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Gaucher DiseasePhase 3NCT01614574

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

A Phase 3 study testing velaglucerase alfa in people with Gaucher Disease, run by Shire at 3 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Gaucher Disease
What is being tested?
velaglucerase alfa
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
3 sitesJapan
Study dates
2 Mar 2012to 25 May 2013
Phase
Phase 3
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 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • The patient has a documented diagnosis of Gaucher disease
  • The patient is at least 2 years of age
  • Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study
  • 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
  • Patients who are switched from imiglucerase ERT must meet the following additional criteria:
Show all 11Show fewer
  • Received treatment with imiglucerase for a minimum of 12 consecutive months
  • Meet predefined limits for hemoglobin concentration and platelet counts
  • Patients naïve to treatment for Gaucher disease must meet the following additional criteria:
  • Not received treatment for Gaucher disease (investigational or approved products) within 12 months prior to study entry
  • Have Gaucher disease related anemia and at least one of the following: moderate splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related enlarged liver

Who cannot join

  • Treatment with any investigational drug or device within the 30 days prior to study entry (time of informed consent); such use during the study is not permitted
  • Positive for hepatitis B or hepatitis C.
  • Non-Gaucher disease related anemia
  • 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
  • Significant comorbidity, as determined by the Investigator that might affect study data or confound the study results
  • The patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator
Show all 11Show fewer
  • The patient has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)
  • Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic systemic corticosteroids in the last 6 months
  • Patient has had a splenectomy or the patient has an active, clinically significant spleen infarction within 12 months of screening
  • Patient has worsening bone necrosis within 12 months of screening
  • The patient is pregnant or lactating.

Trial locations and contacts

3 sites in 1 country.

Japan3 sites

  • Hamamatsu University School of Medicine

    Hamamatsu, Shizuoka, 431-3192

  • The Jikei University School of Medicine

    Minatoku, Toyko, 105-8461

  • Osaka City University Hospital

    Osaka, 545-0051

Other trials in Japan

About this study

Official title: A Multicenter, Open-Label Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and severity of neurological disease. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression.

The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients with Gaucher disease.

Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

Read the detailed description

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain.

Gaucher disease has been designated in the list of Specified Rare and Intractable Diseases by Specified Disease Treatment Research Program of Ministry of Health, Labor and Welfare (MHLW) as one of "lysosomal storage diseases" since 2001. Gaucher disease is also designated in the Medical Aid Program for Specified Categories of Chronic Pediatric Diseases.

The prevalence of mutations and the phenotype of patients with Gaucher disease in Japan differs from that in non-Japanese populations. Some patients with type 1 Gaucher disease in Japan have more severe and progressive disease compared to non-Japanese patients and the disease is characterized by an earlier onset of symptoms.

Velaglucerase alfa, a highly-purified form of the naturally occurring enzyme glucocerebrosidase, has been developed as an enzyme replacement therapy for Gaucher disease for the symptoms (anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone manifestation).

The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients (naive or previously treated with imiglucerase) 2 years of age and older with Gaucher disease.

Conditions
Gaucher Disease
Being tested
velaglucerase alfa
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • velaglucerase alfaBiological

    Also called VPRIV, Gene activated human glucocerebrosidase

    60 U/kg every other week intravenous infusion

Study groups in NCT01614574
Study groupRoleWhat they receive
InvestigationalExperimentalvelaglucerase alfa
Receives: Biological: velaglucerase alfa

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Severe Adverse Events (SAE)

    Measured: Baseline to week 51

  2. Number of Treatment Emergent Adverse Events (TEAE)

    Measured: Baseline to week 51

  3. Development of Anti-velaglucerase Alfa Antibody

    Measured: Baseline to week51

  4. Number of Infusion- Related Adverse Events

    Measured: Baseline to week 51

  5. Number of Patients With Concomitant Medication

    Measured: Baseline to week 51

Secondary outcomes (6)

  1. Change From Baseline in Hemoglobin Concentration

    Measured: Baseline to week 51

  2. Change From Baseline in Platelet Count

    Measured: Baseline to week 51

  3. Change From Baseline in Liver Volume, Normalized to Body Weight

    Measured: Baseline to week 51

  4. Change From Baseline in Spleen Volume, Normalized to Body Weight

    Measured: Baseline to week 51

  5. Change From Baseline in Plasma Chitotriosidase Levels

    Measured: Baseline to week 51

  6. Change From Baseline in CCL18 Levels

    Measured: Baseline to week 51

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Jun 2012 (estimated)
  2. Study start2 Mar 2012
  3. Primary completion25 May 2013
  4. Study completion25 May 2013

Resources

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01614574
Sponsor's protocol ID
HGT-GCB-087
Status last verified
Jun 2021
First submitted
6 Jun 2012
Last updated
28 Jun 2021
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing velaglucerase alfa in people with Gaucher Disease, run by Shire at 3 sites in Japan.

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 11 inclusion and 11 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 28 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 Japan. 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 phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 4 Aug 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?

Number of Severe Adverse Events (SAE) (time frame: Baseline to week 51). The study lists 5 primary outcomes in all. 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 NCT01614574.

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 28 Jun 2021.

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