Gaucher Disease, Type 3Phase 4NCT04002830
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
A Phase 4 study testing Elelyso in people with Gaucher Disease, Type 3, run by Ari Zimran at 3 sites in 3 countries.
Trial summary
This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Gaucher Disease, Type 3
- What is being tested?
- Elelyso
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 20 Nov 2020to 30 Jul 2023
- Phase
- Phase 4
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female of any age; however, if female:
- must be using contraception if of childbearing potential or must be surgically sterile
- must not be lactating
- Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor.
- Splenomegaly at least 5 x multiples of normal (MN).
- Treatment-naïve.
Who cannot join
- Eligible subjects may not have any of the following exclusion criteria:
- Type 2 GD.
- Presence of myoclonic seizures.
- At least one allele of:
- N370S (N409S in recent nomenclature)
- R496H (R535H in recent nomenclature)
Show all 16Show fewer
- Presence of calcification in heart valves or arteries in echocardiography.
- Presence of untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism. (Resolved anemia is not an exclusion criterion.)
- Presence of human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and/or hepatitis C infections.
- Splenectomy and bone marrow transplantation.
- Presence of any medical, emotional, behavioural, or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
- Any other disorder that may interfere with the results of the efficacy endpoints.
- Pregnancy or breastfeeding.
- Currently taking another investigational drug for any condition or any therapeutic drug for Gaucher disease.
- The subject and/or subject's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study.
- Medical history of any food/drugs allergy.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
About this study
This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.
Read the detailed description
Patients with Type 3 GD exhibit both visceral and neurologic manifestations. In addition to the progressive neurologic involvement, somatic disease manifestations, especially splenomegaly and resulting cytopenia, contribute to significant mortality and morbidity . The effects of enzyme replacement therapy (ERT) on patients with Type 1 GD have been clearly documented and have a beneficial effect on visceral and hematologic disease parameters . It is known that recombinant enzyme does not pass the blood-brain barrier and has no effect on neurologic involvement . Probably due to the rarity of Type 3 GD, information on the somatic effects of ERT is largely limited to case reports or single-center series. There are also few reviews of cohorts but the clinical subtype, age, genotype, ERT dosage, accompanying therapies, and treatment response vary widely among patients in these cohorts. This prospective study aims to objectively evaluate the hematologic and visceral effects of ERT with taliglucerase alfa on a rather clinically and genetically homogenous group of treatment-naïve patients with Type 3 GD . For the purposes of this study, subjects receiving no Gaucher-specific medications for at least 12 months will be considered "untreated". The results of this study are expected to provide a more objective view of the degree of response of this patient type, and potentially create new areas of research.
- Conditions
- Gaucher Disease, Type 3
- Being tested
- Elelyso
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElelysoDrug
Also called Taliglucerase Alfa
Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
| Study group | Role | What they receive |
|---|---|---|
| Taliglucerase Alpha | Experimental | Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Receives: Drug: Elelyso |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in Spleen Volume Measured by MRI
Percent change from baseline
Measured: from baseline to month 12
Secondary outcomes (4)
Percent Change From Baseline in Liver Volume Measured by MRI
Percent change from baseline
Measured: from baseline to month 12
Percent Change in Hemoglobin
Percent change from baseline
Measured: from baseline to Months 3, 6, 9, and 12
Percent Change in Platelet Count
Percent change from baseline
Measured: from baseline to Months 3, 6, 9, and 12
Percent Change in Lyso-GB1
Percent change from baseline
Measured: from baseline to Months 3, 6, 9, and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2019
- Study start20 Nov 2020
- Primary completion30 Jul 2023
- Study completion30 Jul 2023
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.
- Other trials for Gaucher Disease, Type 3Studies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Type 3 Gaucher disease patients
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ari Zimran
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor-investigator, Ari Zimran, Shaare Zedek Medical Center
- Investigators
- Ari Zimran, Prof., Principal investigator, Shaare Zedek Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04002830
- Sponsor's protocol ID
- WI224302
- Status last verified
- Nov 2024
- First submitted
- 27 Jun 2019
- Last updated
- 17 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
- ClinicalTrials.gov ID
- NCT04002830
- Acronym / sponsor ID
- WI224302
- Conditions
- Gaucher Disease, Type 3
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Ari ZimranUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 20 Nov 2020 (actual)
- Primary completion
- 30 Jul 2023 (actual)
- Study completion
- 30 Jul 2023 (actual)
- First posted
- 1 Jul 2019 (actual)
- Results
- Posted 17 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.
Detailed description
Patients with Type 3 GD exhibit both visceral and neurologic manifestations. In addition to the progressive neurologic involvement, somatic disease manifestations, especially splenomegaly and resulting cytopenia, contribute to significant mortality and morbidity . The effects of enzyme replacement therapy (ERT) on patients with Type 1 GD have been clearly documented and have a beneficial effect on visceral and hematologic disease parameters . It is known that recombinant enzyme does not pass the blood-brain barrier and has no effect on neurologic involvement . Probably due to the rarity of Type 3 GD, information on the somatic effects of ERT is largely limited to case reports or single-center series. There are also few reviews of cohorts but the clinical subtype, age, genotype, ERT dosage, accompanying therapies, and treatment response vary widely among patients in these cohorts. This prospective study aims to objectively evaluate the hematologic and visceral effects of ERT with taliglucerase alfa on a rather clinically and genetically homogenous group of treatment-naïve patients with Type 3 GD . For the purposes of this study, subjects receiving no Gaucher-specific medications for at least 12 months will be considered "untreated". The results of this study are expected to provide a more objective view of the degree of response of this patient type, and potentially create new areas of research.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Type 3 Gaucher disease patients
| Arm | Type | Receives |
|---|---|---|
| Taliglucerase Alpha | Experimental | Drug: ElelysoIntravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female of any age; however, if female:
- Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor.
- Splenomegaly at least 5 x multiples of normal (MN).
- Treatment-naïve.
Key exclusion criteria (16 listed)
- Type 2 GD.
- Presence of myoclonic seizures.
- At least one allele of:
- Presence of calcification in heart valves or arteries in echocardiography.
- Presence of untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism. (Resolved anemia is not an exclusion criterion.)
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 |
|---|---|---|
| ElelysoAlso: Taliglucerase AlfaTaliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries. | Drug | Taliglucerase Alpha |
Primary outcome
Percent Change From Baseline in Spleen Volume Measured by MRI
Time frame: from baseline to month 12
Percent change from baseline
4 secondary outcomes
Percent Change From Baseline in Liver Volume Measured by MRI
Time frame: from baseline to month 12
Percent Change in Hemoglobin
Time frame: from baseline to Months 3, 6, 9, and 12
Percent Change in Platelet Count
Time frame: from baseline to Months 3, 6, 9, and 12
Percent Change in Lyso-GB1
Time frame: from baseline to Months 3, 6, 9, and 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Ari Zimran, Prof. principal investigator
Shaare Zedek Medical Center
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Elelyso in people with Gaucher Disease, Type 3, run by Ari Zimran 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 6 inclusion and 16 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 17 Dec 2024. 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 India, Israel, Turkey (Türkiye). 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 17 Dec 2024). 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?
Percent Change From Baseline in Spleen Volume Measured by MRI (time frame: from baseline to month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Dec 2024.