ND-L02-s0201 for IPF

No longer recruiting at 38 trial locations

Overseen By Kathy Carr

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Nitto Denko Corporation

Must not be taking: High dose corticosteroids

Disqualifiers: Active smoker, Malignancy, HIV, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ND-L02-s0201 for people with Idiopathic Pulmonary Fibrosis (IPF), a lung disease that makes breathing difficult. The researchers aim to assess the treatment's safety and effectiveness compared to a placebo (a substance with no active medication). Two treatment groups receive different doses of ND-L02-s0201, while one group receives the placebo. Ideal candidates for this trial have IPF and have not experienced recent lung infections or hospital stays due to their condition. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people, offering participants a chance to contribute to important advancements in IPF treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking high dose corticosteroids, cytotoxic agents, unapproved IPF targeted therapy, and cytokine modulating agents at least 8 weeks or 5 half-lives before screening. If you are on these medications, you will need to stop them before participating.

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

Research has shown that ND-L02-s0201 has been studied for its safety and effects on the body. Earlier studies suggested that higher doses of this treatment might reduce fibrosis (scarring) more effectively. However, in a study with people, ND-L02-s0201 did not demonstrate the expected benefits for treating idiopathic pulmonary fibrosis (IPF), a lung disease. Despite its limited effectiveness, the main focus remains on safety.

Previous studies have aimed to confirm that ND-L02-s0201 is safe and well-tolerated. Its progression to this stage of testing suggests that initial safety results were acceptable. Since this treatment is in phase 2 of testing, researchers have already gathered some safety data from earlier trials and are now examining how people handle it more closely. This phase typically involves more participants to better understand any side effects.

In summary, ND-L02-s0201 has undergone previous testing, and while more information on its effectiveness is needed, earlier results have allowed it to reach this trial stage. This suggests a reasonable level of safety so far, but ongoing studies will provide more detailed insights. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for IPF?

Unlike the standard of care for idiopathic pulmonary fibrosis (IPF), which typically includes antifibrotic drugs like nintedanib and pirfenidone, ND-L02-s0201 offers a novel approach by targeting the fibrotic process itself. This treatment is unique because it uses a lipid nanoparticle delivery system to carry a specific siRNA that aims to reduce collagen production, a key factor in lung scarring. Researchers are excited about ND-L02-s0201 because this mechanism could potentially slow or even reverse disease progression more effectively than current options, offering hope for improved outcomes in patients with IPF.

What evidence suggests that ND-L02-s0201 might be an effective treatment for IPF?

Research has shown that ND-L02-s0201 appeared promising in animal studies for treating lung scarring, known as pulmonary fibrosis. These studies found that it significantly reduced lung scarring and improved lung function in animals. However, in a clinical trial with people who have idiopathic pulmonary fibrosis (IPF), ND-L02-s0201 did not perform as well as expected. The results in humans were not as positive as in the animal studies. The treatment targets a protein involved in scarring, but more research is needed to determine its effectiveness in humans. ^{1 2 4 6 7}

Who Is on the Research Team?

Nitto Denko Corporation

Principal Investigator

Nitto Denko Corporation

Are You a Good Fit for This Trial?

Inclusion Criteria

Forced vital capacity (FVC) ≥ 45% of predicted.

Diffusion capacity of the lung for carbon monoxide (DLco) corrected for hemoglobin ≥ 30% of predicted value

Ratio of forced expiratory volume in 1 second (FEV1) to FVC ≥ 0.70.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 6 weeks

Treatment

Participants receive ND-L02-s0201 or placebo for 24 weeks, including 12 doses

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

10 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ND-L02-s0201
• Placebo

How Is the Trial Designed?

Group I: Dose Level 2 Experimental Treatment 1 Intervention

ND-L02-s0201 90mg

Group II: Dose Level 1 Experimental Treatment 1 Intervention

ND-L02-s0201 45mg

Group III: Placebo Placebo Group 1 Intervention

Control Arm

Who Is Running the Clinical Trial?

