Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

NCT01685216 | Completed Phase 1 Results

• 2 other identifiers: HGT-GCB-0682012-003427-38
• Study Type: interventional
• Target: 7
• Locations: 3 countries
• Sites: 5

Brief Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and the severity of these neurological symptoms. Patients with type 2 Gaucher disease present with acute neurological deterioration, and those with type 3 disease typically display a more sub acute neurological course. Type 1 Gaucher disease, the most common form accounting for more than 90% of all Gaucher disease cases, does not involve the central nervous system. The purpose of this clinical research study is to investigate the safety and effectiveness of velaglucerase alfa in patients with type 3 Gaucher disease.

Conditions

Gaucher Disease, Type 3

Keywords

VPRIV; velaglucerase alfa

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration

  Hemoglobin concentration was measured as part of the hematology panel or measured separately when the hematology panel was not scheduled. Samples were measured by a central laboratory. Baseline is the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1/Day 1. A positive change from baseline indicates that hemoglobin concentration increased.

  Baseline, Week 53 or end of study

Secondary Outcomes (6)

• Change From Baseline to 12 Months (Week 53) in Platelet Count

  Baseline, Week 53

• Percent Change From Baseline to 12 Months (Week 51) in Normalized Liver Volume Measured Using Magnetic Resonance Imaging (MRI)

  Baseline, Week 51 or end of study

• Percent Change From Baseline to 12 Months (Week 51) in Normalized Spleen Volume Measured Using Magnetic Resonance Imaging (MRI)

  Baseline, Week 51

• Number of Participants With Abnormal Neurological Status During The Study

  Baseline, Weeks 13, 25, 37, and 53 or end of study

• Number of Participants Who Experienced a Treatment-Emergent Adverse Event

  57 weeks

Study Arms (1)

velaglucerase alfa

EXPERIMENTAL

IV infusion, 60 U/kg, every other week for 1 year

Biological: velaglucerase alfa

Interventions

velaglucerase alfa BIOLOGICAL

lyophilized powder, intravenous infusion, units, Every other week (EOW)

Also known as: VPRIV, Gene activated glucocerebrosidase, GA-GCB, Enzyme replacement therapy

velaglucerase alfa

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• The patient has a confirmed diagnosis of type 3 Gaucher disease.
• The patient is ≥ 2 and \< 18 years of age at the time of enrollment.
• The patient is either näive to treatment or has not received treatment (investigational or approved) for Gaucher disease within 12 months prior to study entry.
• The patient has Gaucher disease-related anemia, defined as hemoglobin concentration below the lower limit of normal for age and sex.
• AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA
• The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation.
• The patient has Gaucher disease-related thrombocytopenia, defined as platelet count \< 120 x 10,000 platelets/cubic mm.
• The patient has a Gaucher disease-related readily palpable enlarged liver.
• Patients who have undergone splenectomy may still be eligible to participate in the study.
• Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy testing will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the Investigator.
• The patient's parent(s) or the patient's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

You may not qualify if:

• The patient is suspected of having type 2 or type 1 Gaucher disease.
• The patient is \< 2 years of age.
• The patient has experienced a severe (Grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any enzyme replacement therapy for Gaucher disease (approved or investigational).
• The patient has received any non-Gaucher disease-related treatment with an investigational drug within 30 days prior to study entry.
• The patient is a pregnant and/or lactating female.

Sponsors & Collaborators

• Shire: lead

Study Sites (5)

Alexandria University Hospital

Alexandria, 21131, Egypt

Location

Abu El Rich Hospital, Cairo University Hospital

Cairo, Egypt

Location

Children's Hospital, Ain Shams University Hospital

Cairo, Egypt

Location

KEM Hospital Research Centre

Pune, Maharashtra, India

Location

Hospital La Rabta

Tunis, 1007, Tunisia

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

Glucosylceramidase; Enzyme Replacement Therapy

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Glucosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Enzyme Therapy; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2012
• First Posted: September 14, 2012
• Study Start: September 14, 2012
• Primary Completion: March 15, 2015
• Study Completion: March 15, 2015
• Last Updated: June 11, 2021
• Results First Posted: October 30, 2015

Record last verified: 2021-05

Locations

IN (1); TU (1); EG (3)