SP-002 + Vismodegib for Basal Cell Carcinoma

Recruiting at 9 trial locations

Overseen By Stamford Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Stamford Pharmaceuticals, Inc.

Must be taking: Hedgehog inhibitors

Must not be taking: Chemotherapy, Radiation therapy

Disqualifiers: Metastatic BCC, Pregnant, others

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The goal of this clinical trial is to evaluate the efficacy of using SP-002 in participants with locally advanced Basal cell carcinoma. The main question it aims to answer is what the objective response rate for a basal cell carcinoma tumor is following 1 or 3 cycles of SP-002 treatment given as an add-on to hedgehog pathway inhibitor therapy. Researchers will compare the objective response rate for treated Basal cell carcinoma tumors between 3 treatment Arms. * Arm 1 participants will receive daily hedgehog pathway inhibitor, and 3 cycles of SP-002 treatment. * Arm 2 participants will receive daily hedgehog pathway inhibitor, and 1 cycle of SP-002 treatment. * Arm 3 participants will receive daily hedgehog pathway inhibitor only.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop taking your current medications. However, you cannot participate if you are on other anti-tumor therapies or have recently been in another experimental drug study.

Is the treatment SP-002 + Vismodegib safe for humans?

Vismodegib (also known as Erivedge) has been studied for safety in treating advanced basal cell carcinoma. Common side effects include muscle spasms, hair loss, taste changes, weight loss, fatigue, nausea, and more. It is also known to cause birth defects, so it should not be used during pregnancy. ^{1 2 3 4 5}

How does the drug SP-002 + Vismodegib differ from other treatments for basal cell carcinoma?

The combination of SP-002 with Vismodegib is unique because it involves Vismodegib, a drug that targets the Hedgehog signaling pathway, which is crucial in the development of basal cell carcinoma. Vismodegib is specifically used for cases that are advanced and not suitable for surgery or radiation, making it a specialized option for difficult-to-treat cases. ^{2 3 4 6 7}

What data supports the effectiveness of the drug Vismodegib (Erivedge) for treating basal cell carcinoma?

Vismodegib (Erivedge) is approved for treating advanced basal cell carcinoma that cannot be treated with surgery or radiotherapy, and studies have shown it to be effective in real-world settings. It works by blocking a specific pathway (Hedgehog pathway) involved in the growth of these cancer cells. ^{2 3 4 8 9}

Who Is on the Research Team?

Stamford Pharmaceuticals

Principal Investigator

Stamford Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for individuals with locally advanced Basal cell carcinoma. Participants must be suitable for hedgehog pathway inhibitor therapy and willing to undergo additional treatment with SP-002. Specific eligibility details are not provided, but typically include factors like age, health status, and cancer stage.

Inclusion Criteria

Radiotherapy is contraindicated or inappropriate in the opinion of the investigator

I am using two forms of birth control or ensuring my partner does.

I can take care of myself and perform daily activities.

Exclusion Criteria

I haven't had any other cancers or uncontrolled illnesses that would stop me from taking a new drug in the last 3 years.

I am not pregnant or breastfeeding.

My basal cell carcinoma has worsened despite treatment with HHPI.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily hedgehog pathway inhibitor and SP-002 treatment in 1 or 3 cycles, depending on the arm

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

36 months

What Are the Treatments Tested in This Trial?

Interventions

• SP-002

Trial Overview The study tests the effectiveness of SP-002 when added to standard hedgehog pathway inhibitor therapy in treating Basal cell carcinoma. It compares tumor response rates across three groups: one receiving daily inhibitors plus 3 cycles of SP-002, another getting daily inhibitors plus 1 cycle of SP-002, and a third taking only the daily inhibitors.

How Is the Trial Designed?

Group I: Arm 2 Experimental Treatment 2 Interventions

vismodegib, with 1 cycles SP-002

Group II: Arm 1 Experimental Treatment 2 Interventions

vismodegib, with 3 cycles SP-002

Group III: Arm 3 Placebo Group 1 Intervention

vismodegib monotherapy

Who Is Running the Clinical Trial?

Stamford Pharmaceuticals, Inc.

Lead Sponsor

Trials

1

Recruited

80+

Published Research Related to This Trial

Vismodegib (Erivedge) is an effective treatment for advanced basal cell carcinoma, targeting the Hedgehog signaling pathway that is often overactive in this type of skin cancer.

Clinical studies have demonstrated that Vismodegib can lead to significant tumor shrinkage and improved outcomes for patients with advanced cases, highlighting its role as a key therapeutic option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vismodegib (erivedge) for advanced Basal cell carcinoma. Fellner, C.[2023]

In a real-world study of 63 patients with locally advanced or metastatic basal cell carcinoma (BCC), vismodegib demonstrated a high objective response rate (ORR) of 73%, with 57% achieving a partial response and 16% achieving a complete response.

While vismodegib was effective, 76.2% of patients experienced at least one adverse event, with common side effects including muscular spasms and dysgeusia, and serious adverse events occurred in 17% of patients, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety profile of vismodegib in a real-world setting cohort of patients with advanced basal cell carcinoma in Argentina. Cozzani, R., Del Aguila, R., Carrizo, M., et al.[2021]

A review of the FDA Adverse Event Reporting System revealed a safety signal for hepatotoxicity associated with vismodegib, with 94 adverse event reports, including 35 serious cases, indicating potential liver-related risks in patients.

