IntraBio Ltd. is pleased to share that the IB1001-201 clinical trial has screened two-thirds (²/₃) the target number of patients.

The study, which investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), will enroll a total of approximately 30 patients across all international sites. Since recruitment commenced in September 2019, twenty (20) patients have been screened across nine (9) international clinical trial sites.

Recruitment is expected to continue rapidly and be completed by December 2019.

To ensure all patients have the opportunity to participate, interested patients are encouraged to contact their nearest study center as soon as possible to schedule a screening visit before enrollment is complete.

Information on each site, including contact details, can be found below.

GERMANY

Ludwig Maximilian University of Munich
Patients aged 6 Years +
Principal Investigator: Susanne Schneider, MD
Contact: Anita Hennig, MD
Email: Anita.Hennig@med.uni-muenchen.de
Tel: +49(0)89 4400 76777

University of Giessen
Patients aged 6 Years +
Principal Investigator: Andreas Hahn, MD
Contact: Kyriakos Martakis, MD
Email: Kyriakos.Martakis@paediat.med.uni-giessen.de
Tel: +49 (0)641 985 43543

SLOVAKIA

Comenius University in Bratislava
Patients aged 6 -17 Years
Principal Investigator: Miriam Koníková, MUDr, PhD
Sub-Investigator: Tomáš Foltán, MUDr
Contact: Katarína Cichovská
Email: katarina.cichovska@dfnsp.sk
Tel: +421 2 59371 274

SPAIN

Bellvitge University Hospital
Patients aged 6 Years +
Principal Investigator: Jordi Gascon Bayarri, MD
Email:  jordigneuro@bellvitgehospital.cat
Tel: +34 932 607500 ext 2958

UNITED KINGDOM

Great Ormond Street Hospital
Patients aged 6 – 17 Years
Principal Investigator: Paul Gissen, MD
Contact: Olivia Rosie-Wilkinson, Senior Research Coordinator
Email: Olivia.Rosie-Wilkinson@gosh.nhs.uk
Tel: +44 (0)2074 059200 ext. 47267

Royal Manchester Children’s Hospital
Trial Site for Patients Aged 6 – 17 Years
Principal Investigator: Simon Jones, MD
Contact: Laura Crowther, Clinical Trials Manager
Email: laura.crowther@mft.nhs.uk
Tel: +44 (0)161 701 9137

Royal Free London NHS Foundation Trust
Trial Site for Patients Aged 13 Years +
Principal Investigator: Uma Ramaswami, MD
Contact: Masoud Kazemi, Senior Research Lead
Email: masoud.kazemi@nhs.net
Tel: +44 (0) 207 830 2814

Salford Royal NHS Foundation Trust
Trial Site for Patients Aged 18 Years +
Principal Investigator: Reena Sharma, MD
Contact: Marie Meehan, Senior Clinical Research Practitioner/ Team Manager
Email: Marie.Meehan@srft.nhs.uk
Tel: +44 (0)161 206 4192

UNITED STATES

The Mayo Clinic (Rochester, MN)
Patients 18 Years +
Principal Investigator: Anhar Hassan, MD
Contact: Sandra Looney, Clinical Research Coordinator
Email: Looney.Sandra@Mayo.edu
Tel: +1 (507) 538-4107       

About IB1001-201
IB1001-201 investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). This international trial is being run in clinical centers in the United States, the United Kingdom, Germany, Spain, and Slovakia. In Europe, patients aged 6 and older with a confirmed diagnosis of NPC may be eligible for recruitment.

The trial will consist of three study phases: a baseline period, a 6-week treatment period, and a 6-week post-treatment washout period followed by a planned one-year extension phase. Patients (and a caregiver) will be reimbursed for reasonable out-of-pocket expenses incurred for participating in the trial, such as travel and parking.

Additional information on Clinical Trial Study IB1001-201 can be found via ClinicalTrials.Gov(NCT03759639).