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Lumizyme Medical Research Studies

Up-to-date List of Lumizyme Medical Research Studies

What Research is Being Done?

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies. Following list includes both interventional and observational studies.

Latest Lumizyme Medical Research Studies

Rank Status Study
1 Recruiting A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated
Conditions: Pompe Disease (Infantile-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenosis 2;   Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa
Outcome Measures: Change from baseline in cardiac function as measured by the left ventricular mass Z-score(LVM-Z);   Estimated probability of survival;   Probability of invasive ventilator-free survival;   Change in motor development status as assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores;   Number of Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs)
2 Recruiting Immune Tolerance Induction Study
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: Myozyme (alglucosidase alfa)
Outcome Measures: Evaluate the efficacy of ITI regimens as assessed by anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and antibodies that inhibit the enzymatic activity and/or uptake of Myozyme;   Evaluate Pompe disease activity in patients receiving the 2 ITI regimens as measured by overall survival.;   Evaluate Pompe disease activity in patients receiving the 2 ITI regimens as measured by respiratory function.;   Evaluate Pompe disease activity in patients receiving the 2 ITI regimens as measured by Left ventricular mass index (LVMI).;   Evaluate Pompe disease activity in patients receiving the 2 ITI regimens as measured by motor function.;   Evaluate Pompe disease activity in patients receiving the 2 ITI regimens as measured by disability index.;   Evaluate the safety of the 2 ITI regimens as assessed by the incidence of adverse events (AEs), serious adverse events (SAEs), and clinical laboratory abnormalities.
3 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention: Biological: alglucosidase alfa
Outcome Measures: alglucosidase alfa accumulation in immature (1-3 months post partum) or mature (4-6 months post partum) breast milk sample from mothers with Pompe disease who are being treated with alglucosidase alfa.;   breast milk production and composition in women with Pompe disease who receive alglucosidase alfa
4 Recruiting Growth and Development Study of Myozyme (Alglucosidase Alfa).
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease
Intervention: Biological: alglucosidase alfa
Outcome Measures: Long-term growth and development as measured by recumbent length/height, weight and head circumference;   Change from baseline in motor development and function, as measured by changes in the motor subscale of the Bayley Scales of Infant and Toddler Development (Bayley-III) (up to 42 months of age) at 10 years;   Change from baseline in motor development and function, as measured by changes in the total score of the Gross Motor Function Measure (GMFM-88) at 10 years;   Change from baseline in the raw scores, normative standard scores and scaled scores for the Functional Skills Mobility and Self-Care domains of the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at 10 years;   Change from baseline in Cognitive Development, as measured by changes in the raw scores, scaled scores and composite scores for the cognitive and language subscales of the Bayley Scales of Infant and Toddler Development (Bayley-III) at 10 years;   Change from baseline in Cognitive Development, as measured by changes in the raw and scaled scores of the modified Leiter International Performance Scale - Revised (Leiter-R) scores (starting at 42 months of age);   Summary of Adverse Events
5 Recruiting A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Condition: Pompe Disease
Intervention: Drug: Alglucosidase alfa
Outcome Measures: Percentage of participants who are clinically stable or improved at Week 52;   Percent survival at Week 52;   Percentage of participants who are invasive ventilator-free at Week 52;   Change from baseline on left ventricular mass Z-score (LVM-Z);   Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score;   Change from baseline in forced vital capacity (FVC) % predicted
6 Recruiting Alglucosidase Alfa Pompe Safety Sub-Registry
Condition: Pompe Disease
Intervention: Biological: Alglucosidase alfa
Outcome Measure: number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
7 Recruiting Pompe Pregnancy Sub-Registry
Conditions: Glycogen Storage Disease Type II (GSD-II);   Pompe Disease (Late-onset);   Glycogenesis 2 Acid Maltase Deficiency
Interventions: Biological: alglucosidase alpha;   Other: No Treatment
Outcome Measures: Pregnancy outcomes, including complications, in women with Pompe disease who receive Myozyme during pregnancy and in women with Pompe disease that do not receive Myozyme;   Follow-up of infants born to women with Pompe disease for 3 years post-partum
8 Recruiting Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenesis 2;   Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa
Outcome Measures: Maximum observed concentration (Cmax);   Actual sampling time to reach maximum observed concentration (Tmax);   Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last);   Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf);   Terminal elimination half-life (T1/2);   Total systemic clearance (CL);   Volume of distribution (Vd);   Impact of anti-rhGAA IgG antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance;   Impact of inhibitory/neutralizing antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance
9 Recruiting Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Condition: Pompe Disease
Intervention: Other: Observational
Outcome Measures: Clinical response to enzyme replacement therapy (ERT) using alglucosidase alfa (Myozyme);   Response to Immune Tolerance Induction (ITI)
10 Recruiting Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD II);   Acid Maltase Deficiency
Intervention: Drug: GZ402666
Outcome Measures: Adverse events;   Laboratory assessments including hematology, biochemistry and urinalysis;   Vital signs;   Electrocardiogram;   Immunogenicity assessments;   Cmax;   AUC;   t1/2;   Skeletal muscle glycogen content;   Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments.;   Urinary Hex4;   Functional assessments including 6 Minute Walk Test (6MWT);   Quality of life assessments
11 Recruiting BMN701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease
Condition: Late-onset Pompe Disease
Intervention: Drug: BMN 701
Outcome Measures: percent predicted MIP (Maximum Inspiratory Pressure) measured at the mouth by the Mueller maneuver;   percent predicted MEP (Maximum Expiratory Pressure);   6 Minute Walk Test
12 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
Outcome Measure: Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease
13 Recruiting NeoGAA Extension Study
Condition: Glycogen Storage Disease Type II Pompe Disease
Intervention: Drug: GZ402666
Outcome Measures: Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs);   Laboratory assessments including hematology, biochemistry and urinalysis;   Vital signs;   Electrocardiogram;   Immunogenicity assessments (anti-neoGAA immunoglobulin G (IgG) antibodies, neutralizing antibody formation in IgG seropositive patients, and anti-alglucosidase alfa IgG antibodies);   Cmax;   AUC;   t1/2;   Skeletal muscle glycogen content;   Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments;   Urinary Hex4;   plasma analyses of circulating mRNA and micro RNA;   serum analyses of skeletal muscle RNA expression
14 Recruiting Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Condition: Pompe Disease
Interventions: Drug: Study Agent Administration;   Other: Safety Labs;   Other: Pulmonary Function Testing;   Other: RMST
Outcome Measures: Assessment of the safety of intramuscular administration of a recombinant adeno-associated virus, rAAV1-CMV-GAA, in children with ventilator dependent Pompe disease.;   Determination of the dose of rAAV1-CMV-GAA vector required to achieve diaphragm transduction and restoration of GAA activity in the diaphragm.;   Evaluation of Ventilatory performance benefit of rAAV1-CMV-GAA gene transfer and RMST compared to RMST alone.

These studies may lead to new treatments and are adding insight into Lumizyme etiology and treatment.

A major focus of Lumizyme research is the development of new drugs and other treatment options. Studies seek to identify new drugs to treat various related disorders and to find safer, more effective doses for medications already being used. Other research is aimed at identifying receptors or drug targets.


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