Clinical trial NCT01526785: A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Official title: A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Pompe Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2012-02-06
Enrollment: 113
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.


Eligibility criteria: Inclusion Criteria:

A participant might meet all of the following criteria to be eligible for this study.

1. The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
2. The participant might be at least 1 year of age at the time of informed consent.
3. The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
4. The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

A participant who met any of the following criteria were to be excluded from this study.

1. The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study.
2. The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
3. The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.

Primary outcomes:
measure: Percentage of Participants Who Were Clinically Stable or Improved at Week 52
timeFrame: Week 52
Secondary outcomes:
measure: Survival Rate at Week 52
timeFrame: Week 52
measure: Invasive Ventilator-Free Survival Rate at Week 52
timeFrame: Week 52
measure: Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52
timeFrame: Baseline, Week 52
measure: Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52
timeFrame: Baseline, Week 52
measure: Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position
timeFrame: Baseline, Week 52
measure: Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position
timeFrame: Baseline, Week 52
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Phoenix
country: United States
state: Arizona
city: Little Rock
country: United States
state: Arkansas
city: Oakland
country: United States
state: California
city: Orange
country: United States
state: California
city: San Diego
country: United States
state: California
city: San Jose
country: United States
state: California
city: Stanford
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Washington, DC
country: United States
state: District of Columbia
city: Gainesville
country: United States
state: Florida
city: Miami
country: United States
state: Florida
city: Decatur
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Indianapolis
country: United States
state: Indiana
city: Kansas City
country: United States
state: Kansas
city: Louisville
country: United States
state: Kentucky
city: Baton Rouge
country: United States
state: Louisiana
city: Shreveport
country: United States
state: Louisiana
city: Boston
country: United States
state: Massachusetts
city: Detroit
country: United States
state: Michigan
city: Grand Rapids
country: United States
state: Michigan
city: Minneapolis
country: United States
state: Minnesota
city: Jackson
country: United States
state: Mississippi
city: Columbia
country: United States
state: Missouri
city: Kansas City
country: United States
state: Missouri
city: St. Louis
country: United States
state: Missouri
city: Omaha
country: United States
state: Nebraska
city: Las Vegas
country: United States
state: Nevada
city: New Brunswick
country: United States
state: New Jersey
city: Glens Falls
country: United States
state: New York
city: New York
country: United States
state: New York
city: Rochester
country: United States
state: New York
city: Syracuse
country: United States
state: New York
city: Valhalla
country: United States
state: New York
city: Asheville
country: United States
state: North Carolina
city: Durham
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Cleveland
country: United States
state: Ohio
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Providence
country: United States
state: Rhode Island
city: Memphis
country: United States
state: Tennessee
city: Nashville
country: United States
state: Tennessee
city: Austin
country: United States
state: Texas
city: Fort Worth
country: United States
state: Texas
city: Salt Lake City
country: United States
state: Utah
city: Norfolk
country: United States
state: Virginia
city: Seattle
country: United States
state: Washington
city: Morgantown
country: United States
state: West Virginia
city: Miliwaukee
country: United States
state: Wisconsin
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Duong T, Kishnani PS, An Haack K, Foster MC, Gibson JB, Wilson C, Hahn SH, Hillman R, Kronn D, Leslie ND, Pena LDM, Sparks SE, Stockton DW, Tanpaiboon P, Day JW; Pompe ADVANCE Study Consortium. Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort. J Neuromuscul Dis. 2022;9(6):713-730. doi: 10.3233/JND-210784.
citation: Byrne BJ, Colan SD, Kishnani PS, Foster MC, Sparks SE, Gibson JB, An Haack K, Stockton DW, Pena LDM, Hahn SH, Johnson J, Tanpaiboon PX, Leslie ND, Kronn D, Hillman RE, Wang RY; Pompe ADVANCE Study Consortium. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort. Cardiol Young. 2022 Mar;32(3):364-373. doi: 10.1017/S1047951121002079. Epub 2021 Aug 23.
citation: Kishnani PS, Gibson JB, Gambello MJ, Hillman R, Stockton DW, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Day JW, Wang RY, Goldstein JL, An Haack K, Sparks SE, Zhao Y, Hahn SH; Pompe ADVANCE Study Consortium. Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease. Genet Med. 2019 Nov;21(11):2543-2551. doi: 10.1038/s41436-019-0527-9. Epub 2019 May 14.
citation: Hahn SH, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Gambello MJ, Gibson JB, Hillman R, Stockton DW, Day JW, Wang RY, An Haack K, Shafi R, Sparks S, Zhao Y, Wilson C, Kishnani PS; Pompe ADVANCE Study Consortium. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study. Genet Med. 2018 Oct;20(10):1284-1294. doi: 10.1038/gim.2018.2. Epub 2018 Mar 22.