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

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

Sponsors

Lead sponsor: Orphazyme

Source Orphazyme
Brief Summary

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

Detailed Description

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.

Overall Status Active, not recruiting
Start Date June 6, 2018
Completion Date June 1, 2021
Primary Completion Date June 1, 2020
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Primary Endpoint 6 months
Secondary Outcome
Measure Time Frame
Growth Endpoint 6 months
Growth Endpoint 6 months
Maturation Endpoint 6 months
Maturation Endpoint 6 - 12 months
Imaging Endpoint 6 months
Enrollment 39
Condition
Intervention

Intervention type: Drug

Intervention name: Arimoclomol

Description: Arimoclomol in 3 different dosages

Intervention type: Drug

Intervention name: Placebo oral capsule

Description: Matching placebo capsule

Arm group label: Placebo

Eligibility

Criteria:

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

Gender: All

Minimum age: 4 Years

Maximum age: 60 Years

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Aabha Nagral, MD Principal Investigator Jaslok Hospital and Reseach Centre, Mumbai
Location
facility
Sir Ganga Ram Hospital | New Delhi, Delhi, 110060, India
King Edward Memorial Hospital | Mumbai, Maharashtra, 4000012, India
Jaslok Hospital and Reseach Centre | Mumbai, Maharashtra, 400026, India
KEM HOSPITAL Research Centre | Pune, Maharashtra, 411011, India
Christian Medical College and Hospital. | Vellore, Tamilnadu, 632004, India
Institute of Child Health | Kolkata, West Bengal, 700017, India
Maulana Azad Medical College | New Delhi, 110002, India
All India Institute of Medical Sciences | New Delhi, 110029, India
Location Countries

India

Verification Date

September 2019

Responsible Party

Responsible party type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 4
Arm Group

Arm group label: Arimoclomol I

Arm group type: Experimental

Description: Arimoclomol, oral capsule

Arm group label: Arimoclomol II

Arm group type: Experimental

Description: Arimoclomol, oral capsule

Arm group label: Arimoclomol III

Arm group type: Experimental

Description: Arimoclomol, oral capsule

Arm group label: Placebo

Arm group type: Placebo Comparator

Description: Placebo oral capsule matching experimental arm

Patient Data Undecided
Study Design Info

Allocation: Randomized

Intervention model: Parallel Assignment

Intervention model description: Randomized

Primary purpose: Treatment

Masking: Triple (Participant, Care Provider, Investigator)

Masking description: Double-blinded, randomized, placebo controlled

Source: ClinicalTrials.gov