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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03746587
Recruitment Status : Active, not recruiting
First Posted : November 19, 2018
Last Update Posted : September 4, 2019
Sponsor:
Information provided by (Responsible Party):
Orphazyme

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

Condition or disease Intervention/treatment Phase
Gaucher Disease, Type 1 Gaucher Disease, Type 3 Drug: Arimoclomol Drug: Placebo oral capsule Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Double-blinded, randomized, placebo controlled
Primary Purpose: Treatment
Official Title: Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Actual Study Start Date : June 6, 2018
Estimated Primary Completion Date : June 1, 2020
Estimated Study Completion Date : June 1, 2021


Arm Intervention/treatment
Experimental: Arimoclomol I
Arimoclomol, oral capsule
Drug: Arimoclomol
Arimoclomol in 3 different dosages

Experimental: Arimoclomol II
Arimoclomol, oral capsule
Drug: Arimoclomol
Arimoclomol in 3 different dosages

Experimental: Arimoclomol III
Arimoclomol, oral capsule
Drug: Arimoclomol
Arimoclomol in 3 different dosages

Placebo Comparator: Placebo
Placebo oral capsule matching experimental arm
Drug: Placebo oral capsule
Matching placebo capsule




Primary Outcome Measures :
  1. Primary Endpoint [ Time Frame: 6 months ]
    The percentage change in serum chitotriosidase levels from baseline to 6 months


Secondary Outcome Measures :
  1. Growth Endpoint [ Time Frame: 6 months ]
    Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months

  2. Growth Endpoint [ Time Frame: 6 months ]
    Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months

  3. Maturation Endpoint [ Time Frame: 6 months ]
    Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening

  4. Maturation Endpoint [ Time Frame: 6 - 12 months ]
    Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)

  5. Imaging Endpoint [ Time Frame: 6 months ]
    Change in size of liver and spleen assessed by ultrasound



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Ages Eligible for Study:   4 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03746587


Locations
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India
Sir Ganga Ram Hospital
New Delhi, Delhi, India, 110060
King Edward Memorial Hospital
Mumbai, Maharashtra, India, 4000012
Jaslok Hospital and Reseach Centre
Mumbai, Maharashtra, India, 400026
KEM HOSPITAL Research Centre
Pune, Maharashtra, India, 411011
Christian Medical College and Hospital.
Vellore, Tamilnadu, India, 632004
Institute of Child Health
Kolkata, West Bengal, India, 700017
Maulana Azad Medical College
New Delhi, India, 110002
All India Institute of Medical Sciences
New Delhi, India, 110029
Sponsors and Collaborators
Orphazyme
Investigators
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Principal Investigator: Aabha Nagral, MD Jaslok Hospital and Reseach Centre, Mumbai

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Responsible Party: Orphazyme
ClinicalTrials.gov Identifier: NCT03746587     History of Changes
Other Study ID Numbers: OR-ARI-GAU-01
First Posted: November 19, 2018    Key Record Dates
Last Update Posted: September 4, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Orphazyme:
Gaucher Disease
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders