Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
Information source: Shire
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease, Type 1
Intervention: velaglucerase alfa (Biological); imiglucerase (Biological)
Phase: Phase 3
Status: Completed
Sponsored by: Shire Official(s) and/or principal investigator(s): Kiran Bhirangi, M.D., Study Director, Affiliation: Shire Human Genetic Therapies, Inc. Priya Kishnani, M.D., Principal Investigator, Affiliation: Duke Children's Hospital & Health Center Isaac Kisinovsky, M.D., Principal Investigator, Affiliation: Your Health S.A. (Hipolito Yrigoyen) Derlis Gonzalez Rodriguez, M.D., Principal Investigator, Affiliation: Sociedad Espanola de Socorros Mutuos Elena A. Lukina, M.D., Principal Investigator, Affiliation: National Research Center for Haematology Atul Mehta, M.D., Principal Investigator, Affiliation: The Royal Free Hospital Ari Zimran, M.D., Principal Investigator, Affiliation: Shaare Zedek Medical Center Pilar Giraldo, M.D., Principal Investigator, Affiliation: Hospital Universitario Miguel Servet Suresh Kumar, M.D., Principal Investigator, Affiliation: Malabar Institute of Medical Sciences Ltd. Madhulika Kabra, M.D., Principal Investigator, Affiliation: All India Institute of Medical Sciences, New Delhi Ashish Bavdekar, M.D., Principal Investigator, Affiliation: KEM Hospital Research Centre Marie-Francoise Ben Dridi, M.D., Principal Investigator, Affiliation: La Rabta Hospital
Summary
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside
accumulates within macrophages leading to cellular engorgement, organomegaly, and organ
system dysfunction. The purpose of this non-inferiority study is to evaluate the efficacy
and safety of GA-GCB (velaglucerase alfa) administered every other week in comparison to
imiglucerase in treatment naive patients with type 1 Gaucher disease.
Clinical Details
Official title: A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease
Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group.
Secondary outcome: Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group.Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group. Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group. Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group. Change From Baseline to Month 9 in Plasma Chemokine (C-C Motif) Ligand 18 (CCL18) for Each Treatment Group. Number of Participants Who Developed Antibody for Each Treatment Group. Time to Response- Comparison of GA-GCB and Imiglucerase on the Earliest Time to Respond as Assessed Via Hemoglobin Concentration
Detailed description:
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and
does not involve the CNS. Typical manifestations of type 1 Gaucher disease include
hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism,
skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life.
Gene-Activated® human glucocerebrosidase (GA-GCB; velaglucerase alfa) is produced in a
continuous human cell line using proprietary gene-activation technology and has an identical
amino acid sequence to the naturally occurring human enzyme. GA-GCB (velaglucerase alfa)
contains terminal mannose residues that target the enzyme to the macrophages-the primary
target cells in Gaucher disease. This study was designed to determine the efficacy and
safety of GA-GCB (velaglucerase alfa) in comparison to imiglucerase in men, women, and
children with Type 1 Gaucher disease.
Eligibility
Minimum age: 2 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria
Includes:
- The patient has a documented diagnosis and clinical manifestation of type 1 Gaucher
disease
- The patient is at least 2 years of age.
- The patient has not received treatment for Gaucher disease (investigational products,
miglustat, or imiglucerase) within 12 months prior to study entry, as documented in
the patient's medical history.
- Female patients of child-bearing potential must agree to use a medically acceptable
method of contraception at all times during the study and must have negative results
to a pregnancy test performed at the time of enrollment and as required throughout
their participation in the study. Male patients must use a medically acceptable
method of birth control throughout their participation in the study and must report
their partner's pregnancy.
- The patient, the patient's parent(s) or legal guardian(s) has provided written
informed consent that has been approved by the Institutional Review Board/Independent
Ethics Committee (IRB/IEC).
- The patient must be sufficiently cooperative to participate in this clinical study as
judged by the Investigator.
Exclusion Criteria
Includes:
- The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher
disease.
- The patient has received treatment with any non-Gaucher disease-related
investigational drug or device within the 30 days prior to study entry; such use
during the study is not permitted.
- The patient is known to be positive for human immunodeficiency virus (HIV).
- The patient is known to be positive for hepatitis B and/or C.
- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to
understand the nature, scope, and possible consequences of the study.
- The patient has a significant comorbidity(ies) that might affect study data or
confound the study results (e. g., malignancies, primary biliary cirrhosis, autoimmune
liver disease, etc.).
- The patient is unable to comply with the protocol, e. g., has a clinically relevant
medical condition making implementation of the protocol difficult, has an
uncooperative attitude, is unable to return for safety evaluations, or is otherwise
unlikely to complete the study, as determined by the Investigator.
Locations and Contacts
Your Health S.A., Buenos Aires B1882AQY, Argentina
All India Institute of Medical Sciences, New Delhi 110 029, India
KEM Hospital Research Centre, Pune, India
Shaare Zedek Medical Center, Jerusalem, Israel
Sociedad Espanola de Socorros Mutuos, Asuncion, Paraguay
National Research Center for Haematology, Moscow, Russian Federation
Hospital Universitario Miguel Servet, Zaragoza, Spain
La Rabta Hospital, Tunis, Tunisia
The Royal Free Hospital, London, United Kingdom
Malabar Institute of Medical Sciences Ltd., Calicut, Kerala 673 016, India
Duke Children's Hospital & Health Center, Durham, North Carolina 27710, United States
Additional Information
Starting date: January 2008
Last updated: July 16, 2015
|