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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
All Individual Drugs Already Approved
No Placebo-Only Group

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.
Select...
I have the target disease or have been treated with pembrolizumab.
Select...
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.
Select...
I am fully active or can carry out light work.
Select...
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.
Select...
I am not currently using any experimental treatments or devices.
Select...
I have previously received CAR-T or allogeneic cellular therapy.
Select...
I am currently on medication to suppress my immune system.
Select...
I have had a solid organ or bone marrow transplant.

Timeline

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
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 study objectives
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 study objectives
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 3 trial • 804 Patients • NCT03040999
64%
Radiation skin injury
63%
Stomatitis
58%
Anaemia
56%
Nausea
48%
Dry mouth
45%
Constipation
45%
Weight decreased
44%
Dysphagia
42%
Neutrophil count decreased
33%
Dysgeusia
33%
Vomiting
32%
Fatigue
31%
White blood cell count decreased
28%
Hypomagnesaemia
26%
Decreased appetite
25%
Hypothyroidism
25%
Hypokalaemia
24%
Lymphocyte count decreased
24%
Platelet count decreased
23%
Oropharyngeal pain
23%
Blood creatinine increased
22%
Diarrhoea
22%
Odynophagia
20%
Hypoacusis
20%
Alanine aminotransferase increased
20%
Hyponatraemia
19%
Tinnitus
19%
Oral candidiasis
19%
Asthenia
16%
Pyrexia
16%
Cough
15%
Aspartate aminotransferase increased
15%
Rash
14%
Insomnia
13%
Acute kidney injury
13%
Pharyngeal inflammation
13%
Pruritus
12%
Dysphonia
12%
Gamma-glutamyltransferase increased
11%
Pneumonia
11%
Dehydration
10%
Hyperthyroidism
10%
Hypoalbuminaemia
10%
Hypocalcaemia
10%
Headache
10%
Productive cough
9%
Neck pain
9%
Peripheral sensory neuropathy
8%
Gastrooesophageal reflux disease
8%
Hiccups
8%
Hyperglycaemia
8%
Hyperuricaemia
8%
Dizziness
8%
Hypophosphataemia
7%
Urinary tract infection
7%
Ear pain
7%
Localised oedema
7%
Hyperkalaemia
7%
Erythema
7%
Oral pain
6%
Abdominal pain upper
6%
Arthralgia
6%
Anxiety
6%
Febrile neutropenia
6%
Dyspepsia
6%
Saliva altered
5%
Back pain
5%
Oedema peripheral
5%
Hypertension
5%
Dyspnoea
4%
Nasopharyngitis
4%
Alopecia
4%
Dry skin
3%
Pneumonia aspiration
3%
Sepsis
3%
Trismus
3%
Pneumonitis
3%
Laryngeal oedema
2%
Malnutrition
2%
Pharyngeal haemorrhage
2%
Cellulitis
1%
Septic shock
1%
Clostridium difficile colitis
1%
Systemic infection
1%
Cardiac arrest
1%
Death
1%
Bronchitis
1%
Hepatitis
1%
Immune-mediated hepatitis
1%
Oesophagitis
1%
General physical health deterioration
1%
Hypophagia
1%
Tumour haemorrhage
1%
Cerebrovascular accident
1%
Syncope
1%
Acute respiratory failure
1%
Aspiration
1%
Colitis
1%
Mouth haemorrhage
1%
Hypersensitivity
1%
Acute myocardial infarction
1%
Abscess neck
1%
Device related infection
1%
Stoma site infection
1%
Vascular device infection
1%
Wound infection
1%
Hypercalcaemia
1%
Pulmonary embolism
1%
Respiratory failure
100%
80%
60%
40%
20%
0%
Study treatment Arm
Pembrolizumab + CRT Followed by Pembrolizumab
Placebo + CRT Followed by Placebo

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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

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.

Find a Location

Who is running the clinical trial?

Neurogene Inc.Lead Sponsor
5 Previous Clinical Trials
88 Total Patients Enrolled
Neoleukin Therapeutics, Inc.Lead Sponsor
Merck Sharp & Dohme LLCIndustry Sponsor
3,965 Previous Clinical Trials
5,176,369 Total Patients Enrolled

Media Library

NL-201 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04659629 — Phase 1
NL-201 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04659629 — Phase 1
Solid Tumors Clinical Trial 2023: NL-201 Highlights & Side Effects. Trial Name: NCT04659629 — Phase 1
Solid Tumors Research Study Groups: Part 1: NL-201 Monotherapy Dose Escalation, Part 2: NL201 Monotherapy Expansion Cohorts, Part 3: NL-201 in Combination with Pembrolizumab Dose Escalation, Part 4: NL-201 in Combination with Pembrolizumab Expansion Cohorts
~13 spots leftby Oct 2025