Gaucher DiseasePhase 1/2NCT00391625
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
A Phase 1/2 study testing GA-GCB in people with Gaucher Disease, run by Shire at 3 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- GA-GCB
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 13 Sep 2004to 31 Jan 2008
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
About this study
Official title: An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy
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.
Read the detailed description
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the central nervous system (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 (the long term safety of enzyme replacement therapy with DRX008A (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 evaluate the long term safety of GA-GCB (velaglucerase alfa) in patients with Type 1 Gaucher disease
- Conditions
- Gaucher Disease
- Being tested
- GA-GCB
- Keywords
- Gaucher disease, Enzyme Replacement Therapy
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GA-GCBDrug
Also called VPRIV®, velaglucerase alfa, gene-activated glucocerebrosidase, DRX008
15-60 U/kg every other week via intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| GA-GCB | Experimental | 15-60 U/kg every other week via intravenous infusion Receives: Drug: GA-GCB |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of Long Term Safety
Overall Summary of Treatment-emergent Adverse Events-Safety Population
Measured: Up to 84 months
Secondary outcomes (4)
Percent Change From Baseline in Hemoglobin Concentration
Measured: Baseline, then every 12 months
Percent Change From Baseline in Platelet Counts
Measured: Baseline, then every 12 months
Percent Change From Baseline in Liver Volume
Measured: Baseline, Month 24, then every 9 or 12 months
Percent Change From Baseline in Spleen Size
Measured: Baseline, Month 24, then every 9 or 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2006 (estimated)
- Study start13 Sep 2004
- Primary completion31 Jan 2008
- Study completion31 Jan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00391625
- Sponsor's protocol ID
- TKT025EXT
- Status last verified
- May 2021
- First submitted
- 20 Oct 2006
- Last updated
- 22 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 20299511
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT00391625
- Acronym / sponsor ID
- TKT025EXT
- Conditions
- Gaucher Disease
- Keywords
- Gaucher disease, Enzyme Replacement Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 13 Sep 2004 (actual)
- Primary completion
- 31 Jan 2008 (actual)
- Study completion
- 31 Jan 2008 (actual)
- First posted
- 24 Oct 2006 (estimated)
- Results
- Posted 23 Jun 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the central nervous system (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 (the long term safety of enzyme replacement therapy with DRX008A (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 evaluate the long term safety of GA-GCB (velaglucerase alfa) in patients with Type 1 Gaucher disease
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GA-GCB | Experimental | Drug: GA-GCB15-60 U/kg every other week via intravenous infusion |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GA-GCBAlso: VPRIV®, velaglucerase alfa, gene-activated glucocerebrosidase, DRX00815-60 U/kg every other week via intravenous infusion | Drug | GA-GCB |
Primary outcome
Evaluation of Long Term Safety
Time frame: Up to 84 months
Overall Summary of Treatment-emergent Adverse Events-Safety Population
4 secondary outcomes
Percent Change From Baseline in Hemoglobin Concentration
Time frame: Baseline, then every 12 months
Percent Change From Baseline in Platelet Counts
Time frame: Baseline, then every 12 months
Percent Change From Baseline in Liver Volume
Time frame: Baseline, Month 24, then every 9 or 12 months
Percent Change From Baseline in Spleen Size
Time frame: Baseline, Month 24, then every 9 or 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20299511Zimran 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.
Related trials for Gaucher Disease
Enrolling by invitationObservational study
Genetic Studies of Lysosomal Storage Disorders
Lysosomal Storage Disorders, Gaucher Disease
Active, not recruitingPhase 1/2
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Gaucher Disease, Type 2
RecruitingObservational study
Parkinson's Disease in Type 1 Gaucher Disease
Parkinson Disease (PD), Type 1 Gaucher Disease
Not yet recruitingPhase 2/3
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
Niemann-Pick Disease Type C, GM2 Gangliosidosis
RecruitingObservational study
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Neurological Regression, Myoclonus
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GA-GCB in people with Gaucher Disease, run by Shire at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Israel, Romania, Serbia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Jun 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of Long Term Safety (time frame: Up to 84 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00391625, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2021.