Place : Jaslok Hospital and Research Centre, Mumbai
Study – This study is being conducted for the drug “Orphazyme A/S”
Eligibility : The study population required for the study is Male or Female patients between 4- 60 years with diagnosis of GD type 1 or GD type 3.
This is a placebo-controlled, randomised, double-blinded trial for GD type I and 3. Patients who are randomised to receive placebo will be further randomised to receive arimoclomol 300 mg/day, 600 mg/day, or 1200 mg/day (provided there are no safety concerns) on a 1:1:1 basis during the open-label extension phase of the trial.
Duration: The duration of the double-blinded, placebo-controlled period of the trial is 6 months. The open-label extension phase will continue until arimoclomol has received marketing authorisation (MA) in India or availability of relevant data from the development program.
Main inclusion criteria:
- Be able to understand and voluntarily sign the informed consent form.(patient/parent)
- A diagnosis of GD, either Type 1 or Type 3, with a GBA enzyme activity of 0% to 15% of normal activity. Note that the local laboratory test result will be used at inclusion.
- For GD3, at least 1 neurological symptom.
- Age ≥ 4 and ≤ 60 years at the time of enrolment.
- Plasma or serum chitotriosidase levels > 3000 nmol/mL/h.
- Either naïve to treatment for GD or has not received treatment (investigational or authorised/approved such as enzyme replacement therapy or substrate reduction therapy, also including procedures such as blood transfusions and splenectomy) for GD within 4 months prior to trial entry.
- Ability to comply with the protocol-specified procedures/evaluations and scheduled visits (if platelet counts are < 50000 platelets per microlitre of circulating blood lumbar puncture should not be performed).
- Ability to travel to the investigational clinical trial site repeatedly (screening, baseline, 1, 3, 6, and 7 months, then every 6 months during the extension part) for evaluation and follow-up.
- All sexually active female patients of child-bearing potential (postmenarcheal) must use highly effective contraception during the trial and until 1 week after the last dose of IMP.
IMP NOTE
All sexually active male patients with female partners of child-bearing potential (postmenarcheal) must use a condom with or without spermicide in addition to the birth control used by their partners during the trial and until 3 months after the last dose of IMP.
Main exclusion criteria:
- Recipient of a liver transplant or planned liver transplantation during the course of the trial.
- Splenectomy within 4 months of trial entry or planned splenectomy during the course of the trial.
- Aspartate aminotransferase and/or alanine aminotransferase greater than 3 times the upper limit of normal for age and sex [central laboratory assessment]).
- Serum creatinine level greater than 1.5 times the upper limit of normal (central laboratory assessment).
- The patient has received any IMP within 30 days prior to trial entry (Note that IMP for GD should not have been taken within the last 4 months prior to enrolment, as per entry criterion #6).
- The patient is pregnant and/or breastfeeding.
- If, in the opinion of the Principal Investigator, the patient has a clinical condition that is not compatible with the requirements of this protocol.
- Body weight < 10 kg.
Contact Details:
Dr Aabha Nagral
Gastroenterologist
Special interest – Hepatology, Pediatric Hepatology and Liver Transplantation
Apollo Hospital, Navi Mumbai and Jaslok Hospital, Mumbai
For appointments
(022) 24222160 after 4pm (Monday to Friday)
