Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Study Overview
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Brief Summary
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Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3

Detailed Description
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Evaluating the response of 3 dose levels of arimoclomol on various pharmacodynamic biomarkers in blood and cerebrospinal fluid as indicators of enhanced GBA in Gaucher Disease Type 1 or 3.

Official Title
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Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Conditions
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Gaucher Disease, Type 1, Gaucher Disease, Type 3

Intervention / Treatment
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* Drug: Arimoclomol
* Drug: Placebo oral capsule


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Be able to understand and voluntarily sign informed consent A diagnosis GD, either Type 1 or Type 3 For GD3 at least 1 neurological symptom Age ≥ 4 years and ≤ 60 years at the time of enrolment Plasma or serum chitotriosidase activity greater than 3 times the upper limit of normal. Key Exclusion Criteria: Recipient of a liver transplant or planned liver transplantation during the course of the study. Splenectomy within 4 months of study entry or planned splenectomy during the course of the study. Severe liver damage. Severe renal insufficiency. Body weight < 10 kg. Other inclusion and exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Randomized
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arimoclomol I<br>Arimoclomol, oral capsule | Drug: Arimoclomol<br>* Arimoclomol in 3 different dosages<br>|
| Experimental: Arimoclomol II<br>Arimoclomol, oral capsule | Drug: Arimoclomol<br>* Arimoclomol in 3 different dosages<br>|
| Experimental: Arimoclomol III<br>Arimoclomol, oral capsule | Drug: Arimoclomol<br>* Arimoclomol in 3 different dosages<br>|
| Placebo Comparator: Placebo<br>Placebo oral capsule matching experimental arm | Drug: Placebo oral capsule<br>* Matching placebo capsule<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Primary Endpoint | The percentage change in serum chitotriosidase levels from baseline to 6 months | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Growth Endpoint | Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months | 6 months | 
| Growth Endpoint | Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months | 6 months | 
| Maturation Endpoint | Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening | 6 months | 
| Maturation Endpoint | Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV) | 6 - 12 months | 
| Imaging Endpoint | Change in size of liver and spleen assessed by ultrasound | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gaucher Disease