Health Condition 1: null- Gaucher disease Type 1 and Type 3 Health Condition 2: E752- Other sphingolipidosis
Conditions
Interventions
Intervention1: Arimoclomol: Arimoclomol N-{[(2R)-2-hydroxy-3-piperidin-1ylpropyl]oxy}pyridine-3-carboximidoyl chloride 1-oxide, citrate is a hydroximic acid derivative with pharmacologic activity that
Sponsors
Orphazyme AS
Covance India Pharmaceutical Services Private Limited
Eligibility
Inclusion criteria
Inclusion criteria: The following are the main inclusion criteria: 1. Be able to understand and voluntarily sign informed consent. 2. A diagnosis GD, either Type 1 or Type 3, with a glucocerebrosidase enzyme activity 0 to 15% of normal activity. Note that the local laboratory test result will be used at inclusion. 3. For GD3 at least 1 neurological symptom. 4. Age >= 4 years and 5. Plasma or serum chitotriosidase levels > 3000 nmol/mL/h. Historical data is acceptable. 6. Either naïve to treatment for GD or has not received treatment (investigational or authorized/approved like ERT or SRT, also including procedures such as blood transfusions and splenectomy) for Gaucher disease within 4 months prior to study entry. 7. Ability to comply with the protocol-specified procedures/evaluations and scheduled visits; (if platelet counts are 8. Ability to travel to the investigational clinical trial site repeatedly (screening, baseline, 1 month, 3 months, and 6 months, then every 6 months during the extension part) for evaluation and follow-up. 9. All sexually active female patients of child-bearing potential (post-menarchal) must use highly effective contraception during the study and until 1 week after the last dose of IMP. All sexually active male patients with female partners of child-bearing potential (post-menarchal) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of IMP.
Exclusion criteria
Exclusion criteria: 1. Recipient of a liver transplant or planned liver transplantation during the course of the study. 2. Splenectomy within 4 months of study entry or planned splenectomy during the course of the study. 3. Severe liver damage (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal for age and gender (central laboratory assessment). 4. Severe renal insufficiency, with serum creatinine level greater than 1.5 times the upper limit of normal (ULN) (central laboratory assessment). 5. The patient has received any investigational drug within 30 days prior to study entry (Note that investigational drug for GD should not have been taken within the last 4 months prior to enrolment, as per entry criterion no.6). 6. The patient is a pregnant and/or lactating female. 7. If, in the opinion of the principal investigator, the patient has a clinical condition that is not compatible with the requirements of this protocol; such as confirmed history of serious adverse reaction to sedation or anesthesia (if sedation is necessary for lumbar puncture), uncontrolled severe epileptic seizures and/or severe malnutrition. 8. Body weight less than 10 kg.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of the study is to evaluate the response in GD biomarkers versus placebo after 6 months of treatment.Timepoint: After 6 months of treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary objectives include the evaluation of patient safety (after 6 months and long term) as well as long term effects on clinical endpoints and biomarkers during the extension phase.Timepoint: After 6 months of treatment and long-term | — |
Countries
India
Contacts
Public ContactShekhar Dawkhar
Covance (Asia) Pte. Ltd.
Outcome results
None listed