inoperable, Progressive, Somatostatin Receptor Positive Midgut Carcinoid Tumours	Completed	Advanced Accelerator Applications	Phase 3	2012-09-06	This was a multicenter, stratified, open, randomized, comparator-controlled, parallel-group phase III study comparing treatment with Lutathera plus best supportive care (30 mg Octreotide LAR) to treatment with high dose (60 mg) Octreotide LAR in participants with metastasized or locally advanced, inoperable, somatostatin receptor positive, histologically proven midgut carcinoid tumours with progression despite LAR treatment.
NCT01876771 ↗	A Trial to Assess the Safety and Effectiveness of Lutetium-177 Octreotate Therapy in Neuroendocrine Tumours	Recruiting	AHS Cancer Control Alberta	Phase 2	2014-04-29	Neuroendocrine tumours (NETs) are rare, slow growing, and diagnosis is often delayed with advanced metastases at presentation. In select patient populations, radioisotope therapy with Lutetium-177 (Lu-DOTA-TATE) has been shown to be a safe and effective palliative therapy, and has been widely used by research groups in Europe. A brand of Lu-DOTA-TATE (Lutathera(R)) is approved for the treatment of gastroenteropancreatic NETs in Europe, the U.S., and more recently in Canada. While Lutathera(R) is approved in Canada, it is not publicly funded in Alberta. Lu-DOTA-TATE has been used at the Cross Cancer Institute to treat more than 300 patients with NETs since August, 2010. Our Lu-DOTA-TATE treatment was initially given under Health Canada's Special Access Programme (SAP), with each individual treatment requiring separate approval. In 2014, Health Canada requested we conduct a clinical trial with Lu-DOTA-TATE instead. The purpose of this study is to: 1) assess the efficacy of Lu-DOTA-TATE treatment in patients with somatostatin receptor positive tumours; 2) assess the safety of Lu-DOTA-TATE; 3) assess the effect of Lu-DOTA-TATE on Quality of Life and survival.
NCT02705313 ↗	EAP 177Lu-DOTA0-Tyr3-Octreotate for Inoperable, SSR+, NETs, Progressive Under SSA Tx	Approved for marketing	Advanced Accelerator Applications		1969-12-31	Advanced Accelerator Applications is currently pursuing marketing approval for 177Lu-DOTA0-Tyr3-Octreotate (Lutathera). This expanded access therapeutic protocol aims to allow patients suffering from inoperable, somatostatin receptor positive, neuroendocrine tumors, progressive under somatostatin analogue therapy to access the investigational product, 177Lu-DOTA0-Tyr3-Octreotate (Lutathera), prior to its commercial availability.
NCT03206060 ↗	Lu-177-DOTATATE (Lutathera) in Therapy of Inoperable Pheochromocytoma/ Paraganglioma	Recruiting	National Cancer Institute (NCI)	Phase 2	2017-10-10	Background: Pheochromocytoma and paraganglioma are rare tumors. They usually form inside and near the adrenal gland or in the neck region. Not all these tumors can be removed with surgery, and there are no good treatments if the disease has spread. Researchers think a new drug may be able to help. Objective: To learn the safety and tolerability of Lu-177-DOTATATE. Also, to see if it improves the length of time it takes for the cancer to return. Eligibility: Adults who have an inoperable tumor of the study cancer that can be detected with Ga-68-DOTATATE PET/CT imaging Design: Participants will be screened with a medical history, physical exam, and blood tests. Eligible participants will be admitted to the NIH Clinical Center. Participants will get the study drug in an intravenous infusion. They will get 4 doses, given about 8 weeks apart. Between 4 and 24 hours after each study drug dose, participants will have scans taken. They will lie on their back on a scanner table. Participants will have vital signs taken. They will give blood and urine samples. During the study, participants will have other scans taken. Some scans will use a radioactive tracer. Participants will complete quality of life questionnaires. Participants will be contacted by phone 1-3 days after they leave the Clinical Center. They will then be followed every 3 to 6 months for 3 years or until their disease gets worse.

