Fabry DiseasePhase 4HEARTNCT00487630
Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
A Phase 4 study testing recombinant alpha-galactosidase A in people with Fabry Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Read more about this study
- Who is it for?
- Ages 15 years and older, women only
- Conditions the study is for
- Fabry Disease
- What is being tested?
- recombinant alpha-galactosidase A
- How many people
- 34 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2005to Jun 2009
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 15 years and older
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Female patients over 15 years with clinical and biological evidence of Fabry disease (GLA gene mutation detected)
Who cannot join
- Pregnancy
- Allergy to agalsidase beta
- Congestive heart failure
- Creatinaemia > 135 µmol/l
- Medical history of stroke during the last year
- Medical history of more than 2 transient ischemic attack
Show all 10Show fewer
- Blood pressure > 160/95
- Modification in medications treating for blood pressure during the last 3 months before enrollment
- Complete absence of clinical or biological symptoms
- Weight > 87 kg or < 35 kg
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Centre de reference de la maladie de Fabry et des maladies hereditaires du tissu conjonctif. Assistance Publique - Hôpitaux de Paris
Paris
Dominique P GERMAIN, MD, PhD
Karelle BENISTAN, MD
About this study
Official title: A Multicenter, Phase 4, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Recombinant Alpha-Galactosidase A (Agalsidase Beta, FABRAZYME) in Heterozygous Females for Fabry Disease
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Clearance of Gb3 and stabilization of renal function has been demonstrated in male patients treated with agalsidase beta (FABRAZYME). In contrast, no randomized, controlled study of the efficacy of recombinant alpha-galactosidase A has been reported in heterozygotes for Fabry disease.
Read the detailed description
The primary objective is to evaluate cardiac left ventricular mass (measured with echocardiography by unique investigator) in females over 15 years of age affected with Fabry disease receiving 70 mg of agalsidase beta every other week, as compared with an untreated controlled group matched for gender and age.
The secondary objectives include evaluation of :
- left ventricular posterior wall thickness (echocardiography)
- interventricular septum thickness (echocardiography)
- tissue doppler imaging (myocardial function)
- EKG
- creatinaemia
- serum cystatin C level
- urinary protein/creatinine ratio
- microalbuminuria
- Gb3 urinary levels
Evaluation of tolerance and safety with :
- Home therapy infusions follow up
- Vitals
- Physical examination
- Adverse events
- Antibodies levels
- Conditions
- Fabry Disease
- Being tested
- recombinant alpha-galactosidase A
- Keywords
- Heterozygous females, Cardiomyopathy
- Medical subject headings
- Fabry Disease, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant alpha-galactosidase ADrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Left ventricular mass
Measured: 2 years
Secondary outcomes (1)
Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2007 (estimated)
- Study startJun 2005
- Study completionJun 2009 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 34 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Investigators
- Dominique P GERMAIN, MD, PhD, Principal investigator, Centre de reference de la maladie de Fabry et des maladies hereditaires du tissu conjonctif. Assistance Publique Hopitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00487630
- Sponsor's protocol ID
- AOM-01-076
- Other IDs
- PHRC National 2001
- Acronym
- HEART
- Status last verified
- Jun 2007
- First submitted
- 15 Jun 2007
- Last updated
- 18 Jun 2007
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 4, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Recombinant Alpha-Galactosidase A (Agalsidase Beta, FABRAZYME) in Heterozygous Females for Fabry Disease
- ClinicalTrials.gov ID
- NCT00487630
- Acronym / sponsor ID
- HEART / AOM-01-076
- Conditions
- Fabry Disease
- Keywords
- Heterozygous females, Cardiomyopathy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 34 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Jun 2005
- Study completion
- Jun 2009 (estimated)
- First posted
- 18 Jun 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Clearance of Gb3 and stabilization of renal function has been demonstrated in male patients treated with agalsidase beta (FABRAZYME). In contrast, no randomized, controlled study of the efficacy of recombinant alpha-galactosidase A has been reported in heterozygotes for Fabry disease.
Detailed description
The primary objective is to evaluate cardiac left ventricular mass (measured with echocardiography by unique investigator) in females over 15 years of age affected with Fabry disease receiving 70 mg of agalsidase beta every other week, as compared with an untreated controlled group matched for gender and age.
The secondary objectives include evaluation of :
- left ventricular posterior wall thickness (echocardiography)
- interventricular septum thickness (echocardiography)
- tissue doppler imaging (myocardial function)
- EKG
- creatinaemia
- serum cystatin C level
- urinary protein/creatinine ratio
- microalbuminuria
- Gb3 urinary levels
Evaluation of tolerance and safety with :
- Home therapy infusions follow up
- Vitals
- Physical examination
- Adverse events
- Antibodies levels
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
Who may qualify
- Ages
- 15 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Female patients over 15 years with clinical and biological evidence of Fabry disease (GLA gene mutation detected)
Key exclusion criteria (10 listed)
- Pregnancy
- Allergy to agalsidase beta
- Congestive heart failure
- Creatinaemia > 135 µmol/l
- Medical history of stroke during the last year
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 |
|---|---|---|
| recombinant alpha-galactosidase A | Drug | – |
Primary outcome
Left ventricular mass
Time frame: 2 years
1 secondary outcome
Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2007.
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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.
Dominique P GERMAIN, MD, PhD principal investigator
Centre de reference de la maladie de Fabry et des maladies hereditaires du tissu conjonctif. Assistance Publique Hopitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing recombinant alpha-galactosidase A in people with Fabry Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 1 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 18 Jun 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Dominique P GERMAIN, MD, PhD (+33156092306, dominique.germain@egp.aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Left ventricular mass (time frame: 2 years). 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 18 Jun 2007.