Despite the lack of formal safety notifications from the manufacturer or FDA, the findings suggest that vismodegib should be used cautiously in patients with severe liver disease and that hepatotoxic medications should be avoided to minimize serious liver complications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hepatotoxicity with Vismodegib: An MD Anderson Cancer Center and Research on Adverse Drug Events and Reports Project. Edwards, BJ., Raisch, DW., Saraykar, SS., et al.[2018]

In a case series of 13 patients with locally advanced periocular basal cell carcinoma (BCC) treated with vismodegib, 38% achieved a complete response and 62% had a partial response, indicating its efficacy in managing this type of cancer.

Vismodegib was generally well-tolerated, with fatigue being the most common side effect, and it may allow for globe-sparing surgeries, potentially reducing the need for more drastic procedures like exenteration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vismodegib for periocular basal cell carcinoma: an international multicentre case series. Oliphant, H., Laybourne, J., Chan, K., et al.[2021]

Vismodegib is an orally bioavailable drug that inhibits the Hedgehog signaling pathway by targeting the smoothened (SMO) protein, which is crucial in the development of tumors like basal cell carcinoma.

Approved in the U.S. in January 2012, vismodegib has shown clinical activity in treating basal cell carcinoma, highlighting its potential as a targeted therapy for this type of cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Investigational agents in metastatic basal cell carcinoma: focus on vismodegib. Batty, N., Kossoff, E., Dy, GK.[2020]

Erivedge (vismodegib) was approved by the FDA for treating recurrent, locally advanced, or metastatic basal cell carcinoma (BCC) based on a trial showing an overall response rate of 30.3% in metastatic cases and 42.9% in locally advanced cases, with median response durations of 7.6 months for both groups.

The treatment was generally well-tolerated, though common side effects included muscle spasms, alopecia, and fatigue; however, it is important to note that vismodegib is a potent teratogenic agent, indicating significant safety considerations for pregnant patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

U.S. Food and Drug Administration approval: vismodegib for recurrent, locally advanced, or metastatic basal cell carcinoma. Axelson, M., Liu, K., Jiang, X., et al.[2022]

A survey of oncologists and dermato-oncologists across several European countries showed that 95% of respondents in 2018 were aware of the teratogenic risks associated with vismodegib, indicating effective communication of safety information.

The majority of physicians (95% in 2018) reported taking appropriate precautions to minimize the risk of teratogenicity when prescribing vismodegib, demonstrating a strong adherence to safety protocols established by the Erivedge Pregnancy Prevention Program.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vismodegib (ERIVEDGE) pregnancy prevention programme: assessment of risk awareness. Schnetzler, G., Cuberos, M., Bucher, R., et al.[2022]

In a study involving 74 patients with operable basal cell carcinoma, vismodegib treatment resulted in complete histologic clearance rates of 42%, 16%, and 44% across three different treatment cohorts, but did not meet the predefined efficacy endpoints of over 50%.

The most common side effects included muscle spasms (76%), alopecia (58%), and dysgeusia (50%), with 7% of patients discontinuing treatment due to adverse events; however, these side effects were found to be reversible after a period of observation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase II, multicenter, open-label, 3-cohort trial evaluating the efficacy and safety of vismodegib in operable basal cell carcinoma. Sofen, H., Gross, KG., Goldberg, LH., et al.[2015]

Vismodegib is the first approved Hedgehog pathway inhibitor specifically for treating metastatic basal cell carcinoma (BCC) and advanced BCC that cannot be surgically removed or treated with radiation.

The drug's development is notable for its unique origins, which include natural sources like corn lilies and sheep, as well as research involving Drosophila flies, highlighting the diverse pathways leading to its discovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vismodegib and the Hedgehog Pathway Inhibitors: A Historical Perspective to Current Clinical Application. Tang, JC., Hanke, CW., Caro, I.[2018]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Vismodegib (erivedge) for advanced Basal cell carcinoma. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety profile of vismodegib in a real-world setting cohort of patients with advanced basal cell carcinoma in Argentina. [2021]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Hepatotoxicity with Vismodegib: An MD Anderson Cancer Center and Research on Adverse Drug Events and Reports Project. [2018]

4. England pubmed.ncbi.nlm.nih.gov

Vismodegib for periocular basal cell carcinoma: an international multicentre case series. [2021]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Investigational agents in metastatic basal cell carcinoma: focus on vismodegib. [2020]

6. United States pubmed.ncbi.nlm.nih.gov

U.S. Food and Drug Administration approval: vismodegib for recurrent, locally advanced, or metastatic basal cell carcinoma. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Vismodegib (ERIVEDGE) pregnancy prevention programme: assessment of risk awareness. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

A phase II, multicenter, open-label, 3-cohort trial evaluating the efficacy and safety of vismodegib in operable basal cell carcinoma. [2015]

9. United States pubmed.ncbi.nlm.nih.gov

Vismodegib and the Hedgehog Pathway Inhibitors: A Historical Perspective to Current Clinical Application. [2018]