Rare Disease Studies

Supporting rare and orphan lung disease clinical trials.

What Vitalograph Offer

We offer a full range of specialised pulmonary function tests supported by key opinion leaders. We are flexible and work with you to develop customised solutions for the unique nature of trials in rare and orphan diseases.

We provide:

Over-reading (QA review) for spirometry and MIP/MEP.
NIOX VERO ® centralized FeNO data capture to assess underlying inflammation.
Respiratory Muscle Strength assessment.
Specialized tests to measure peak cough flow (PCF), IFR, SNIP.
Home monitoring through patient reported outcome questionnaires and lung function endpoints.
eCOA and ePRO through our In2itive e-Diary to meet study needs.
Adaptable reports for data monitoring and analysis. 
Access to subject matter experts throughout the life of your study
Software is adapted to modified ATS/ERS criteria for the study population
Clinical trial devices and software.
Professional and dedicated study services.

Our Experience

We have extensive experience in supporting niche studies and Rare Disease respiratory clinical trials.

We have provided flexible and specialist support to rare disease clinical trials and orphan disease clinical trials. Examples include: Duchenne Muscular Dystrophy (DMD), POMPE, Limb-Girdle Muscular Dystrophy and Systemic lupus erythematosus (SLE). 

Our flexible and adaptable systems allow us to deliver data tailored to your requirements for Phase I – IV, adult and pediatric trials. We work closely with KOLs who can support your protocol development.

 Internationally renowned Over-readers ensure high-quality data and feedback for your trial, which improves site data, compliance, and performance.

A highly qualified and responsive team of project and data managers will be dedicated to your study, allowing for informed decisions to be made quickly and for prompt response and feedback to any queries. It also ensures key milestones and timelines are met.

We assure the rapid delivery of your rare or orphan disease clinical trial.

Objectives & Endpoints

Accurate, reliable and precise capture of PFT data for rare and orphan disease clinical trials.

*Criteria*	*Example Endpoints*
*Subjective Endpoints*	Symptom scores Study medication use eCOA and questionnaires can be integrated to meet study needs
*Objective Endpoints*	In clinic Lung Function Testing: PEF VC FVC FVC% Predicted FEV1 FEV1% Predicted Sitting and Supine Spirometry Respiratory Muscle Testing: MEP, MIP, SNIP, PCF, IFR Home monitoring FEV1 & PEF 6MWD