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Pompe DiseasePhase 4NCT01526785

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

A Phase 4 study testing Alglucosidase alfa in people with Pompe Disease, run by Genzyme, a Sanofi Company at 51 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year and older
Conditions the study is for
Pompe Disease
What is being tested?
Alglucosidase alfa
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
113 participants
Where
51 sitesUnited States
Study dates
Mar 2012to Dec 2014
Phase
Phase 4
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 1 year and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • A participant might meet all of the following criteria to be eligible for this study.
  • The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
  • The participant might be at least 1 year of age at the time of informed consent.
  • The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
  • 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.

Who cannot join

  • A participant who met any of the following criteria were to be excluded from this study.
  • 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.
  • 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.
  • The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.

Trial locations and contacts

51 sites in 1 country.

51 sites in 1 country

United States51 sites

  • Study site

    Asheville, North Carolina

  • Study site

    Aurora, Colorado

  • Study site

    Austin, Texas

  • Study site

    Baton Rouge, Louisiana

  • Study site

    Birmingham, Alabama

  • Study site

    Boston, Massachusetts

  • Study site

    Chicago, Illinois

  • Study site

    Cincinnati, Ohio

  • Study site

    Cleveland, Ohio

  • Study site

    Columbia, Missouri

  • Study site

    Decatur, Georgia

  • Study site

    Detroit, Michigan

  • Study site

    Durham, North Carolina

  • Study site

    Fort Worth, Texas

  • Study site

    Gainesville, Florida

  • Study site

    Glens Falls, New York

  • Study site

    Grand Rapids, Michigan

  • Study site

    Indianapolis, Indiana

  • Study site

    Jackson, Mississippi

  • Study site

    Kansas City, Kansas

  • Study site

    Kansas City, Missouri

  • Study site

    Las Vegas, Nevada

  • Study site

    Little Rock, Arkansas

  • Study site

    Louisville, Kentucky

  • Study site

    Memphis, Tennessee

  • Study site

    Miami, Florida

  • Study site

    Miliwaukee, Wisconsin

  • Study site

    Minneapolis, Minnesota

  • Study site

    Morgantown, West Virginia

  • Study site

    Nashville, Tennessee

  • Study site

    New Brunswick, New Jersey

  • Study site

    New York, New York

  • Study site

    Norfolk, Virginia

  • Study site

    Oakland, California

  • Study site

    Omaha, Nebraska

  • Study site

    Orange, California

  • Study site

    Philadelphia, Pennsylvania

  • Study site

    Phoenix, Arizona

  • Study site

    Pittsburgh, Pennsylvania

  • Study site

    Providence, Rhode Island

  • Study site

    Rochester, New York

  • Study site

    Salt Lake City, Utah

  • Study site

    San Diego, California

  • Study site

    San Jose, California

  • Study site

    Seattle, Washington

  • Study site

    Shreveport, Louisiana

  • Study site

    St Louis, Missouri

  • Study site

    Stanford, California

  • Study site

    Syracuse, New York

  • Study site

    Valhalla, New York

  • Study site

    Washington D.C., District of Columbia

Other trials in United States

About this study

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

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

Conditions
Pompe Disease
Being tested
Alglucosidase alfa
Medical subject headings
Glycogen Storage Disease Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Alglucosidase alfaDrug

    Also called Lumizyme®

    4000 L alglucosidase alfa administered by IV infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.

Study groups in NCT01526785
Study groupRoleWhat they receive
Alglucosidase alfaExperimentalAlglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.
Receives: Drug: Alglucosidase alfa

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants Who Were Clinically Stable or Improved at Week 52

    Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.

    Measured: Week 52

Secondary outcomes (6)

  1. Survival Rate at Week 52

    Percentage of participants who were alive at Week 52, were reported. Survival rate was calculated by Kaplan-Meier estimate.

    Measured: Week 52

  2. Invasive Ventilator-Free Survival Rate at Week 52

    Percentage of participants, who were invasive ventilator-free at week 52, are reported. Invasive ventilation was defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator-free survival rate was calculated by Kaplan-Meier estimate.

    Measured: Week 52

  3. Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52

    Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates an increase in LVM-Z score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.

    Measured: Baseline, Week 52

  4. Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52

    GMFM-88 (88-item measure to detect gross motor function) consists of 5 components, each measured on a 4-point Likert scale.The score for each dimension was expressed as a percentage of the maximum score for that dimension.Total score ranges from 0% to 100%, where higher scores indicate better motor functions.

    Measured: Baseline, Week 52

  5. Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position

    Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.

    Measured: Baseline, Week 52

  6. Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position

    Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.

    Measured: Baseline, Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Feb 2012 (estimated)
  2. Study startMar 2012
  3. Primary completionDec 2014
  4. Study completionDec 2014

Why it stopped: Terminated due to approved label expansion

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
113 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01526785
Sponsor's protocol ID
AGLU09411
Other IDs
EFC12720
Status last verified
Nov 2015
First submitted
2 Feb 2012
Last updated
8 Dec 2015
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 36214004

  • 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.

    PubMed 34420548

  • 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.

    PubMed 31086307

  • 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.

    PubMed 29565424

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Alglucosidase alfa in people with Pompe Disease, run by Genzyme, a Sanofi Company at 51 sites in United States.

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 5 inclusion and 4 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: stopped early, as of the sponsor's last update on 8 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 51 sites in United States. 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?

Yes. Results are posted in the registry (first posted 27 Oct 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants Who Were Clinically Stable or Improved at Week 52 (time frame: Week 52). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01526785, which also lists 4 linked publications.

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 8 Dec 2015.

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