Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were follo...
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| Published in | Molecular genetics and metabolism Vol. 94; no. 4; pp. 469 - 475 |
|---|---|
| Main Authors | , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Inc
01.08.2008
|
| Subjects | |
| Online Access | Get full text |
| ISSN | 1096-7192 1096-7206 1096-7206 |
| DOI | 10.1016/j.ymgme.2008.04.001 |
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| Abstract | The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97–260 Weeks. All patients received weekly infusions of rhASB at 1
mg/kg. Efficacy was evaluated by (1) distance walked in a 12-minute walk test (12MWT) or 6-minute walk test (6MWT), (2) stairs climbed in the 3-minute stair climb (3MSC), and (3) reduction in urinary glycosaminoglycans (GAG). Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment.
Results: A significant reduction in urinary GAG (71–79%) was sustained. For the 12MWT, subjects in Phase 2 showed improvement of 255
±
191
m (mean
±
SD) at Week 144; those in Phase 3 Extension demonstrated improvement from study baseline of 183
±
26
m (mean
±
SE) in the rhASB/rhASB group at Week 96 and from treatment baseline (Week 24) of 117
±
25
m in the placebo/rhASB group. The Phase 1/2 6MWT and the 3MSC from Phase 2 and 3 also showed sustained improvements through the final study measurements. Compliance was 98% overall. Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe.
Conclusion: rhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile. |
|---|---|
| AbstractList | The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97-260 Weeks. All patients received weekly infusions of rhASB at 1 mg/kg. Efficacy was evaluated by (1) distance walked in a 12-minute walk test (12MWT) or 6-minute walk test (6MWT), (2) stairs climbed in the 3-minute stair climb (3MSC), and (3) reduction in urinary glycosaminoglycans (GAG). Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment.UNLABELLEDThe objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97-260 Weeks. All patients received weekly infusions of rhASB at 1 mg/kg. Efficacy was evaluated by (1) distance walked in a 12-minute walk test (12MWT) or 6-minute walk test (6MWT), (2) stairs climbed in the 3-minute stair climb (3MSC), and (3) reduction in urinary glycosaminoglycans (GAG). Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment.A significant reduction in urinary GAG (71-79%) was sustained. For the 12MWT, subjects in Phase 2 showed improvement of 255+/-191 m (mean+/-SD) at Week 144; those in Phase 3 Extension demonstrated improvement from study baseline of 183+/-26 m (mean+/- SE) in the rhASB/rhASB group at Week 96 and from treatment baseline (Week 24) of 117+/-25 m in the placebo/rhASB group. The Phase 1/2 6MWT and the 3MSC from Phase 2 and 3 also showed sustained improvements through the final study measurements. Compliance was 98% overall. Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe.RESULTSA significant reduction in urinary GAG (71-79%) was sustained. For the 12MWT, subjects in Phase 2 showed improvement of 255+/-191 m (mean+/-SD) at Week 144; those in Phase 3 Extension demonstrated improvement from study baseline of 183+/-26 m (mean+/- SE) in the rhASB/rhASB group at Week 96 and from treatment baseline (Week 24) of 117+/-25 m in the placebo/rhASB group. The Phase 1/2 6MWT and the 3MSC from Phase 2 and 3 also showed sustained improvements through the final study measurements. Compliance was 98% overall. Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe.rhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile.CONCLUSIONrhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile. The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97–260 Weeks. All patients received weekly infusions of rhASB at 1 mg/kg. Efficacy was evaluated by (1) distance walked in a 12-minute walk test (12MWT) or 6-minute walk test (6MWT), (2) stairs climbed in the 3-minute stair climb (3MSC), and (3) reduction in urinary glycosaminoglycans (GAG). Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment. Results: A significant reduction in urinary GAG (71–79%) was sustained. For the 12MWT, subjects in Phase 2 showed improvement of 255 ± 191 m (mean ± SD) at Week 144; those in Phase 3 Extension demonstrated improvement from study baseline of 183 ± 26 m (mean ± SE) in the rhASB/rhASB group at Week 96 and from treatment baseline (Week 24) of 117 ± 25 m in the placebo/rhASB group. The Phase 1/2 6MWT and the 3MSC from Phase 2 and 3 also showed sustained improvements through the final study measurements. Compliance was 98% overall. Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe. Conclusion: rhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile. The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97-260 Weeks. All patients received weekly infusions of rhASB at 1 mg/kg. Efficacy was evaluated by (1) distance walked in a 12-minute walk test (12MWT) or 6-minute walk test (6MWT), (2) stairs climbed in the 3-minute stair climb (3MSC), and (3) reduction in urinary glycosaminoglycans (GAG). Safety was evaluated by compliance, adverse event (AE) reporting and adherence to treatment. A significant reduction in urinary GAG (71-79%) was sustained. For the 12MWT, subjects in Phase 2 showed improvement of 255+/-191 m (mean+/-SD) at Week 144; those in Phase 3 Extension demonstrated improvement from study baseline of 183+/-26 m (mean+/- SE) in the rhASB/rhASB group at Week 96 and from treatment baseline (Week 24) of 117+/-25 m in the placebo/rhASB group. The Phase 1/2 6MWT and the 3MSC from Phase 2 and 3 also showed sustained improvements through the final study measurements. Compliance was 98% overall. Only 560 of 4121 reported AEs (14%) were related to treatment with only 10 of 560 (2%) described as severe. rhASB treatment up to 5 years results in sustained improvements in endurance and has an acceptable safety profile. |
| Author | Wraith, J. Edmond Scarpa, Maurizio D. Schwartz, Ida Vanessa Teles, Elisa Leão Swiedler, Stuart J. Hopwood, John J. Kaplan, Paige Miranda, M. Clara Sá Giugliani, Roberto Beck, Michael Guffon, Nathalie Steiner, Robert Decker, Celeste Arash, Laila Whitley, Chester B. Plecko, Barbara Harmatz, Paul Yu, Zi-Fan Ketteridge, David |
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| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/18502162$$D View this record in MEDLINE/PubMed |
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| ContentType | Journal Article |
| Contributor | Bernard, Philippe Pastores, Gregory Lee, Claudia Bennett-Jones, D N Keppen, Laura Valadares, Eugênia Ribeiro Coopman, Eduardo Oates, Stephanie Sorge, Giovanni Waber, Lewis Lin, Shuan-Pei Pozzi, Silvio Pavone, Lorenzo Lubitz, Lionel Boy, Raquel Barone, Rita Preiss, Uwe de Villemeur, Billette Waterson, John de Souza, Isabel Cristina Neves Santos, Emerson Santana Pais, Ray Gizzi, Elio da Silva, Luiz Carlos Santana Azevedo, Ana Cecília Arroyo, Javier Martins, Ana Maria Frischman, William Victor, Bonito O'Meara, Anne Kretz, Michel Amraoui, Yasmina Fiumara, Agata Korinthenberg, Rudolf Sillence, David Simon, Julie |
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| Copyright | 2008 Elsevier Inc. |
