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Myozyme

Phase 3

Pompe Disease (Late-onset) | Monoclonal antibody | Rare Disease |Sanofi|Last Updated: Feb 6, 2014

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials2
Total Enrollment10

FDA Designations

No designations recorded

Clinical trial landscape

Myozyme · 7 trials · 9 indications

Phase 3 1Phase 2 5Phase 1 1
NCT00268944Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportPompe Disease (Late-onset)
COMPLETED5 Analytics
PHASE3COMPLETED
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
Pompe Disease (Late-onset)
NCT002689445
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Study Endpoints

Primary Endpoints

Treatment effect on muscle strength and functional status.
six months and one year
Treatment effect on pulmonary function and/or ventilation conditions.
six months and one year
Treatment effect on cardiomyopathy noted at inclusion
six months and one year
Treatment effect on fatigue.
six months and one year
Treatment effect on quality of life.
six months and one year
Treatment effect on muscular atrophy.
six months and one year
Overall patient satisfaction with treatment (visual analog scale).
six months and one year
Pharmacodynamics assessment.
six months and one year
Long-term Safety and Efficacy
52 weeks
safety and PK profile rhGAA
74 weeks
FVC
74 weeks
MMT
74 weeks
Effect of treatment on muscle function
74 weeks
Evaluate the safety profile of MZ
52 weeks
To estimate the proportion of patients treated w/ MZ who were alive and free of ventilator support at 12 months of age; compared to historical cohort
52 weeks
Determine PK/PD profile of MZ
52 weeks
Determine effect of different doses of MZ on safety and efficacy
52 weeks
The objective of this extension study was to monitor the long-term safety and efficacy
3 years
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
3 years
Evaluate the safety of Myozyme
52 weeks
Determine proportion of patients alive over the course of treatment
52 weeks
PK profile of MZ
52 weeks
PD profile of MZ
52 weeks
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
1EXPERIMENTAL -

Interventions

NameTypeDescription
MyozymeBIOLOGICAL20 mg/kg qow
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * male or female aged greater than or equal to 18 years * patient's legally authorized guardian(s) must provide signed, informed consent prior to initiation of study; patient's signature required if patient understands informed consent * patient must have a documented deficit in...

Countries:FranceUnited StatesGermanyIsraelItalyNetherlandsTaiwanUnited KingdomSouth Africa
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Frequently asked questions about Myozyme

What is Myozyme used for?

Myozyme (alglucosidase alfa) is an investigational enzyme replacement therapy studied for Glycogen Storage Disease Type II, also known as Pompe disease, including infantile-onset and late-onset forms. It is developed by Sanofi (SNY) and is currently in Phase 2 clinical development.

What does Myozyme target?

Myozyme is a monoclonal antibody designed to replace the deficient acid alpha-glucosidase enzyme in patients with Pompe disease. It targets the underlying enzyme deficiency that causes glycogen accumulation in tissues, addressing the root cause of the disease.

Who makes Myozyme?

Myozyme is developed by Sanofi, a multinational pharmaceutical company listed on the stock exchange under the ticker SNY. Sanofi is conducting clinical trials to evaluate the safety and efficacy of Myozyme in patients with Pompe disease.

What phase is Myozyme in?

Myozyme is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials are ongoing to assess its safety and efficacy in treating Pompe disease.

What clinical trials is Myozyme in?

Myozyme has been studied in several clinical trials, including NCT00053573, NCT00059280, NCT00125879, and NCT00250939. These trials evaluated the drug in patients with infantile-onset and late-onset Pompe disease across multiple countries, including the United States, France, and the Netherlands.

Is Myozyme the same as alglucosidase alfa?

Myozyme is the brand name for alglucosidase alfa, a recombinant human acid alpha-glucosidase enzyme. It is being developed by Sanofi for the treatment of Pompe disease, a rare genetic disorder.