A Phase I Study in Niemann Pick: CTD Holdings

The Challenge

Aptus Clinical was asked by Cyclo Therapeutics to join their International Program Team to support the development of TRAPPSOL® CYCLO® for the treatment of Niemann Pick type C (NPC-1).  Aptus was contracted to support the global delivery of a their Phase I/IIrandomised, double-blind trial with sites in the UK, Israel and Sweden.

NPC-1 is a rare, progressive, genetically inherited, fatal and complex autosomal recessive lysosomal storage disorder.  Patients are treated for their symptoms to help with posture, speech, movement, digestion, breathing among others.

The Solution

  • Step 1 – Providing  strategic input into the development plan for TRAPPSOL® CYCLO® and implementing a quality management infrastructure to support the compliant conduct of the study
  • Step 2  – Global project management and operational support for site set-up, recruitment and monitoring

The Aptus study team was established to support the identification, set-up and  monitoring  of  UK sites. A partner CRO was  carefully selected to support  us in managing the other sites in Europe,  with  full project management oversight provided by Aptus including numerous third-party vendors

The Aptus study team were proactive in seeking and implementing solutions to support patient retention, including

  • Patient travel provision across countries to support adherence to protocol visits and assessments, particularly with the paediatric patient population

The Outcome

  • The Aptus team secured UK ethics approval and NIHR adoption, adapting our resources to support set-up of 3 sites
  • Recruitment was completed, along with the successful delivery of the interim DBL in Q4 2020, which was critical to support the dose for the pivotal Phase III study
  • Following LSLV the final database lock was delivered on time and quality, within 22 calendar days in Q1 2021.  Despite the significant additional challenges of COVID-19, 93% of source data verification was achieved
  • All deliverables including data entry, sample shipment and analysis and central imaging review, were completed effectively and efficiently throughout and were critical success factors in the delivery of interim and final database locks

The Feedback

The client is delighted with the operational progress to date.