Pulmozyme (dornase alfa) / Roche - LARVOL DELTA

Efficacy of Pulmozyme® on Arterial Recanalization in Post-thrombectomy Patients Managed for Ischemic Stroke (NETs-target) (clinicaltrials.gov) - P2 | N=39 | Completed | Sponsor: Fondation Ophtalmologique Adolphe de Rothschild | Trial completion date: Nov 2025 ➔ Aug 2025 | Recruiting ➔ Completed

Trial completion • Trial completion date • Cardiovascular • Ischemic stroke

DACT-GCT: Dornase Alfa and Cisplatin in Refractory Germ Cell Cancer. (clinicaltrials.gov) - P2 | N=33 | Recruiting | Sponsor: National Cancer Institute, Slovakia

New P2 trial • Germ Cell Tumors • Oncology

RESET: Efficacy of Daily IV Administration of Dornase Alfa Up to 14 Days Post Subarachnoid Hemorrhage on Functional Independence At 6 Months (clinicaltrials.gov) - P2 | N=304 | Not yet recruiting | Sponsor: Fondation Ophtalmologique Adolphe de Rothschild

New P2 trial • Cardiovascular • Hematological Disorders • Subarachnoid Hemorrhage

Vabysmo, Hemlibra Drive Roches Sales in Q3 & First 9 Months of 2024 (Yahoo Finance) - "Immuno-oncology drug Tecentriq’s (for advanced lung cancer, urothelial cancer and breast cancer) sales grew 1% to CHF 2.7 billion....Asthma drug Xolair generated sales of CHF 1.7 billion, up 11% year over year....Pulmozyme (cystic fibrosis) sales were flat year over year at CHF 329 million."

Sales • Asthma • Breast Cancer • Cystic Fibrosis • Lung Cancer • Urothelial Cancer

A Study of the Comparability of the Pharmacodynamic, Toxicological, and Pharmacokinetic Properties of the Reference Drug Pulmozyme® and the Biosimilar Drug Tigerase®. (PubMed, Dokl Biochem Biophys) - "The pharmacokinetic parameters of the drugs in the blood serum, bronchi, and lungs, as well as the main physiological parameters (body weight and temperature, the state of the cardiovascular, respiratory, excretory systems, hematological and biochemical blood parameters, pathomorphological changes in internal organs (including the state of the cornea), and mortality rates) were investigated in comparative studies of subchronic toxicity in juvenile and mature rats with 28-day inhalation at doses of 0.2 mg/kg for mature animals and 0.26 mg/kg for juvenile animals (the dose was 6 times higher than the dose recommended for clinical use). The results of the studies allow us to conclude that the drugs are comparable in enzymatic, mucolytic (secretolytic) DNase activity, safety profile and main pharmacokinetic parameters."

Journal • PK/PD data • Cardiovascular • Cystic Fibrosis • Genetic Disorders • Hematological Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

TRAUMADOR: Inhaled Dornase Alpha to Reduce Respiratory Failure After Severe Trauma (clinicaltrials.gov) - P3 | N=500 | Recruiting | Sponsor: University Hospital, Strasbourg, France | Trial completion date: Dec 2024 ➔ Dec 2027 | Trial primary completion date: Sep 2024 ➔ Sep 2027

Trial completion date • Trial primary completion date • Acute Respiratory Distress Syndrome • Critical care • Mood Disorders • Respiratory Diseases

Efficacy and tolerability of dornase alfa biosimilar in children and adults with cystic fibrosis (ECFS 2024) - "There was no statistically significant decrease in FEV 1 data during the study period, and in children and adolescents there was a tendency of increase in FEV 1 . Child patients tolerated DA well; in adults, AEs were observed, which in most cases did not require discontinuation of the drug. In 2 children ADAs were identified."

Clinical • Cystic Fibrosis • Dysphonia • Genetic Disorders • Immunology • Otorhinolaryngology • Pain • Pulmonary Disease • Respiratory Diseases • Xerostomia

SIMPLIFY-DN: Impact of Discontinuing Dornase Alfa in People With CF on Highly Effective CFTR Modulator Therapy-A SIMPLIFY Sub-Study (clinicaltrials.gov) - P=N/A | N=477 | Completed | Sponsor: Nicole Hamblett

New trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Reduction of SystemiC Inflammation After Ischemic Stroke by Intravenous DNase Administration (ReSCInD) (clinicaltrials.gov) - P2 | N=36 | Not yet recruiting | Sponsor: Ludwig-Maximilians - University of Munich | Trial completion date: Jan 2025 ➔ Jan 2026 | Initiation date: Dec 2023 ➔ Dec 2024 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Trial initiation date • Trial primary completion date • Cardiovascular • Inflammation • Ischemic stroke • IL1B

I-SPY_COVID: I-SPY COVID-19 TRIAL: An Adaptive Platform Trial for Critically Ill Patients (clinicaltrials.gov) - P2 | N=1500 | Recruiting | Sponsor: QuantumLeap Healthcare Collaborative | Trial completion date: Nov 2024 ➔ Jul 2030 | Trial primary completion date: Jul 2023 ➔ Jul 2028

Trial completion date • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease

I-SPY_COVID: I-SPY COVID-19 TRIAL: An Adaptive Platform Trial for Critically Ill Patients (clinicaltrials.gov) - P2 | N=1500 | Recruiting | Sponsor: QuantumLeap Healthcare Collaborative | Trial completion date: Nov 2023 ➔ Nov 2024

Trial completion date • Infectious Disease • Novel Coronavirus Disease

Reduction of SystemiC Inflammation After Ischemic Stroke by Intravenous DNase Administration (ReSCInD) (clinicaltrials.gov) - P2 | N=36 | Recruiting | Sponsor: Ludwig-Maximilians - University of Munich | Initiation date: Jul 2023 ➔ Oct 2023

