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?

This trial explores a new treatment, SP-002, for individuals with locally advanced basal cell carcinoma, a type of skin cancer difficult to treat with surgery. Researchers aim to determine if adding SP-002 to the standard hedgehog pathway inhibitor therapy enhances its effectiveness. They will compare results among three groups: one receiving three cycles of SP-002, one receiving one cycle, and one receiving only the standard therapy. Individuals with a single basal cell carcinoma lesion, who are not suitable candidates for surgery, might be well-suited for this trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in cancer treatment.

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 there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that vismodegib, a type of medication, is generally well-tolerated by patients with advanced basal cell carcinoma. In studies with 108 patients, it was used safely, although some experienced side effects like muscle cramps and hair loss, which are common with cancer treatments.

For SP-002, less information is available. As this trial is in an early stage, researchers are still learning about its safety. This means unknown risks might exist, but scientists are closely monitoring to ensure participant safety. Participants in this trial will receive SP-002 along with vismodegib, so researchers will carefully watch for any potential side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about SP-002 combined with vismodegib for basal cell carcinoma because it introduces a unique approach to treatment. Unlike the standard of care, which primarily revolves around surgical excision and vismodegib monotherapy, SP-002 offers potential enhanced efficacy by synergizing with vismodegib. SP-002's novel mechanism may improve treatment outcomes by targeting additional pathways involved in cancer growth, offering hope for patients with advanced or resistant forms of basal cell carcinoma.

What evidence suggests that this trial's treatments could be effective for Basal cell carcinoma?

This trial will compare different treatment approaches for basal cell carcinoma. Research has shown that combining SP-002 with vismodegib yields promising results for treating this type of skin cancer. Participants in this trial may receive vismodegib with either three cycles or one cycle of SP-002. Studies highlight that this combination is both safe and effective. Vismodegib alone, another treatment arm in this trial, is already effective, especially for challenging cases of this cancer. Adding SP-002 appears to enhance the treatment, helping to shrink tumors more effectively. Early findings suggest that patients receiving both treatments may experience better outcomes than those using only vismodegib. ^{4 6 7 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

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 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 (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]

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]

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]

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]

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]

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]

Citations

1. erivedge.com erivedge.com/hcp/efficacy/clinical-results.html

Clinical Results | Erivedge® (vismodegib) Erivedge® (vismodegib) clinical results for patients with locally advanced and metastatic basal cell carcinoma. See full safety & Boxed Warning.

2. stamfordpharmaceuticals.com stamfordpharmaceuticals.com/stamford-announces-positive-results-from-phase-2-study-of-sp-002-in-combination-with-4-weeks-of-vismodegib-in-multi-lesional-basal-cell-carcinoma-patients

Stamford announces positive results from Phase 2 Study of ... Stamford announces positive results from Phase 2 Study of SP-002 in combination with 4-weeks of vismodegib in multi-lesional Basal Cell ...

3. synapse.patsnap.com synapse.patsnap.com/article/stamford-reports-positive-phase-2-results-of-sp-002-with-vismodegib-in-basal-cell-carcinoma-patients

Stamford reports positive Phase 2 results of SP-002 with ... The results from this Phase 2 study have been encouraging. They underscore both the safety and the effectiveness of the gene therapy for individuals dealing ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10218270/

Retrospective Real-Life Data, Efficacy and Safety of ... Vismodegib is a safe and effective treatment for locally advanced BCC, and its role in unresectable BCC seems to be an important option in these challenging ...

5. 1stoncology.com 1stoncology.com/blog/stamford-announces-positive-results-from-phase-2-study-of-sp-002-in-combination-with-4-weeks-of-vismodegib-in-multi-lesional-basal-cell-carcinoma-patients1234649770/

Stamford announces positive results from Phase 2 Study of ... On January 17, 2025 Stamford reported positive results for its ASN-002-003 multi-lesional clinical trial (NCT 04416516.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9541446/

Analysis of efficacy and safety of vismodegib therapy in ... The study aimed to retrospectively analyse the efficacy and safety of treatment with vismodegib in 108 patients with locally advanced or metastatic disease.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT06344052?term=vismodegib&aggFilters=status:rec%20enr%20act%20ava&viewType=Table&rank=6

To Assess the Safety and Efficacy of SP-002 with ... The goal of this clinical trial is to evaluate the efficacy of using SP-002 in participants with locally advanced Basal cell carcinoma.

8. nejm.org nejm.org/doi/full/10.1056/NEJMoa1113713

Efficacy and Safety of Vismodegib in Advanced Basal-Cell ... A phase 1 study of vismodegib involving 33 patients with advanced basal-cell carcinoma showed a 58% confirmed response rate and a median duration of response ...

9. erivedge.com erivedge.com/content/dam/gene/erivedge/hcp/pdfs/patient_case_series_2.pdf

Case studies in advanced basal cell carcinoma (BCC) The safety data described below reflect exposure to ERIVEDGE in 138 patients with advanced basal cell carcinoma (BCC) who received ERIVEDGE at doses ≥ 150 ...