scholarly journals TELO-SCOPE study: a randomised, double-blind, placebo-controlled, phase 2 trial of danazol for short telomere related pulmonary fibrosis

2021 ◽  
Vol 8 (1) ◽  
pp. e001127
Author(s):  
John A Mackintosh ◽  
Maria Pietsch ◽  
Viviana Lutzky ◽  
Debra Enever ◽  
Sandra Bancroft ◽  
...  
Keyword(s):  
Pulmonary Fibrosis ◽  
Telomere Length ◽  
In Situ Hybridisation ◽  
Randomised Trial ◽  
Standard Of Care ◽  
Dyskeratosis Congenita ◽  
Fluorescence In Situ Hybridisation ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Intention To Treat

IntroductionRecent discoveries have identified shortened telomeres and related mutations in people with pulmonary fibrosis (PF). There is evidence to suggest that androgens, including danazol, may be effective in lengthening telomeres in peripheral blood cells. This study aims to assess the safety and efficacy of danazol in adults and children with PF associated with telomere shortening.Methods and analysisA multi-centre, double-blind, placebo-controlled, randomised trial of danazol will be conducted in subjects aged >5 years with PF associated with age-adjusted telomere length ≤10th centile measured by flow fluorescence in situ hybridisation; or in children, a diagnosis of dyskeratosis congenita. Adult participants will receive danazol 800 mg daily in two divided doses or identical placebo capsules orally for 12 months, in addition to standard of care (including pirfenidone or nintedanib). Paediatric participants will receive danazol 2 mg/kg/day orally in two divided doses or identical placebo for 6 months. If no side effects are encountered, the dose will be escalated to 4 mg/kg/day (maximum 800 mg daily) orally in two divided doses for a further 6 months. The primary outcome is change in absolute telomere length in base pairs, measured using the telomere shortest length assay (TeSLA), at 12 months in the intention to treat population.Ethics and disseminationEthics approval has been granted in Australia by the Metro South Human Research Ethics Committee (HREC/2020/QMS/66385). The study will be conducted and reported according to Standard Protocol Items: Recommendations for Interventional Trials guidelines. Results will be published in peer-reviewed journals and presented at international and national conferences.Trial registration numbersNCT04638517; Australian New Zealand Clinical Trials Registry (ACTRN12620001363976p).

PAciFy Cough – A multicentre, double blind, placebo controlled, crossover trial of morphine sulfate for the treatment of PulmonAry Fibrosis Cough

2021 ◽  
Author(s):  
Zhe Wu ◽  
Winston Banya ◽  
Nazia Chaudhuri ◽  
Ira Jakupovic ◽  
Toby Maher ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Pulmonary Fibrosis ◽  
Crossover Trial ◽  
Randomised Trial ◽  
Morphine Sulfate ◽  
Double Blind ◽  
Double Blind Placebo ◽  
The Uk

Abstract Background: Idiopathic pulmonary fibrosis (IPF) is a progressive disease that leads to lung scarring, Cough is reported by 85% of patients with IPF and can be a distressing symptom with a significant impact on patients’ quality of life. There are no proven effective therapies for IPF related cough. While morphine is frequently used as a palliative agent for breathlessness in IPF, its effects on cough have never been tested. PAciFy Cough is a multicenter, double-blind, placebo-controlled, crossover trial of morphine sulfate for the treatment of cough in IPF. Methods: We will recruit 44 subjects with IPF prospectively from three interstitial lung disease units in the UK, namely the Royal Brompton Hospital, Manchester University NHS Foundation Trust (MFT) and Aintree University Hospital NHS Foundation Trust. Patients will be randomized (1:1) to either placebo twice daily or morphine sulfate 5mg twice daily for 14 days. They will then crossover after a 7 day washout period. The primary endpoint is the percent change in daytime cough frequency (coughs per hour) from baseline as assessed by objective cough monitoring at Day 14 of treatment.Discussion: This multicentre, randomised trial will assess the effect of opioids on cough counts and cough associated quality of life in IPF subjects. If proven to be an effective intervention, it represents a readily available treatment for patients.Trial registration: The study was approved by the UK Medicines and Healthcare Regulatory Agency (Ref: CTA 21268/0224/001-0001 – EUDRACT 2019-003571-19 – Protocol Number RBH2019/001) on 08 April 2020, in compliance with the European Clinical Trials Directive and the Medicines for Human Use (Clinical Trials) Regulations 2004 and its subsequent amendments. The study was provided with ethical approval by the London Brent Research Ethics Committee (Ref: 20/LO/0368) on 21 May 2020 and is registered with clinicaltrials.gov (NCT04429516) on 12 June 2020, available at https://clinicaltrials.gov/ct2/show/NCT04429516

scholarly journals Pelargonium sidoides root extract for the treatment of acute cough due to lower respiratory tract infection in adults: a feasibility double-blind, placebo-controlled randomised trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Merlin Willcox ◽  
Catherine Simpson ◽  
Sam Wilding ◽  
Beth Stuart ◽  
Dia Soilemezi ◽  
...  
Keyword(s):  
Primary Care ◽  
Controlled Trial ◽  
Randomised Trial ◽  
Acute Bronchitis ◽  
Antibiotic Prescribing ◽  
Root Extract ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Link Type ◽  
Pelargonium Sidoides

