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

Gaucher Disease, Type 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03746587
• Other study ID #: OR-ARI-GAU-01
• Status: Terminated
• Phase: Phase 2
• Start date: June 6, 2018
• Est. completion date: October 15, 2021

Study information

• Verified date: January 2023
• Source: KemPharm Denmark A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 39
• Est. completion date: October 15, 2021
• Est. primary completion date: April 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 60 Years

Eligibility

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

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type 1
• Gaucher Disease, Type 3

Intervention

Drug:
• Arimoclomol
Arimoclomol in 3 different dosages
• Placebo oral capsule
Matching placebo capsule

Locations

Country	Name	City	State
India	Institute of Child Health	Kolkata	West Bengal
India	Jaslok Hospital and Reseach Centre	Mumbai	Maharashtra
India	King Edward Memorial Hospital	Mumbai	Maharashtra
India	All India Institute of Medical Sciences	New Delhi
India	Maulana Azad Medical College	New Delhi
India	Sir Ganga Ram Hospital	New Delhi	Delhi
India	KEM HOSPITAL Research Centre	Pune	Maharashtra
India	Christian Medical College and Hospital.	Vellore	Tamilnadu

Sponsors (1)

Lead Sponsor	Collaborator
KemPharm Denmark A/S

Country where clinical trial is conducted

India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Primary Endpoint	The percentage change in serum chitotriosidase levels from baseline to 6 months	6 months
Secondary	Growth Endpoint	Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months	6 months
Secondary	Growth Endpoint	Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months	6 months
Secondary	Maturation Endpoint	Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening	6 months
Secondary	Maturation Endpoint	Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)	6 - 12 months
Secondary	Imaging Endpoint	Change in size of liver and spleen assessed by ultrasound	6 months