Nitto Denko Corporation

Lead Sponsor

Trials

6

Recruited

550+

Published Research Related to This Trial

A new checklist called TIDieR-Placebo has been developed to improve the reporting of placebo or sham interventions in clinical trials, ensuring that researchers clearly describe these components alongside active treatments.

The checklist was created through expert consultation and aims to enhance the transparency of trial results, which is crucial for accurately assessing the benefits and harms of active interventions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

TIDieR-Placebo: A guide and checklist for reporting placebo and sham controls. Howick, J., Webster, RK., Rees, JL., et al.[2020]

The study systematically analyzed placebo effects from randomized, placebo-controlled trials, revealing that while placebos can influence clinical symptoms, they do not affect laboratory values like blood glucose in diabetics.

Placebo side effects were found to be similar to those of active treatments, highlighting the importance of careful placebo use in clinical research to ensure patient safety and informed consent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo treatment is effective differently in different diseases--but is it also harmless? A brief synopsis. Weihrauch, TR.[2019]

In a study involving 100 patients undergoing lumbar spine fusion surgeries, batroxobin and its combination with tranexamic acid significantly reduced both intraoperative and postoperative blood loss compared to a placebo.

The mean intraoperative blood loss was lowest in the batroxobin group (268.32 mL) and the combination group (256.96 mL), indicating their effectiveness, while no significant differences were found in blood transfusion needs or complications like deep vein thrombosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and Safety of Batroxobin, Tranexamic Acid and a Combination in Reduction of Blood Loss in Lumbar Spinal Fusion Surgery. Nagabhushan, RM., Shetty, AP., Dumpa, SR., et al.[2021]

In a study involving 24 rabbits undergoing spine fusion, daily subcutaneous injections of abaloparatide resulted in a 100% fusion rate compared to only 45% in the control group, indicating its strong efficacy in promoting spinal fusion.

Radiographic analysis showed that abaloparatide significantly increased bone formation and quality, with higher bone volume and improved trabecular structure, suggesting it enhances the biological processes necessary for successful spinal fusion.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Abaloparatide Enhances Fusion and Bone Formation in a Rabbit Spinal Arthrodesis Model. Morse, KW., Moore, H., Kumagai, H., et al.[2023]

Citations

1. nitto.com nitto.com/us/en/press/2023/0915.jsp

Results from Phase 2 Clinical Trial of ND-L02-s0201 for the ... ND-L02-s0201 did not show the expected efficacy in the Phase 2 clinical trial for the treatment of Idiopathic Pulmonary Fibrosis.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8181707/

Anti-HSP47 siRNA lipid nanoparticle ND-L02-s0201 ... Results from two robust chronic rodent models of pulmonary fibrosis demonstrated significant anti-fibrotic effects and improved lung function ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03538301

NCT03538301 | JUNIPER: A Phase 2 Study to Evaluate ... All subjects were treated with ND-L02-s0201 or placebo for 24 weeks (a total of 12 doses). Subject's participation in the study was approximately 40 weeks ...

4. publications.ersnet.org publications.ersnet.org/content/erj/50/suppl61/pa881

ND-L02-s0201 treatment leads to efficacy in preclinical IPF ... Results: In the BLM model, we demonstrated dose-dependent and statistically significant reduction in lung weight, collagen deposition and histology and fibrosis ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT02227459

Study Details | NCT02227459 | Phase 1b/2, Open Label, ... The purpose of this study is to evaluate the safety and tolerability of multiple doses of ND-L02-s0201 in subjects with moderate to extensive hepatic fibrosis.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03241264

A Study of Safety, Tolerability, and the Effects Two ND-L02- ... The purpose of this study is to evaluate the safety, tolerability, and the effects two ND-L02-s0201 have on the body. Official Title. A Phase 1, Open-Label ...

7. publications.ersnet.org publications.ersnet.org/content/erj/50/suppl61/PA881

ND-L02-s0201 treatment leads to efficacy in preclinical IPF ... ND-L02-s0201 is being evaluated for the treatment of hepatic fibrosis. IPF patient data show elevated HSP47 lung expression and linkage to poor prognosis. Our ...