What side effects are associated with this type of intervention?

Common treatments for solid tumors, such as immune checkpoint inhibitors (e.g., pembrolizumab, nivolumab) and chemotherapies, have a range of side effects. Immune checkpoint inhibitors can cause immune-related adverse events including fatigue, diarrhea, rash, pruritus, pneumonitis, and endocrinopathies. Severe side effects may include colitis, hepatitis, and myocarditis. Chemotherapies often result in hematologic toxicities like neutropenia, anemia, and thrombocytopenia, as well as non-hematologic toxicities such as nausea, vomiting, fatigue, and alopecia. Both treatment types require careful monitoring and management of side effects to ensure patient safety.

What prior FDA approvals exist?

The FDA has approved several immune checkpoint inhibitors for the treatment of solid tumors. Pembrolizumab (Keytruda) and nivolumab (Opdivo) are notable examples, both of which target the PD-1 pathway to enhance the body's immune response against cancer cells. These drugs have been approved for various types of solid tumors, including melanoma, non-small cell lung cancer, and renal cell carcinoma. Combination therapies, such as nivolumab with ipilimumab (Yervoy), have also been approved to improve treatment efficacy in certain cancers. These approvals highlight the growing role of immunotherapy in the management of solid tumors.

What other types of research exist?

Promising novel alternatives to NL-201 for solid tumor patients include: 1) Pembrolizumab, an immune checkpoint inhibitor that has shown efficacy in various solid tumors. 2) Cabozantinib, a tyrosine kinase inhibitor used in renal cell carcinoma and other cancers. 3) Lutetium-177-PSMA-617, a radioligand therapy for prostate cancer. 4) Nivolumab plus Ipilimumab, a combination of immune checkpoint inhibitors used in renal cell carcinoma and other cancers. These therapies have demonstrated safety and efficacy in clinical trials and represent potential treatment options for solid tumor patients in 2024.

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NL-201 + Pembrolizumab for Cancer

Phase 1

Waitlist Available

Research Sponsored by Neurogene Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Part 1 Only: Patients with relapsed or refractory advanced solid tumor, other than prostate cancer, who have progressed, not tolerated or are ineligible for all approved lines of therapy

Part 4 Only: Patients with diagnosed target disease OR previously received pembrolizumab

Must not have

Prior IL-2-based cancer therapy

Concurrent therapy with any other investigational agent, vaccine, or device

Timeline

Screening 3 weeks

Treatment Varies

Follow Up upto 36 months

Awards & highlights

Summary

This trial tests the safety and effectiveness of NL-201 alone and with pembrolizumab in patients with advanced cancer. NL-201 may directly affect cancer cells, and pembrolizumab helps the immune system attack cancer. The goal is to find a safe and effective dose and schedule for these treatments. Pembrolizumab has shown efficacy and safety in treating various cancers, including melanoma.

Who is the study for?

This trial is for adults with advanced solid tumors who have tried all approved treatments without success, or can't tolerate them. They must be at least 6 weeks out from certain cancer drugs and have a good performance status. People with prostate cancer, active COVID-19, recent transplants, ongoing immunosuppression, another progressing cancer within the last 2 years (except some skin cancers), or those on other investigational therapies are excluded.

What is being tested?

The study tests NL-201 alone (Parts 1 & 2) and combined with Pembrolizumab (Parts 3 & 4) in patients with relapsed or refractory cancer. It aims to find safe dosages and schedules by evaluating how well participants tolerate these drugs and observing any potential benefits.

What are the potential side effects?

Possible side effects of NL-201 and Pembrolizumab may include reactions at the infusion site, immune-related issues affecting organs like lungs or intestines, fatigue, skin conditions, changes in blood counts leading to increased infection risk or bleeding problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have an advanced solid tumor (not prostate cancer) and no standard treatments work for me.

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I have the target disease or have been treated with pembrolizumab.

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It's been over 6 weeks since my last nitrosurea or mitomycin C therapy, 4 weeks since any chemotherapy or checkpoint inhibitor, and 2 weeks since any kinase inhibitor.

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I am fully active or can carry out light work.

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My cancer can be measured by tests.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have previously received IL-2-based cancer treatment.

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I am not currently using any experimental treatments or devices.

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I have previously received CAR-T or allogeneic cellular therapy.

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I am currently on medication to suppress my immune system.

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I have had a solid organ or bone marrow transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ upto 36 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~upto 36 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and upto 36 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of treatment-emergent adverse events

Recommended dose schedule for NL-201 (Parts 1 and 2)

Recommended dose schedule for NL-201 in combination with Pembrolizumab (Parts 3 and 4)

+3 more

Secondary outcome measures

Best Objective Response according to RECIST version 1.1

Duration of Response (DOR) according to RECIST version 1.1

Immunogenicity of NL-201

+7 more

Other outcome measures

Analysis of immune characteristics of the tumor microenvironment

Estimate additional measures of anti-tumor activity of NL- 201 per iRECIST criteria

Flow cytometry analysis of immune cells in blood

+1 more

Side effects data

From 2024 Phase 2 trial • 57 Patients • NCT03004183

21%

Fatigue

13%

Nausea

11%

Back pain

Diarrhea

Anemia

Shortness of Breath

Abdominal pain

Kidney Injury and/or Infection

Pneumonia

Weight Loss

Dyspnea

Malnutrition, Hypercalcemia and Weakness

Pneumothorax

Intractable pain, back pain, hip pain

Activated partial thromboplastin time prolonged

Atrial fibrillation with rapid ventricular response

Pleural effusion

colitis

Thrombocytopenia

Respiratory failure

Skin rash

100%

80%

60%

40%

20%

Study treatment Arm

Single Arm

Trial Design

4Treatment groups

Experimental Treatment

Group I: Part 4: NL-201 in Combination with Pembrolizumab Expansion CohortsExperimental Treatment2 Interventions

NL-201 in combination with Pembrolizumab in indication specific cohorts at a dose and schedule determined in Part 3

Group II: Part 3: NL-201 in Combination with Pembrolizumab Dose EscalationExperimental Treatment2 Interventions

NL-201, in combination with a set Pembrolizumab dose, testing ascending doses and two different schedules

Group III: Part 2: NL201 Monotherapy Expansion CohortsExperimental Treatment1 Intervention

NL-201 given as monotherapy by intravenous administration in indication specific cohorts at a dose and schedule determined in Part 1.

Group IV: Part 1: NL-201 Monotherapy Dose EscalationExperimental Treatment1 Intervention

NL-201 given as monotherapy by intravenous administration testing ascending doses and two different schedules.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pembrolizumab

FDA approved

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for solid tumors include chemotherapy, immunotherapy, and targeted therapy. Chemotherapy works by killing rapidly dividing cells, which includes cancer cells, but also affects normal cells, leading to side effects. Immunotherapy, such as the combination of NL-201 and pembrolizumab, enhances the body's immune system to recognize and attack cancer cells. Targeted therapy involves drugs designed to target specific genetic mutations or proteins that are involved in tumor growth and progression. These treatments are crucial for solid tumor patients as they offer different mechanisms to control or eliminate cancer, potentially improving survival rates and quality of life.

Current trends and future directions in the genetic therapy of human neoplastic disease.