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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

Page last updated: August 23, 2015

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