Simbec-Orion Case Study: Rare & Orphan Disease

CASE STUDY THERAPY AREA: RARE & ORPHAN DISEASE SUPERIOR STAKEHOLDER COORDINATION ENSURES RAPID STUDY SUCCESS FOR AN ULTRA-RARE INDICATION IN PAEDIATRIC PATIENTS

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CASE STUDY A STUDY IN PATIENTS DIAGNOSED WITH NIEMANN-PICK DISEASE TYPE C TO CHARACTERISE THE INDIVIDUAL PATIENT DISEASE PROFILE AND HISTORIC SIGNO-SYMPTOMATOLOGY PROGRESSION PATTERN, LEADING TO AN INTERVENTIONAL STUDY.

SPONSOR OBJECTIVES: The sponsor needed to work with a CRO who could identify centres where patients with this ultra-rare disease are diagnosed and treated to meet the scientific objectives of their study, and their patient’s medical needs. The observational study needed to be completed in order to move to an interventional study with IMP.

CHALLENGES OF THE STUDY: •

Niemann–Pick type C affects an estimated 1:150,000 people. 50% of cases present before the age of 10, complex recruitment regulatory and logistical co -ordination were required to meet the sponsor objectives.

• To characterize the individual patient disease progression profile (disease burden and progression) through the clinical, imaging, biological status, and quality of life prospectively recorded, together with the historic disease information collected from patient medical records.

• To evaluate the safety data of the disease-related therapy and to record every adverse event (AE) linked to the disease.

PATIENT PROFILE • Paediatric males and females aged from 2 years to 18 years and, with a Diagnosis of Niemann-Pick disease type C (NP-C),NPC1/NPC2.

DRUG TYPE Recombinant humanised protein.

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CASE STUDY STUDY CHALLENGES: Simbec- Orion were tasked to find centres where children with this ultra-rare disease are diagnosed and treated.

The children had to be mobilised and recruitment had to be coordinated with the centres to be able to enrol patients in the shortest possible time.

Due the complex biological markers, a central lab was required to coordinate the sampling, delivery and process of the samples within a few hours after sampling.

Upon interaction with the Paediatric Committee (PDCO), the protocol of the interventional study had to be amended and resubmitted to all the Competent Authorities (CA) and Ethical Committees (EC). This was a critical activity to avoid delay.

OUR SOLUTIONS: Simbec-Orion deployed our seasoned CRAs in more than 18 European countries, to evaluate the centres of excellence with capabilities to perform this complex clinical trial.

Because the sponsor had to produce a new batch of the product with concentrations adapted to different doses based on the weight of the paediatric patients, an observational study was initiated with 2 main objectives: collect information on disease progression and include patients in the clinical development programme.

Simbec-Orion organised the sample preparation under special conditions, precisely coordinating the time-critical delivery to our central laboratory division, Seirian Laboratories. Our regulatory team prepared the necessary documentation and directly interacted with each of the clinical authorities and ethics committees to promptly address any potential concerns. We took an active role in the writing of the protocol, and were responsible for designing the eCRF. As well as collaborating with the sponsor to prepare the innovative statistical plan.

We designed the PICF (Patient Informed Consent Form) in all the required languages, adapting them to be suitable for the different paediatric age groups concerned.

RESULT FOR CUSTOMER: •

Rapid identification of the centres of expertise in the selected disease area.

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Mobilising the patients and families to be included in the study and provide the appropriate legal support to adapt the sites contracts upon the individual requirements.

We were able to achieve this due to our extensive experience regarding the stringent quality requirements in rare diseases clinical research as well as the special requirements of paediatric patients.

The observational study was swiftly integrated into the interventional study once the investigational medicinal product (IMP) was ready and the drug supply was organised.

Simbec-Orion worked as true partners with the sponsor adapting to the evolving reality while surpassing timelines. The observational study was completed and the interventional study initiated in a record time of less than 10 months.

The sponsor was able expand the Phase II/III interventional study in the US and Europe and we are pleased to say that enrolment for the interventional study has been completed in April 2017 in record time.

Number of countries:

11

Number of sites:

16

Number of enrolled patients:

33

Observational study completed in:

<10 MONTHS

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MISSION STATEMENT We are Simbec-Orion. An international, full service, boutique CRO; growing by bringing together the best possible people, healthcare professionals and drug developers, from all areas of clinical development.

We focus on a defined series of core therapeutic areas, where we can make best use of our skills elegantly to design, execute and deliver our clientsâ&#x20AC;&#x2122; clinical development needs.

We are making Simbec-Orion a highly respected and profitable boutique CRO. We do this by working for our clients with excellence, commitment and passion for our trade. We provide an environment in which our colleagues can continue to grow and develop.

We will always remember that our work leads to the improvement of patientsâ&#x20AC;&#x2122; lives

To find out how Simbec-Orion can help with your clinical development program email info@SimbecOrion.com Or call our business development team on +44 1753 578080 FRONT COVER IMAGE: NIEMANN PICK TYPE B CELLS

Simbec-Orion 7 Bath Road, Slough Berkshire SL1 3UA United Kingdom