Abstract Background Pelargonium sidoides DC (Geraniaceae) root extract, EPs®7630 or “Kaloba®”, is a widely used herbal remedy for respiratory infections, with some evidence of effectiveness for acute bronchitis. However, it is not yet widely recommended by medical professionals in the UK. There is a need to undertake appropriately designed randomised trials to test its use as an alternative to antibiotics. The aim was to assess the feasibility of conducting a double-blind randomised controlled trial of Pelargonium sidoides root extract for treatment of acute bronchitis in UK primary care, investigating intervention compliance, patient preference for dosage form and acceptability of patient diaries. Study design Feasibility double-blind randomised placebo-controlled clinical trial. Methods We aimed to recruit 160 patients with cough (≤ 21 days) caused by acute bronchitis from UK general practices. Practices were cluster-randomised to liquid or tablet preparations and patients were individually randomised to Kaloba® or placebo. We followed participants up for 28 days through self-reported patient diaries with telephone support and reviewed medical records at one month. Outcomes included recruitment, withdrawal, safety, reconsultation and symptom diary completion rates. We also assessed treatment adherence, antibiotic prescribing and consumption, mean symptom severity (at days 2–4 after randomisation) and time to symptom resolution. We interviewed 29 patients and 11 health professionals to identify barriers and facilitators to running such a randomised trial. Results Of 543 patients screened, 261 were eligible, of whom 134 (51%) were recruited and 103 (77%) returned a completed diary. Overall, 41% (41/100) of patients took antibiotics (Kaloba® liquid group: 48% [15/31]; placebo liquid group: 23% [6/26]; Kaloba® tablet group: 48% [9/21]; placebo tablet group: 50% [11/22]). Most patients adhered to the study medication (median 19 out of 21 doses taken in week 1, IQR 18–21 - all arms combined). There were no serious adverse events relating to treatment. Most patients interviewed found study recruitment to be straightforward, but some found the diary too complex. Conclusions It was feasible and acceptable to recruit patients from UK primary care to a double-blind placebo-controlled trial of herbal medicine (Kaloba®) for the treatment of acute bronchitis, with good retention and low data attrition. Trial registration HATRIC was registered on the ISRCTN registry (ISRCTN17672884) on 16 August 2018, retrospectively registered. The record can be found at http://www.isrctn.com/ISRCTN17672884.

Supplemental prophylactic intervention for chemotherapy‐induced nausea and emesis (SPICE) trial: Protocol for a multicentre double‐blind placebo‐controlled randomised trial

2018 ◽  
Vol 77 (1) ◽  
pp. 144-150 ◽  
Author(s):  
Wolfgang Marx ◽  
Alexandra McCarthy ◽  
Skye Marshall ◽  
Megan Crichton ◽  
Alex Molassiotis ◽  
...  
Keyword(s):  
Randomised Trial ◽  
Trial Protocol ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Nausea And Emesis

scholarly journals Efficacy and tolerance of early administration of tranexamic acid in patients with cirrhosis presenting with acute upper gastrointestinal bleeding: a study protocol for a multicentre, randomised, double-blind, placebo-controlled trial (the EXARHOSE study)

BMJ Open ◽  
2018 ◽  
Vol 8 (8) ◽  
pp. e021943 ◽  
Author(s):  
Matthieu Heidet ◽  
Roland Amathieu ◽  
Etienne Audureau ◽  
Oriane Augusto ◽  
Violaine Nicolazo de Barmon ◽  
...  
Keyword(s):  
Gastrointestinal Bleeding ◽  
Tranexamic Acid ◽  
Upper Gastrointestinal Bleeding ◽  
Controlled Trial ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Intention To Treat ◽  
Acute Upper Gastrointestinal Bleeding ◽  
Shunt Procedure ◽  
Upper Gastrointestinal

IntroductionThe management of acute upper gastrointestinal bleeding (UGIB) is challenging in patients with cirrhosis, as it is responsible for severe complications and high mortality rates. Tranexamic acid (TXA) may help control the bleeding by counterbalancing cirrhosis-related hyperfibrinolysis. Still, there is a lack of unbiased data to conclude on its efficacy. The aim of this study is to evaluate the efficacy of TXA in the early treatment of acute UGIB in patients with cirrhosis.Methods and analysisThis study is a multicentre, randomised, double-blind, placebo-controlled trial, for adult patients with cirrhosis presenting with an acute UGIB and allocated to one of two arms: TXA or placebo (saline). Physicians from emergency mobile services, emergency departments (EDs) or intensive care units (ICUs) can include patients. Besides study intervention, standard care for UGIB will be performed as recommended. Intervention will consist an intravenous infusion of 10 mL of TXA (1 g) or saline, immediately followed by three identical intravenous infusions over 8 hours each (total dose of 4 g of TXA or 40 mL of placebo over 24 hours). Main analyses will be conducted in intention to treat on every patient included, then in modified intention to treat on patients with underlying lesion of portal hypertension visualised by endoscopy. The main objective is to show efficacy of TXA until day 5 on a composite criterion (bleeding control, rebleeding episodes and mortality). Secondary objectives aim at showing the efficacy of TXA on each individual component of the main outcome measure and others at 6 weeks and later (transjugular intrahepatic portosystemic shunt procedure, cirrhosis-specific complications, length of stay in ICU and in hospital, safety and tolerance of TXA, liver transplantation). Included patients will be followed up to 1 year after inclusion.500 patients will be necessary to show a reduction in the prevalence of the primary outcome from 30% to 18% with a bilateral alpha risk of 5% and a power of 80%.Ethics and disseminationEthical approval has been obtained from the Comité de Protection des Personnes Ile-de-France 1 (CPP-IDF1). Results will be disseminated via publications in peer-review medical journals and scientific forums.Protocol versionThis protocol is based on the latest version, as established on 11 October 2017 and validated by the IRB CPP Ile-de-France 1.Trial registration numberNCT03023189.

Sign in / Sign up

Export Citation Format

Share Document