| CTRI Number |
CTRI/2009/091/000695 [Registered on: 09/09/2009] |
| Last Modified On: |
22/05/2024 |
| Post Graduate Thesis |
No |
| Type of Trial |
Observational |
Type of Study
Modification(s)
|
Case Control Study |
| Study Design |
Single Arm Study |
Public Title of Study
Modification(s)
|
A Screening Study Evaluating Disease Status of Gaucher Type I Patients |
Scientific Title of Study
Modification(s)
|
A screening protocol to characterize the disease status of Gaucher Type I patients for potential inclusion in a subsequent phase 3 clinical study.
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| Trial Acronym |
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Secondary IDs if Any
|
| Secondary ID |
Identifier |
| GZGD02808 |
Protocol Number |
| NCT00795197 |
ClinicalTrials.gov |
|
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Details of Principal Investigator or overall Trial Coordinator (multi-center study)
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| Name |
|
| Designation |
|
| Affiliation |
|
| Address |
Not Applicable
N/A
India |
| Phone |
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| Fax |
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| Email |
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Details of Contact Person
Scientific Query
Modification(s)
|
| Name |
Dr Ravinder Makkar |
| Designation |
Medical Director |
| Affiliation |
Medical Director |
| Address |
Genzyme India, 1st Floor, Technopolis,
Golf Course Road, Sec-54
Gurgaon
HARYANA
122001
India |
| Phone |
0124-4528323 |
| Fax |
0124-4528400 |
| Email |
ravinder.makkar@genzyme.com |
|
Details of Contact Person
Public Query
Modification(s)
|
| Name |
Dr Ravinder Makkar |
| Designation |
Medical Director |
| Affiliation |
Medical Director |
| Address |
Genzyme India Pvt. Ltd.1st Floor, Technopolis
Golf Course Road, Sector-54
Gurgaon
HARYANA
122001
India |
| Phone |
0124-4528323 |
| Fax |
0124-4528400 |
| Email |
ravinder.makkar@genzyme.com |
|
Source of Monetary or Material Support
Modification(s)
|
| Genzyme Europe B.V.
Gooimeer 10, 1411 DD Naarden, The Netherlands |
|
Primary Sponsor
Modification(s)
|
| Name |
Genzyme Europe BV |
| Address |
Gooimeer 10 1411 DD Naarden The Netherlands |
| Type of Sponsor |
Pharmaceutical industry-Global |
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Details of Secondary Sponsor
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Countries of Recruitment
|
India |
Sites of Study
Modification(s)
|
| No of Sites = 4 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Sumita Danda |
Christian Medical College & Hospital |
Christian Medical College & Hospital
Department of Gastrointestinal Sciences
Vellore-632004
Vellore
TAMIL NADU |
0416-2282148
0416-2232035
sdanda@cmcvellore.ac.in |
| Dr Aabha Nagral |
Jaslok Hospital & Research Centre |
Jaslok Hospital & Research Centre
Department - Hepatology
15-Dr.Deshmukh Marg
Pedder road
Mumbai-400026
Mumbai
MAHARASHTRA |
022-24370066
022-23520508
aabhanagral@gmail.com |
| Dr Shubha R Phadke |
Sanjay Gandhi Postgraduate Institute of Medical Sciences |
Sanjay Gandhi Postgraduate Institute of Medical Sciences
Department of Medical Genetics
Raebareli Road
Lucknow-226014
Lucknow
UTTAR PRADESH |
0522-2668005-08
0522-2668017
shubha@sgpgi.ac.in |
| Dr Ishwar Chander Verma |
Sir Gangaram Hospital |
Sir Ganga Ram Hospital
Dept. of Genetic Medicine
Rajinder Nagar
New Delhi-110060
India
New Delhi
DELHI |
011-25861767
011-26589474
icverma@vsnl.com |
|
Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 4 |
| Name of Committee |
Approval Status |
| Christian Medical College Institutional Review Board, Vellore |
Approved |
| Ethics Committee, Jaslok Hospital & Research Centre, Mumbai |
Approved |
| Ethics Committee- Sir Gangaram Hospital |
Approved |
| Institutional Ethics Committee for Human Research, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow |
Approved |
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Regulatory Clearance Status from DCGI
Modification(s)
|
| Status |
| No Objection Certificate |
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Health Condition / Problems Studied
Modification(s)
|
|
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Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
NA |
NA |
| Comparator Agent |
NA |
NA |
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Inclusion Criteria
Modification(s)
|
| Age From |
16.00 Year(s) |
| Age To |
65.00 Year(s) |
| Gender |
Both |
| Details |
1) The patient (or their parent/legal guardian) is willing and able to provide written informed consent.
