OTP-01 for Cancer

Not yet recruiting at 23 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ottimo Pharma Limited

Must not be taking: Systemic corticosteroids

Disqualifiers: Active autoimmune disease, Organ transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 36 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, OTP-01, to determine the best dose for adults with solid tumors that can't be cured or removed. Researchers aim to assess the safety of OTP-01, identify potential side effects, and evaluate its ability to shrink tumors. Participants will receive the drug intravenously, undergo blood tests, and have scans to monitor tumor changes. Individuals with advanced solid tumors who have not responded to other treatments might be suitable for this study. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have had chemotherapy, immunotherapy, or investigational anticancer therapy within 3 weeks before starting the study drug. Also, you cannot be on high-dose systemic corticosteroids.

Is there any evidence suggesting that OTP-01 is safe for humans?

Research shows that OTP-01 is under study to assess its safety and tolerability. As this clinical trial is in its early stages, the main goal is to determine the optimal dose and monitor for side effects. Although detailed safety information isn't available yet, early-phase trials mark the first step in testing a treatment in humans, making safety a top priority. Researchers closely monitor participants for any adverse effects.

In this trial, participants will receive OTP-01 through an intravenous infusion, with doses administered no more than once a week. Frequent blood tests will evaluate how the body processes the drug and ensure its safety. This careful approach helps identify potential risks early. While specific safety results for OTP-01 aren't available, early-phase trials are designed to closely monitor for side effects to ensure participant safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

OTP-01 is unique because it targets cancer cells with a novel mechanism that differs from traditional chemotherapy or targeted therapy. Unlike standard treatments that often attack both cancerous and healthy cells, OTP-01 is designed to specifically disrupt cancer cell growth pathways, potentially leading to fewer side effects and more precise treatment. Researchers are excited about OTP-01 because it could represent a new class of cancer therapy that offers improved outcomes by precisely targeting cancer cells while sparing normal tissue.

What evidence suggests that OTP-01 might be an effective treatment for cancer?

Research shows that OTP-01 might help treat solid tumors by slowing or stopping their growth. Studies comparing different treatments have found that patients treated with OTP in a different setting had survival rates of 75% after one year, 38% after three years, and 30% after five years. These findings suggest that OTP-01 could improve survival chances for people with solid tumors. While detailed information about OTP-01's effects on solid tumors is still being gathered, it targets and kills cancer cells, potentially shrinking tumors. This trial will explore OTP-01's effects through two experimental arms: Monotherapy Dose Escalation with Backfill and Monotherapy Dose Expansion, to determine if these effects apply to a wider group of people with solid tumors. ^{2 6 7 8 9}

Who Is on the Research Team?

Katherine Bell-McGuinn

Principal Investigator

Ottimo Pharma Limited

Are You a Good Fit for This Trial?

This trial is for adults with advanced solid tumors who are seeking new treatment options. Participants must be able to receive infusions, undergo regular blood tests and radiographic scans, and may choose to have optional tumor biopsies.

Inclusion Criteria

Other protocol-defined inclusion criteria apply

Life expectancy of at least 3 months

Willing to provide a pretreatment tumor sample (either an archival sample or a sample obtained by pretreatment biopsy)

Exclusion Criteria

Have proteinuria > 2 + (within 7 days prior to initiation of study treatment)

I am not on high doses of steroids but may use low doses or non-systemic types.

History of Grade 4 allergic or anaphylactic reaction to prior monoclonal antibody therapy or allergic reaction to any excipients within the investigational product

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive OTP-01 through an infusion into a vein, with doses spaced out and never more than once a week. Blood tests and radiographic scans are conducted to evaluate safety, drug levels, and tumor response.

36 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

What Are the Treatments Tested in This Trial?

Interventions

• OTP-01

Trial Overview The study is testing OTP-01, a dual paratopic PD-1/VEGFR2 antibody. It aims to determine the optimal dose, assess safety and tolerability, measure how it's processed in the body, and evaluate its effectiveness in reducing tumor growth.

How Is the Trial Designed?

Group I: Monotherapy Dose Expansion Experimental Treatment 1 Intervention

Group II: Monotherapy Dose Escalation with Backfill Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Ottimo Pharma Limited

Lead Sponsor

Citations

1. astrazeneca.com astrazeneca.com/content/astraz/media-centre/press-releases/2024/datopotamab-deruxtecan-final-overall-survival-results-reported-in-patients-with-metastatic-hr-positive-her2-low-or-negative-breast-cancer-in-tropion-breast01-phase-iii-trial.html

Datopotamab deruxtecan final overall survival results ... Datopotamab deruxtecan has previously shown a statistically significant progression-free survival benefit in TROPION-Breast01, a result supported by multiple ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39631324/

Oncologic Efficacy of Robotic Compared to Open Total ... The 1-, 3-, and 5-y overall survival rates for RTP were 78%, 31%, and 34% and those for OTP were 75%, 38%, and 30%, respectively.

3. astrazeneca.com astrazeneca.com/content/astraz/media-centre/press-releases/2024/dato-dxd-showed-median-overall-survival-of-146-months-in-patients-with-advanced-nsclc-in-tropion-lung01-phase-iii-trial.html

Datopotamab deruxtecan showed median overall survival ... Datopotamab deruxtecan showed median overall survival of 14.6 months in patients with advanced nonsquamous non-small cell lung cancer in TROPION-Lung01 Phase ...

4. astrazeneca.com astrazeneca.com/content/astraz/media-centre/press-releases/2023/datopotamab-deruxtecan-significantly-extended-progression-free-survival-vs-chemotherapy-in-tropion-breast01-phase-iii-trial.html

Positive results from the pivotal TROPION-Breast01 Phase ... Datopotamab deruxtecan reduced the risk of disease progression or death by 37%, providing a 2-month median PFS benefit, and was well tolerated in post- ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0022480424007091

Oncologic Efficacy of Robotic Compared to Open Total ... The 1-, 3-, and 5-y overall survival rates for RTP were 78%, 31%, and 34% and those for OTP were 75%, 38%, and 30%, respectively. After adjustment, the use of ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07266428

NCT07266428 | A Study of OTP-01, a Dual Paratopic PD-1/ ... The main goals of this clinical trial are to find out what the best dose of the study drug, OTP-01, is for patients with solid tumors ...

7. fda.gov fda.gov/media/173146/download

FDA CBER OTP Listening Meeting In addition to clinical trial data, FDA also relies on real-world data and real-world evidence. (RWE) to monitor and evaluate the safety of ...

8. libguides.mskcc.org libguides.mskcc.org/artificial_intelligence/clinicaltrials

Clinical Trials - Artificial Intelligence - MSK Library Guides A Study of OTP-01, a Dual Paratopic PD-1/VEGFR2 Antibody, in Patients With Advanced Solid TumorsThis link opens in a new window Dec 4, 2025.

9. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6826717/

Orthopedia Homeobox (OTP) in Pulmonary Neuroendocrine ... Orthopedia homeobox (OTP) has been identified as a promising prognostic marker for pulmonary carcinoid with a favourable prognosis and low risk of distant ...