Clinical Trial Conditions for Lutathera

Condition Name

Condition Name for Lutathera
Intervention	Trials
Neuroendocrine Tumors	13
Pheochromocytoma	3
Paraganglioma	3
Neuroendocrine Neoplasms	2

Condition MeSH

Condition MeSH for Lutathera
Intervention	Trials
Neuroendocrine Tumors	28
Neoplasms	12
Carcinoma	5
Pheochromocytoma	5

Clinical Trial Locations for Lutathera

Trials by Country

Trials by Country for Lutathera
Location	Trials
United States	80
France	10
Italy	10
Australia	5
Germany	5

Trials by US State

Trials by US State for Lutathera
Location	Trials
Texas	6
Iowa	6
Pennsylvania	5
Ohio	5
Minnesota	5

Clinical Trial Progress for Lutathera

Clinical Trial Phase

Clinical Trial Phase for Lutathera
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	2
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Lutathera
Clinical Trial Phase	Trials
Not yet recruiting	21
Recruiting	15
Withdrawn	2

Clinical Trial Sponsors for Lutathera

Sponsor Name

Sponsor Name for Lutathera
Sponsor	Trials
National Cancer Institute (NCI)	12
Advanced Accelerator Applications	11
Novartis Pharmaceuticals	3

Sponsor Type

Sponsor Type for Lutathera
Sponsor	Trials
Other	33
Industry	22
NIH	13

LUTATHERA: A Breakthrough in Radioligand Therapy for Gastroenteropancreatic Neuroendocrine Tumors

Introduction to LUTATHERA

LUTATHERA, also known as lutetium Lu 177 dotatate, is a radioligand therapy (RLT) developed by Novartis for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs). This therapy has marked significant milestones in clinical trials, received regulatory approvals, and is poised to transform the treatment landscape for patients with GEP-NETs.

Clinical Trials Update

NETTER-2 Trial

The Phase III NETTER-2 trial has been a pivotal study for LUTATHERA. This open-label, multi-center, randomized trial compared the efficacy of LUTATHERA in combination with long-acting octreotide (LAR) versus high-dose octreotide LAR alone as a first-line treatment for patients with somatostatin receptor-positive (SSTR+) Grade 2 and 3 advanced GEP-NETs.

• Primary Endpoint: The trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS) for patients treated with LUTATHERA plus octreotide LAR compared to those receiving high-dose octreotide LAR alone. The risk of disease progression or death was reduced by 72% in the LUTATHERA group[2][4].
• Secondary Endpoints: The trial also achieved key secondary endpoints, including an improved objective response rate (ORR), where tumors were more likely to shrink in patients treated with LUTATHERA[4].
• Safety Profile: The study reported no new or unexpected safety findings, with the data consistent with LUTATHERA’s established safety profile. Common adverse events included nausea, diarrhea, and abdominal pain, with reduced lymphocyte count being the most common grade ≥3 adverse event[2].

NETTER-P Study

For pediatric patients, the NETTER-P study provided crucial data. This study evaluated the pharmacokinetics, dosimetry, and safety of LUTATHERA in pediatric patients aged 12 to 17 years. The results showed that the safety profile of LUTATHERA in pediatric patients was consistent with that observed in adult patients, leading to FDA approval for this age group[3][5].

Regulatory Approvals

Adult Patients

LUTATHERA has been approved by regulatory bodies in several regions for the treatment of adult patients with SSTR+ GEP-NETs. In the United States, it is approved for adults with SSTR+ GEP-NETs, including tumors in the foregut, midgut, and hindgut. Similar approvals have been granted in Europe and Japan[2].

Pediatric Patients

In a significant development, LUTATHERA was approved by the FDA on April 23, 2024, for the treatment of pediatric patients aged 12 years and older with SSTR+ GEP-NETs. This makes LUTATHERA the first therapy specifically approved for pediatric patients with GEP-NETs[3][5].

Market Analysis

Current Market Position

LUTATHERA has established itself as a leading treatment option for GEP-NETs, particularly with its recent approval for pediatric patients. The drug's efficacy and safety profile have been well-documented, and it is part of the emerging field of radioligand therapies that are reshaping cancer care.