2) The patient has the following manifestations of Gaucher Type 1 disease identified prior to
screening:
A. Splenomegaly
B. Hepatomegaly
C. Anemia and/or thrombocytopenia
3) The patient is 16 to 65 years of age. For patients <18 years of age, the patient?s Tanner stage should
be ≥ 4.
|
|
| ExclusionCriteria |
| Details |
The patient has had a partial splenectomy within 36 months prior to screening or has had a total
splenectomy.
2) The patient has received miglustat within 3 months prior to screening.
3) The patient has received enzyme replacement therapy within 9 months prior to screening.
4) The patient is known to have any evidence of neurologic (e.g., peripheral neuropathy, tremor,
seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonary
hypertension) as related to Gaucher Type 1 disease.
5) The patient has documentation of new pathological bone involvement (e.g. osteonecrosis,
pathological fractures, etc.) or has a bone crisis (pain with acute onset which requires
immobilization of the affected area, narcotics for relief of pain, and may be accompanied by
periosteal elevation, increased white cell count, fever, or immobility of > 3 days) in the 12 months
prior to screening.
6) The patient is transfusion-dependent.
7) The patient has ever had any radiation treatment.
8) The patient is known to have prior esophageal varices or liver infarction.
9) The patient is known to have a clinically significant disease, other than Gaucher Type 1 disease,
including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine,
metabolic, or psychiatric disease, other medical condition, or serious intercurrent illness.
10) The patient is known to have any of the following: Clinically significant family history (sudden
cardiac death in 1st or 2nd degree relatives), cardiac medical history (including myocardial
infarction [MI] in the past year), or cardiac assessments/symptoms consistent with ischemia.
11) The patient is known to have any of the following: Specific arrhythmias or findings on cardiac
monitoring such as severe 1st degree atrioventricular (AV) block, any 2nd or 3rd degree AV
block, highly frequent or runs (3 or more) of atrial premature contractions (APCs) or premature
ventricular contractions (PVCs), complete left and right as well as partial left bundle branch block, or prolonged QT interval.
12) The patient has previously tested positive for the human immunodeficiency virus (HIV) antibody,
Hepatitis C antibody, or Hepatitis B surface antigen.
13) The patient has received an investigational product within 30 days prior to screening.
14) The patient has a history of cancer, with the exception of basal cell carcinoma.
15) The patient is pregnant or lactating.
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Method of Generating Random Sequence
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Not Applicable |
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Method of Concealment
|
Not Applicable |
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Blinding/Masking
|
Not Applicable |
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Primary Outcome
|
| Outcome |
TimePoints |
| A gaucher enzyme assay will be performed to confirm deficiency of glucocerebrosidase activity if no historic data is available |
Approx. 1 week |
|
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Secondary Outcome
|
| Outcome |
TimePoints |
| Spleen and liver volumes will be assessed using spiral computed tomography (CT) scan or magnetic resonance imaging (MRI) (preferred method). |
Approx. 1 week |
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Target Sample Size
Modification(s)
|
Total Sample Size="100"
Sample Size from India="20"
Final Enrollment numbers achieved (Total)= ""
Final Enrollment numbers achieved (India)="" |
|
Phase of Trial
|
N/A |
Date of First Enrollment (India)
Modification(s)
|
16/03/2009 |
| Date of Study Completion (India) |
Date Missing |
| Date of First Enrollment (Global) |
16/03/2009 |
| Date of Study Completion (Global) |
Date Missing |
|
Estimated Duration of Trial
|
Years="0"
Months="6"
Days="0" |
Recruitment Status of Trial (Global)
Modification(s)
|
Completed |
| Recruitment Status of Trial (India) |
Completed |
Publication Details
Modification(s)
|
Not yet available |
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Individual Participant Data (IPD) Sharing Statement
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Will individual participant data (IPD) be shared publicly (including data dictionaries)?
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Brief Summary
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Short description of the primary purpose of the protocol, including a brief statement of the study hypothesis.
This will be a multi-center, multi-national screening study to assess the disease status of patients with Gaucher Type 1 disease utilizing criteria to be eligible for enrollment in a subsequent Phase 3 clinical study. The assessments performed in this study are standard of care for the evaluation of the symptoms of Gaucher Type 1 disease. No study drug will be administered in this screening study.
Target sample size for India is 20 patients.
The date of enrollment in India is 16 March 2009
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