CLINICAL TRIALS PROFILE FOR SOTRET

All Clinical Trials for Sotret

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00005576 ↗	Monoclonal Antibody Therapy With Sargramostim and Interleukin-2 in Treating Children With Neuroblastoma	Completed	National Cancer Institute (NCI)	Phase 1	2001-01-01	Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood. Interleukin-2 may stimulate a person's white blood cells to kill cancer cells. Combining monoclonal antibody therapy with sargramostim or interleukin-2 may kill more tumor cells. Phase I trial to study the effectiveness of monoclonal antibody therapy given with sargramostim and interleukin-2 in treating children with neuroblastoma who have just completed bone marrow or peripheral stem cell transplantation
NCT00025038 ↗	Combination Chemotherapy Followed By Donor Bone Marrow or Umbilical Cord Blood Transplant in Treating Children With Newly Diagnosed Juvenile Myelomonocytic Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	2001-06-01	Giving chemotherapy drugs, such as R115777, isotretinoin, cytarabine, and fludarabine, before a donor bone marrow transplant or an umbilical cord transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. This phase II trial is studying how well giving combination chemotherapy together with donor bone marrow or umbilical cord blood transplant works in treating children with newly diagnosed juvenile myelomonocytic leukemia
NCT00026312 ↗	Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2001-10-18	This partially randomized phase III trial studies isotretinoin with dinutuximab, aldesleukin, and sargramostim to see how well it works compared to isotretinoin alone following stem cell transplant in treating patients with neuroblastoma. Drugs used in chemotherapy, such as isotretinoin, 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. Monoclonal antibodies, such as dinutuximab, may block tumor growth in different ways by targeting certain cells. Aldesleukin and sargramostim may stimulate a person's white blood cells to kill cancer cells. It is not yet known if chemotherapy is more effective with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating neuroblastoma.
NCT00098891 ↗	MS-275 and Isotretinoin in Treating Patients With Metastatic or Advanced Solid Tumors or Lymphomas	Completed	National Cancer Institute (NCI)	Phase 1	2004-10-01	Phase I trial to study the effectiveness of combining MS-275 with isotretinoin in treating patients who have metastatic or advanced solid tumors or lymphomas. MS-275 may stop the growth of cancer cells by blocking the enzymes necessary for their growth. Isotretinoin may help cancer cells develop into normal cells. MS-275 may increase the effectiveness of isotretinoin by making cancer cells more sensitive to the drug. MS-275 and isotretinoin may also stop the growth of solid tumors or lymphomas by stopping blood flow to the cancer. Combining MS-275 with isotretinoin may kill more cancer cells

Clinical Trial Conditions for Sotret

Condition Name

Condition Name for Sotret
Intervention	Trials
Recurrent Neuroblastoma	8
Regional Neuroblastoma	6
Stage 4 Neuroblastoma	5
Stage 4S Neuroblastoma	5

Condition MeSH

Condition MeSH for Sotret
Intervention	Trials
Neuroblastoma	14
Ganglioneuroblastoma	5
Neuroectodermal Tumors, Primitive	3
Leukemia	3

Clinical Trial Locations for Sotret

Trials by Country

Trials by Country for Sotret
Location	Trials
United States	402
Canada	44
Australia	20
New Zealand	6
Puerto Rico	5

Trials by US State

Trials by US State for Sotret
Location	Trials
California	17
Pennsylvania	16
Illinois	13
Texas	13
Ohio	13

Clinical Trial Progress for Sotret

Clinical Trial Phase

Clinical Trial Phase for Sotret
Clinical Trial Phase	Trials
Phase 3	7
Phase 2	4
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for Sotret
Clinical Trial Phase	Trials
Completed	9
Active, not recruiting	7
Recruiting	3

Clinical Trial Sponsors for Sotret

Sponsor Name

Sponsor Name for Sotret
Sponsor	Trials
National Cancer Institute (NCI)	21
Children's Oncology Group	7
Comprehensive Cancer Center of Wake Forest University	1

Sponsor Type

Sponsor Type for Sotret
Sponsor	Trials
NIH	21
Other	9

Sotatercept: A Groundbreaking Treatment for Pulmonary Arterial Hypertension (PAH) - Clinical Trials, Market Analysis, and Projections

Introduction to Sotatercept

Sotatercept, an investigational activin receptor type IIA-Fc (ActRIIA-Fc) fusion protein, has been making significant waves in the medical community, particularly in the treatment of pulmonary arterial hypertension (PAH). This drug is the first to target the root cause of PAH, offering a new hope for patients suffering from this debilitating condition.

Clinical Trials Overview

The pivotal Phase 3 STELLAR trial has been a cornerstone in the clinical development of sotatercept. Here are the key findings:

Primary Efficacy Outcome

• The STELLAR trial demonstrated a statistically significant and clinically meaningful improvement in the 6-minute walk distance (6MWD) from baseline at 24 weeks when sotatercept was added to stable background therapy[1].

