Clinical Trials Logo

Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT03746587
Other study ID # OR-ARI-GAU-01
Secondary ID
Status Terminated
Phase Phase 2
First received
Last updated
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
Health authority
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

Study Design


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
See also
  Status Clinical Trial Phase
Active, not recruiting NCT00943111 - A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) Phase 3
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Active, not recruiting NCT00891202 - A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) Phase 3
Recruiting NCT05253560 - Prodromal Parkinsonian Features in GBA1 Mutation Carriers
Completed NCT00813865 - A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients Phase 2
Completed NCT00433147 - A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy Phase 2
Completed NCT00635427 - An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease Phase 3
Completed NCT03950050 - Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy Phase 2
Active, not recruiting NCT00358150 - A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients Phase 2
Completed NCT00446550 - A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease Phase 2
Recruiting NCT05447494 - Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease Phase 1/Phase 2
Completed NCT00553631 - Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease Phase 3
Completed NCT03333447 - Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
Completed NCT04120506 - Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) Phase 4
Completed NCT04353466 - Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs N/A
Completed NCT00430625 - A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease Phase 3
Withdrawn NCT00795197 - A Screening Study Evaluating Disease Status of Gaucher Type I Patients N/A
Completed NCT00364858 - Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease Phase 4
Recruiting NCT05487599 - A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) Phase 1/Phase 2
Recruiting NCT05324943 - A Gene Therapy Study in Patients With Gaucher Disease Type 1 Phase 1