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CompletedNCT01410890PAPAYAUpdated Mar 28, 2022Results posted

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

A Phase 4 interventional study of alglucosidase alfa in Pompe Disease and Glycogen Storage Disease Type II (GSD II), sponsored by Genzyme, a Sanofi Company. Completed at 12 sites in 6 countries. Per ClinicalTrials.gov, last updated 2022-03-28.

Sponsored by Genzyme, a Sanofi Company · Phase 4, Interventional, and Other

Phase
Phase 4
Study type
Interventional
Enrollment
21
Allocation
Not applicable
Sex
All
01

Study summary

  • The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease.
  • A secondary objective of this study was to evaluate and explore the relationship between anti-recombinant human acid alpha-glucosidase antibody titers and the PK of alglucosidase alfa.
Read the detailed description

The total study duration per participant was 4 to 8 weeks that consisted of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (greater than or equal to 30 days). Two participants enrolled prior to protocol amendment 2 (dated 17 December 2015), which changed the study to single-dose, and were treated for 26 weeks, with a 4-week follow up.

02

Conditions studied

  • Pompe Disease
  • Glycogen Storage Disease Type II (GSD II)
03

In context

Glycogen Storage Disease Type II

151 studies on the registry are indexed under Glycogen Storage Disease Type II; 30 are open to participants now.

This study's enrollment of 21 is above the median of 17 across 81 interventional studies indexed under Glycogen Storage Disease Type II.

Browse Glycogen Storage Disease Type II studies →

Lead sponsor

Genzyme, a Sanofi Company is the lead sponsor of 303 studies on the registry; 5 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

A participant was to meet all of the following criteria to be eligible for this study:

  • The participant and/or the participant's parent/legal guardian was willing and able to provide signed informed consent.
  • The participant had a confirmed acid alpha-glucosidase (GAA) enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
  • Infant and toddler Pompe disease participants could be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) would not exceed 3 percent (%) of the total blood volume during a period of 4 weeks and would not exceed 1 % at any single time.
  • The participant, if female and of childbearing potential, must have had a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female participants of childbearing potential and sexually mature males must have agreed to use a medically accepted method of contraception throughout the study.
  • For participants previously treated with alglucosidase alfa the participant had received alglucosidase alfa for at least 6 months.

Exclusion criteria

Exclusion Criteria:

A participant who met any of the following criteria was excluded from this study:

  • The participant was participating in another clinical study using an investigational product.
  • The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study.

The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

05

Study design

Phase
Phase 4
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
21 participants (actual)

Study arms

  • Experimental
    Alglucosidase alfa

    Participants received intravenous (IV) infusion of Alglucosidase alfa 20 milligrams per kilogram (mg/kg) body weight on Day 1. Infusion was administered at an initial rate of approximately 1 milligram per kilogram per hour (mg/kg/hr) with allowed rate increased of 2 mg/kg/hr every 30 minutes, if there were no signs of infusion-associated reactions (IARs), until a maximum rate of approximately 7 mg/kg/hr was reached.

    Biological: alglucosidase alfa

Interventions

  • Biologicalalglucosidase alfa

    Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)

    Also known as: Lumizyme

06

What researchers measure

Primary outcomes

  1. Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa

    Cmax was defined as maximum observed plasma concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  2. Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa

    Tmax was defined as time to reach maximum observed plasma concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  3. Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa

    AUC was defined as area under the plasma concentration-time curve from time 0 to 24 hours post-dose.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  4. Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa

    AUC0-last was defined as area under the concentration-time curve from time 0 to the time of the last quantifiable concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  5. Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa

    T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  6. Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa

    CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  7. Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa

    Volume of distribution (Vd) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

Secondary outcomes

  1. Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants

    Cmax was defined as maximum observed plasma concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  2. Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants

    Tmax was defined as time to reach maximum observed plasma concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  3. Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants

    T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  4. Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants

    AUC0-last was defined as area under the plasma concentration-time curve from time 0 to the time of the last quantifiable concentration.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  5. Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants

    AUC0-inf was defined as area under the concentration-time curve from time 0 extrapolated to infinite time.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  6. Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants

    CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

  7. Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants

    Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.

    Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1

07

Results

Posted Jun 11, 2021

Participant flow

The study was conducted at 12 sites in 6 countries. A total of 27 participants were screened between 03-Nov-2014 and 23-Sep-2020, of whom 6 participants were screen failures and 21 participants were enrolled in the study.

