Scientific Publications

2023

Intravenous Trappsol® Cyclo ™ in patients with Niemann Pick Disease type C1: Updates on the Results from Phase I and Phase I/II studies and the international Phase III pivotal Transport NPC trial

          NP Association of China, Patient Group Meeting, July 16, 2023    
          Authors: Andreas Brecht, PhD – Director Medical Affairs Europe Cyclo Therapeutics

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial

Authors: Reena Sharmaa / Caroline Hastings / Orna Staretz-Chachamc / Julian Raimand / Martin Paucar / Ronen Spiegel /
Bryan Murray / Bryan Hurst / Benny Liu / Lise Kjems / Sharon Hrynkow

TransportNPC: A Phase 3 Global Trial of Trappsol® Cyclo™ Administered Intravenously to Patients with Niemann-Pick Disease Type C1 (NPC1)

          Authors: Joseph Mejia, MD / Lise Kjems, PhD, MD / Rebecca Fine / Caroline Hastings, MD

2022

 Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial

          Authors: Caroline Hastings, MD / Benny Liu, MD / Bryan Hurst / Gerald F. Cox, MD, PhD / Sharon Hrynkow, PhD

Intravenous Trappsol® Cyclo™in patients with Niemann Pick Disease type C1: Updates on the Results from Phase I and Phase I/II studies and launch of the international Phase III pivotal Transport NPC trial

• • • NNPDF/INPDA Annual Meeting, Orlando, FL
    • Caroline Hastings, MD / Lise Kjems, MD, PhD
    • July 30, 2022

From Bench to Bedside – A Review of the Trappsol^® Cyclo™ Program for the Treatment of Niemann Pick Disease Type C

• • • World Orphan Drug Congress USA 2022
    • Lise Kjems, MD, PhD
    • July 13, 2022

2021

Topline Results from Phase 1/2 Study to Evaluate Safety and Efficacy of Intravenous Trappsol^® Cyclo™ in Patients with Niemann-Pick Type C1 Disease

• • • Australian NPC Disease Foundation Annual Conference
    • Julian Raiman, MD
    • June 25, 2021

• • • Australian NPC Disease Foundation Annual Conference
    • Caroline Hastings, MD
    • June 25, 2021

Phase 3 Study to Evaluate Intravenous Trappsol^® Cyclo™ Versus Placebo in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

• • • Australian NPC Disease Foundation Annual Conference
    • Gerald Cox, MD, PhD
    • June 25, 2021

• • • WORLDSymposium presentation
    • Julian Raiman, MD
    • Feb 9, 2021

Update on Phase 1 study to evaluate the single- and multiple-dose pharmacokinetics of intravenous hydroxypropyl betacyclodextrin (HPβCD) in patients with Niemann-Pick disease type C (NPC-1) and the effects of dosing upon biomarkers of NPC disease (NCT02939547)

• • WORLDSymposium presentation
  • Caroline Hasting, MD
  • Feb 9, 2021

2020

• • NNPDF Presentation
  • Julian Raiman, MD
  • Jul 9, 2020

Top Line Results of Phase I Intravenous Trial for NPC

• • NNPDF Presentation
  • Caroline Hasting, MD
  • Jul 9, 2020