Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

NCT00391625 | Completed Phase 1 Results

• 1 other identifier: TKT025EXT
• Study Type: interventional
• Target: 10
• Locations: 3 countries
• Sites: 3

Brief 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 study is to evaluate the long term safety of enzyme replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.

Conditions

Gaucher Disease

Keywords

Gaucher disease, Enzyme Replacement Therapy

Outcome Measures

Primary Outcomes (1)

• Evaluation of Long Term Safety

  Overall Summary of Treatment-emergent Adverse Events-Safety Population

  Up to 84 months

Secondary Outcomes (4)

• Percent Change From Baseline in Hemoglobin Concentration

  Baseline, then every 12 months

• Percent Change From Baseline in Platelet Counts

  Baseline, then every 12 months

• Percent Change From Baseline in Liver Volume

  Baseline, Month 24, then every 9 or 12 months

• Percent Change From Baseline in Spleen Size

  Baseline, Month 24, then every 9 or 12 months

Study Arms (1)

GA-GCB

EXPERIMENTAL

15-60 U/kg every other week via intravenous infusion

Drug: GA-GCB

Interventions

GA-GCB DRUG

15-60 U/kg every other week via intravenous infusion

Also known as: VPRIV®, velaglucerase alfa, gene-activated glucocerebrosidase, DRX008

GA-GCB

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients who have completed through Week 41 visit in the TKT025 study.
• Patients must have voluntarily signed an IRB/EC approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
• Patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
• Female and male patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Female patients must have a negative serum pregnancy test on enrollment.

You may not qualify if:

• Patient has received treatment with non-Gaucher disease related investigational drug or device within the past 30 days prior to study entry; such use during the study is not permitted.
• Patient has a clinically relevant medical condition (e.g., HIV, hepatitis B or C) that would make implementation of the protocol difficult and/or confound an assessment of the effects of the experimental therapy and its adverse events.
• Patient, patient's parent(s), or patient's legal guardian is unable to understand the nature, scope and possible consequences of the study.
• Patient is unable to comply with the protocol, e.g. uncooperative attitude, medical condition, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator.

Sponsors & Collaborators

• Shire: lead

Study Sites (3)

Shaare Zedek Medical Center

Jerusalem, Israel

Location

Maria Sklodowska Curie Children's Hospital

Bucharest, 75544, Romania

Location

Mother and Child Health Care Institute of Serbia

Belgrade, Serbia

Location

Related Publications (1)

• Zimran A, Altarescu G, Philips M, Attias D, Jmoudiak M, Deeb M, Wang N, Bhirangi K, Cohn GM, Elstein D. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Blood. 2010 Jun 10;115(23):4651-6. doi: 10.1182/blood-2010-02-268649. Epub 2010 Mar 18.

  PMID: 20299511 DERIVED

MeSH Terms

Conditions

Gaucher Disease

Interventions

Glucosylceramidase

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

Limitations and Caveats

Imputation was applied to missing data.

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: October 20, 2006
• First Posted: October 24, 2006
• Study Start: September 13, 2004
• Primary Completion: January 31, 2008
• Study Completion: January 31, 2008
• Last Updated: June 22, 2021
• Results First Posted: June 23, 2014

Record last verified: 2021-05

Locations

RO (1); IL (1); SE (1)