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| Keywords | Mucopolysaccharidosis VI Clinical trial N-Acetylgalactosamine-4-sulfatase Glycosaminoglycans Enzyme replacement therapy |
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| References | Harmatz, Giugliani, Schwartz, Guffon, Teles, Miranda, Wraith, Beck, Arash, Scarpa, Yu, Wittes, Berger, Newman, Lowe, Kakkis, Swiedler (bib6) 2006; 148 Solway, Brooks, Lacasse, Thomas (bib7) 2001; 119 Geiger, Strasak, Treml, Gasser, Kleinsasser, Fischer, Geiger, Loeckinger, Stein (bib11) 2007; 150 Neufeld, Muenzer (bib1) 2001 Swiedler, Beck, Bajbouj, Giugliani, Schwartz, Harmatz, Wraith, Roberts, Ketteridge, Hopwood, Guffon, Sa Miranda, Teles, Berger, Piscia-Nichols (bib3) 2005; 134 Giugliani, Harmatz, Wraith (bib2) 2007; 120 Harmatz, Ketteridge, Giugliani, Guffon, Teles, Miranda, Yu, Swiedler, Hopwood (bib4) 2005; 115 Paap, van der Net, Helders, Takken (bib9) 2005; 53 Crawley, Brooks, Muller, Petersen, Isaac, Bielicki, King, Boulter, Moore, Fazzalari, Anson, Byers, Hopwood (bib13) 1996; 97 Gulmans, van Veldhoven, de Meer, Helders (bib8) 1996; 22 Harmatz, Whitley, Waber, Pais, Steiner, Plecko, Kaplan, Simon, Butensky, Hopwood (bib5) 2004; 144 Auclair, Hopwood, Brooks, Lemontt, Crawley (bib12) 2003; 78 Moalla, Gauthier, Maingourd, Ahmaidi (bib10) 2005; 26 Harmatz (10.1016/j.ymgme.2008.04.001_bib6) 2006; 148 Solway (10.1016/j.ymgme.2008.04.001_bib7) 2001; 119 Geiger (10.1016/j.ymgme.2008.04.001_bib11) 2007; 150 Paap (10.1016/j.ymgme.2008.04.001_bib9) 2005; 53 Giugliani (10.1016/j.ymgme.2008.04.001_bib2) 2007; 120 Neufeld (10.1016/j.ymgme.2008.04.001_bib1) 2001 Swiedler (10.1016/j.ymgme.2008.04.001_bib3) 2005; 134 Gulmans (10.1016/j.ymgme.2008.04.001_bib8) 1996; 22 Auclair (10.1016/j.ymgme.2008.04.001_bib12) 2003; 78 Moalla (10.1016/j.ymgme.2008.04.001_bib10) 2005; 26 Harmatz (10.1016/j.ymgme.2008.04.001_bib4) 2005; 115 Crawley (10.1016/j.ymgme.2008.04.001_bib13) 1996; 97 Harmatz (10.1016/j.ymgme.2008.04.001_bib5) 2004; 144 |
| References_xml | – volume: 134 start-page: 144 year: 2005 end-page: 150 ident: bib3 article-title: Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) publication-title: Am. J. Med. Genet. A – volume: 144 start-page: 574 year: 2004 end-page: 580 ident: bib5 article-title: Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) publication-title: J. 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Pediatr. doi: 10.1016/j.jpeds.2004.03.018 – volume: 53 start-page: 351 year: 2005 ident: 10.1016/j.ymgme.2008.04.001_bib9 article-title: Physiologic response of the six-minute walk test in children with juvenile idiopathic arthritis publication-title: Arthritis Rheum. doi: 10.1002/art.21175 – volume: 134 start-page: 144 year: 2005 ident: 10.1016/j.ymgme.2008.04.001_bib3 article-title: Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) publication-title: Am. J. Med. Genet. A doi: 10.1002/ajmg.a.30579 |
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| SubjectTerms | Adolescent Adult Child Child, Preschool Clinical trial Enzyme replacement therapy Female Follow-Up Studies Glycosaminoglycans Glycosaminoglycans - urine Humans Male Motor Activity Mucopolysaccharidosis VI Mucopolysaccharidosis VI - physiopathology Mucopolysaccharidosis VI - therapy N-Acetylgalactosamine-4-sulfatase N-Acetylgalactosamine-4-Sulfatase - administration & dosage N-Acetylgalactosamine-4-Sulfatase - adverse effects N-Acetylgalactosamine-4-Sulfatase - therapeutic use Recombinant Proteins - administration & dosage Recombinant Proteins - adverse effects Recombinant Proteins - therapeutic use Treatment Outcome Walking |
| Title | Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase |
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