Secondary Efficacy Outcomes

• Eight out of nine secondary efficacy outcome measures achieved statistical significance. These included improvements in NT-pro-B-type natriuretic peptide (NT-proBNP) levels, World Health Organization Functional Class (WHO FC), and time to death or the first occurrence of a clinical worsening event (TTCW)[1].

Safety Profile

• The overall safety profile of sotatercept in the STELLAR trial was consistent with what has been observed in Phase 2 trials. This suggests a favorable safety profile for the drug when used as an add-on to current therapies[1].

Impact on PAH Treatment

Targeting the Root Cause

• Sotatercept is unique because it targets the underlying pathophysiology of PAH, making it a potential game-changer in the treatment landscape. Unlike existing therapies that primarily manage symptoms, sotatercept addresses the root cause of the disease[4].

Additional Benefit on Existing Therapies

• The majority of patients in the clinical trials were already on significant therapy packages, yet sotatercept showed additional benefit. This indicates that sotatercept can enhance the efficacy of current treatments, offering improved outcomes for patients with PAH[4].

Market Analysis and Projections

Clinical Trials Market Growth

• The clinical trials market, which includes the development and testing of novel drugs like sotatercept, is expected to grow significantly. The market size is projected to increase from USD 52.92 billion in 2024 to USD 120 billion by 2037, with a compound annual growth rate (CAGR) of around 6.5% during the forecast period[3].

Pharmaceutical Industry Trends

• The rising adoption and development of novel drugs and therapies are driving the growth of the clinical trials market. For instance, around 50 novel drugs were approved by the Center for Drug Evaluation and Research (CDER) in recent years, indicating a robust pipeline of new treatments[3].

Regulatory Approval and Market Entry

• Although sotatercept has not yet been licensed anywhere in the world, the positive results from the STELLAR trial are expected to expedite its regulatory approval. Once approved, sotatercept is likely to become a significant player in the PAH treatment market, given its demonstrated efficacy and safety[4].

Economic and Market Outlook

Global Economic Context

• The global economic outlook for 2025 suggests moderate growth, with most economies expected to avoid recession. This stable economic environment could support the development and commercialization of new drugs like sotatercept[5].

Stock Market Performance

• Wall Street predictions indicate continued growth in the stock market, albeit at a slower pace than in previous years. The average 2025 year-end price target for the S&P 500 is around 6,539, representing a potential gain of about 8% from current levels. This positive market outlook could favor pharmaceutical companies investing in innovative treatments[2].

Expert Insights and Quotes

Dr. Dean Y. Li, President, Merck Research Laboratories

• "In the Phase 3 STELLAR study, sotatercept added to currently approved background therapy showed a profound effect on the primary efficacy outcome measure of improvement from baseline to 24 weeks in six-minute walk distance. The results from the secondary efficacy outcomes, including a favorable benefit seen in patients’ time to a clinical-worsening event, are especially noteworthy," highlighting the potential of sotatercept to transform PAH treatment[1].

Key Takeaways

• Clinical Efficacy: Sotatercept has demonstrated significant improvements in exercise capacity and key secondary outcome measures in the STELLAR trial.
• Market Potential: The drug is expected to become a major player in the PAH treatment market once approved, given its unique mechanism of action and positive clinical trial results.
• Economic Context: A stable global economic outlook and positive stock market predictions support the development and commercialization of innovative treatments like sotatercept.
• Regulatory Approval: While not yet licensed, the positive trial results are expected to expedite regulatory approval, bringing this therapy to patients sooner.

FAQs

Q: What is sotatercept and how does it work?

• Sotatercept is an investigational activin receptor type IIA-Fc (ActRIIA-Fc) fusion protein that targets the root cause of pulmonary arterial hypertension (PAH) by inhibiting the activin receptor type IIA.

Q: What were the key findings of the STELLAR trial?

• The STELLAR trial showed a statistically significant improvement in 6-minute walk distance and several secondary efficacy outcomes, including improvements in NT-proBNP levels and time to clinical worsening events.

Q: Is sotatercept safe to use?

• The safety profile of sotatercept in the STELLAR trial was consistent with Phase 2 observations, indicating a favorable safety profile when used as an add-on to current therapies.

Q: When is sotatercept expected to be approved?

• While not yet licensed, the positive results from the STELLAR trial are expected to expedite regulatory approval. However, the exact timeline for approval is not specified.

Q: How does sotatercept fit into the current PAH treatment landscape?

• Sotatercept offers additional benefit on top of existing therapies, making it a potential game-changer in the treatment of PAH by addressing the underlying pathophysiology of the disease.

Sources

1. Merck Announces Positive Top-line Results from Pivotal Phase 3 STELLAR Trial Evaluating Sotatercept for the Treatment of Adults with Pulmonary Arterial Hypertension (PAH). Merck.
2. Here's a Complete Rundown of Wall Street's 2025 Stock Market Predictions. Business Insider.
3. Clinical Trials Market Size & Share, Growth Trends 2037. Research Nester.
4. An update on sotatercept, December 2023. PHA UK.
5. 2025 Global Outlook: Clearing the Hurdles. Charles Schwab.

Last updated: 2025-01-03