Market Projections

• Revenue Growth: With the additional approval for pediatric patients, LUTATHERA is expected to see significant revenue growth. DelveInsight’s analysts predict a blockbuster status for LUTATHERA in the coming years, with revenues projected to be higher than previous years[5].
• Value-Based Pricing: Novartis has adopted a value-based pricing model for LUTATHERA, with a list price (WAC) of $55,896 per dose. Most patients require about four doses, making the total treatment cost substantial but reflective of the drug's clinical value[5].
• Competitive Edge: Novartis holds a competitive edge due to LUTATHERA’s first-mover advantage in the pediatric market. However, the company faces challenges from new rivals and potential generic entrants, necessitating a robust market strategy[5].

Future Directions

Research and Development

Novartis is actively investigating new isotopes, ligands, and combination therapies to extend the use of radioligand therapies beyond GEP-NETs. Potential areas of expansion include prostate, breast, colon, lung, and pancreatic cancers. This ongoing research underscores the potential of RLTs in shaping the future of cancer care[5].

Market Expansion

The success of LUTATHERA is likely to expand the market for neuroendocrine tumor treatments, encouraging further research and development in this area. As more innovative therapies emerge, the therapeutic landscape for GEP-NETs and other cancers is expected to evolve significantly[5].

Patient Impact

Clinical Benefits

LUTATHERA offers significant clinical benefits, particularly in prolonging progression-free survival and reducing the risk of disease progression or death. For patients with recently diagnosed, faster-growing NETs, LUTATHERA in combination with long-acting octreotide provides more time without progression and a higher likelihood of tumor shrinkage[4].

Quality of Life

The therapy's ability to slow down tumor progression and reduce symptoms can significantly improve the quality of life for patients. This is particularly important for pediatric patients, who now have a specifically approved treatment option that aligns with their unique needs[3].

Key Takeaways

• Clinical Efficacy: LUTATHERA has demonstrated statistically significant and clinically meaningful improvements in progression-free survival and objective response rates in patients with SSTR+ GEP-NETs.
• Regulatory Approvals: Approved for adult and pediatric patients with SSTR+ GEP-NETs, marking a significant milestone in cancer treatment.
• Market Potential: Expected to achieve blockbuster status with significant revenue growth, driven by its value-based pricing model and first-mover advantage in the pediatric market.
• Future Directions: Ongoing research aims to expand the use of radioligand therapies to other types of cancers.

FAQs

What is LUTATHERA and how does it work?

LUTATHERA is a radioligand therapy that uses lutetium Lu 177 dotatate to target and destroy cancer cells in gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that express somatostatin receptors.

What are the key findings from the NETTER-2 trial?

The NETTER-2 trial showed that LUTATHERA in combination with long-acting octreotide significantly improved progression-free survival and reduced the risk of disease progression or death by 72% compared to high-dose octreotide alone.

Is LUTATHERA approved for pediatric patients?

Yes, LUTATHERA was approved by the FDA in April 2024 for the treatment of pediatric patients aged 12 years and older with SSTR+ GEP-NETs, making it the first therapy specifically approved for this age group.

What is the safety profile of LUTATHERA?

The safety profile of LUTATHERA is consistent with previous studies, with common adverse events including nausea, diarrhea, and abdominal pain. There were no new or unexpected safety findings in the recent trials.

What are the market projections for LUTATHERA?

LUTATHERA is expected to achieve blockbuster status with significant revenue growth, driven by its approval for pediatric patients and its value-based pricing model.

Sources

1. Novartis Media Release: "Novartis radioligand therapy Lutathera® demonstrated statistically significant and clinically meaningful progression-free survival in first line advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs)".
2. PharmExec: "Novartis' Lutathera Shows Significant Survival Benefit as First-Line Treatment for GEP-NETs".
3. Novartis Media Release: "Novartis radioligand therapy Lutathera® FDA approved as first medicine specifically for pediatric patients with gastroenteropancreatic neuroendocrine tumors".
4. LUTATHERA Official Website: "How LUTATHERA Can Help | Clinical Trial Results".
5. DelveInsight Blog: "LUTATHERA Reshapes Pediatric GEP-NET Treatment Paradigm".

Last updated: 2025-01-05