Trial initiation date • Cardiovascular • Inflammation • Ischemic stroke • CASP1 • IL1B • IL6

Pleural Irrigation With Normal Saline Versus Intrapleural Fibrinolytic (clinicaltrials.gov) - P=N/A | N=78 | Recruiting | Sponsor: National University of Malaysia | Not yet recruiting ➔ Recruiting

Enrollment open • Infectious Disease • CRP

Dissolving the glue in glue ear: Assessment of the efficacy of the use of Dornase alfa as an adjunct therapy to ventilation tube insertion. (ANZCTR) - P1/2 | N=60 | Completed | Sponsor: Telethon Kids Institute | Initiation date: May 2000 ➔ May 2012

Trial initiation date • Otorhinolaryngology

Reduction of SystemiC Inflammation After Ischemic Stroke by Intravenous DNase Administration (ReSCInD) (clinicaltrials.gov) - P2 | N=36 | Recruiting | Sponsor: Ludwig-Maximilians - University of Munich | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Inflammation • Ischemic stroke • IL1B • IL6

Efficacy of Pulmozyme® on Arterial Recanalization in Post-thrombectomy Patients Managed for Ischemic Stroke (NETs-target) (clinicaltrials.gov) - P2 | N=44 | Recruiting | Sponsor: Fondation Ophtalmologique Adolphe de Rothschild | Not yet recruiting ➔ Recruiting | Trial completion date: Mar 2024 ➔ Nov 2025 | Trial primary completion date: Mar 2024 ➔ Aug 2025

Enrollment open • Trial completion date • Trial primary completion date • Cardiovascular • Ischemic stroke

Pleural Irrigation With Normal Saline Versus Intrapleural Fibrinolytic (clinicaltrials.gov) - P=N/A | N=78 | Not yet recruiting | Sponsor: National University of Malaysia

New trial • Infectious Disease • CRP

Reduction of SystemiC Inflammation After Ischemic Stroke by Intravenous DNase Administration (ReSCInD) (clinicaltrials.gov) - P2 | N=36 | Not yet recruiting | Sponsor: Ludwig-Maximilians - University of Munich

New P2 trial • Cardiovascular • Inflammation • Ischemic stroke • IL1B • IL6

Possibilities of using recombinant human deoxyribonuclease I in otorhinolaryngology (PubMed, Vestn Otorinolaringol) - "Analysis of studies on the use of Dornase alfa demonstrates an improvement in clinical symptoms in all patients with CF and chronic rhinosinusitis. In experimental studies on an animal model, as well as in vitro research on exudate from the middle ear, Dornase alfa has demonstrated high efficacy and safety. Dornase alfa is a drug with high potential, further research is needed for wider use in ENT practice, especially in otiatrics."

Journal • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Otorhinolaryngology • Pulmonary Disease • Respiratory Diseases • Sinusitis

DISmantling COvid iNduced Neutrophil ExtraCellular Traps (DISCONNECT-1) (clinicaltrials.gov) - P1 | N=15 | Completed | Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre | Recruiting ➔ Completed | N=25 ➔ 15

Enrollment change • Trial completion • Infectious Disease • Novel Coronavirus Disease

Usefulness of DORNASE in COVID-19 on HFNO (clinicaltrials.gov) - P4 | N=10 | Terminated | Sponsor: University Medical Centre Ljubljana | N=60 ➔ 10 | Trial completion date: Jun 2022 ➔ Feb 2023 | Not yet recruiting ➔ Terminated | Trial primary completion date: Mar 2022 ➔ Feb 2023; Due to the Omicron version of SARS-CoV-2 the COVID-pneumonia patients ran out

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Infectious Disease • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases

Efficacy of Pulmozyme® on Arterial Recanalization in Post-thrombectomy Patients Managed for Ischemic Stroke (NETs-target) (clinicaltrials.gov) - P2 | N=44 | Not yet recruiting | Sponsor: Fondation Ophtalmologique Adolphe de Rothschild | Trial completion date: Oct 2024 ➔ Mar 2024 | Trial primary completion date: Oct 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Cardiovascular • Ischemic stroke

CF Foundation | NACFC 2022 Highlights (YouTube) - "Researchers and clinicians gather every year at the North American Cystic Fibrosis Conference to discuss the latest developments in cystic fibrosis research and care. During this one-hour webinar, Raksha Jain, MD, and Jennifer Taylor-Cousar, MD, will share highlights from the conference and answer audience questions."

Video

I-SPY_COVID: I-SPY COVID-19 TRIAL: An Adaptive Platform Trial for Critically Ill Patients (clinicaltrials.gov) - P2 | N=1500 | Recruiting | Sponsor: QuantumLeap Healthcare Collaborative | Trial completion date: Nov 2022 ➔ Nov 2023 | Trial primary completion date: Jul 2022 ➔ Jul 2023

Trial completion date • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease

Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials (Lancet) - P=NA | N=870 | SIMPLIFY (NCT04378153) | "Participants across both trials had an average ppFEV1 of 96·9%. Discontinuing treatment was non-inferior to continuing treatment with respect to the absolute 6-week change in ppFEV1 in both the hypertonic saline trial (–0·19% [95% CI –0·85 to 0·48] in the discontinuation group [n=133] vs 0·14% [–0·51 to 0·78] in the continuation group [n=140]; between-group difference –0·32% [–1·25 to 0·60]) and dornase alfa trial (0·18% [–0·38 to 0·74] in the discontinuation group [n=199] vs –0·16% [–0·73 to 0·41] in the continuation group [n=193]; between-group difference 0·35% [–0·45 to 1·14]), with consistent results in the intention-to-treat populations."

Clinical data • Cystic Fibrosis