Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
39
Arimoclomol in 3 different dosages
Matching placebo capsule
King Edward Memorial Hospital
Mumbai, Maharashtra, India
Jaslok Hospital and Reseach Centre
Mumbai, Maharashtra, India
KEM HOSPITAL Research Centre
Pune, Maharashtra, India
Sir Ganga Ram Hospital
New Delhi, National Capital Territory of Delhi, India
Primary Endpoint
The percentage change in serum chitotriosidase levels from baseline to 6 months
Time frame: 6 months
Growth Endpoint
Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months
Time frame: 6 months
Growth Endpoint
Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months
Time frame: 6 months
Maturation Endpoint
Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening
Time frame: 6 months
Maturation Endpoint
Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)
Time frame: 6 - 12 months
Imaging Endpoint
Change in size of liver and spleen assessed by ultrasound
Time frame: 6 months
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Christian Medical College and Hospital.
Vellore, Tamil Nadu, India
Institute of Child Health
Kolkata, West Bengal, India
Maulana Azad Medical College
New Delhi, India
All India Institute of Medical Sciences
New Delhi, India