Participant flow — Overall Study
MilestoneAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Started1011
Treated1010
Completed1010
Not completed01
Withdrew: Withdrawal by subject01

Outcome measures

PrimaryPharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa

Cmax was defined as maximum observed plasma concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · nanograms per milliliter (ng/mL)
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa
nanograms per milliliter (ng/mL)Alglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa204000 ± 94600307000 ± 143000
PrimaryPharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa

Tmax was defined as time to reach maximum observed plasma concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Median · hours
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa
hoursAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa4.23 (1.92 to 6.42)3.85 (1.42 to 5.33)
PrimaryPharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa

AUC was defined as area under the plasma concentration-time curve from time 0 to 24 hours post-dose.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · hours per nanograms per milliliter
Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa
hours per nanograms per milliliterAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa1110000 ± 7530001890000 ± 969000
PrimaryPharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa

AUC0-last was defined as area under the concentration-time curve from time 0 to the time of the last quantifiable concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · hours per nanograms per milliliter
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa
hours per nanograms per milliliterAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa1040000 ± 5900001840000 ± 901000
PrimaryPharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa

T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · hours
Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa
hoursAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa5.43 ± 3.823.84 ± 0.801
PrimaryPharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa

CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · Liters per hour
Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa
Liters per hourAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa0.529 ± 0.6131.26 ± 1.24
PrimaryPharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa

Volume of distribution (Vd) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · Liters
Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa
LitersAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa2.35 ± 2.025.59 ± 3.72
SecondaryPharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants

Cmax was defined as maximum observed plasma concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · ng/mL
Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants
ng/mLAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants256000 ± 121000262000 ± 160000
SecondaryPharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants

Tmax was defined as time to reach maximum observed plasma concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Median · hours
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants
hoursAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants3.94 (1.92 to 5.27)4.33 (1.42 to 6.42)
SecondaryPharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants

T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · hours
Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants
hoursAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants4.68 ± 2.954.79 ± 2.93
SecondaryPharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants

AUC0-last was defined as area under the plasma concentration-time curve from time 0 to the time of the last quantifiable concentration.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · hours per nanograms per milliliter
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants
hours per nanograms per milliliterAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants1410000 ± 8650001610000 ± 86800
SecondaryPharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants

AUC0-inf was defined as area under the concentration-time curve from time 0 extrapolated to infinite time.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · hours per nanograms per milliliter
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants
hours per nanograms per milliliterAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants1450000 ± 9410001700000 ± 985000
SecondaryPharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants

CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · Liters per hour
Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants
Liters per hourAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants0.765 ± 0.5181.21 ± 1.60
SecondaryPharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants

Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.

Time frame:
Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Reported as:
Mean · Liters
Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants
LitersAnti-rhGAA Antibody Negative ParticipantsAnti-rhGAA Antibody Positive Participants
Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants5.82 ± 6.497.26 ± 9.16

Adverse events

Collected over All Adverse Events (AEs) were collected from signature of the informed consent form up to Week 4.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Alglucosidase Alfa: <18 Years0/10 (0%)1/10 (10%)4/10 (40%)
Alglucosidase Alfa: >=18 Years0/10 (0%)0/10 (0%)3/10 (30%)
Most frequent serious events
Most frequent serious events
EventAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
Respiratory DistressRespiratory, thoracic and mediastinal disorders1/100/10
Most frequent other events
Showing 10 of 12
Most frequent other events
EventAlglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 Years
NasopharyngitisInfections and infestations2/100/10
UrticariaSkin and subcutaneous tissue disorders2/100/10
Fungal Skin InfectionInfections and infestations0/101/10
RhinitisInfections and infestations1/100/10
PapuleSkin and subcutaneous tissue disorders1/100/10
OsteoporosisMusculoskeletal and connective tissue disorders1/100/10
Pain In ExtremityMusculoskeletal and connective tissue disorders0/101/10
FatigueGeneral disorders0/101/10
PyrexiaGeneral disorders1/100/10
Blood Pressure IncreasedInvestigations1/100/10

Baseline characteristics

Analysis was performed on Full analysis set (FAS) that included all participants who received any amount of alglucosidase alfa.

Age, Continuous
Age, Continuous(years)Alglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 YearsTotal
Mean5.1 ± 3.9541.8 ± 12.4423.4 ± 20.86
Sex: Female, Male
Sex: Female, Male(Participants)Alglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 YearsTotal
Female527
Male5813
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Alglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 YearsTotal
Hispanic or Latino112
Not Hispanic or Latino6814
Unknown or Not Reported314
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Alglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 YearsTotal
American Indian or Alaska Native000
Asian314
Native Hawaiian or Other Pacific Islander000
Black or African American000
White5914
More than one race202
Unknown or Not Reported000
Anti-Recombinant Human Acid Alpha-Glucosidase (rhGAA) Immunoglobulin G (IgG) Antibody Status
Anti-Recombinant Human Acid Alpha-Glucosidase (rhGAA) Immunoglobulin G (IgG) Antibody Status(Participants)Alglucosidase Alfa: <18 YearsAlglucosidase Alfa: >=18 YearsTotal
Positive347
Negative6612
Missing101
08

Study locations

12 sites
  • Investigational Site Number 840008
    Valhalla, New York 10595, United States
  • Investigational Site Number 840007
    Cincinnati, Ohio 45219, United States
  • Investigational Site Number 840005
    Salt Lake City, Utah 84108, United States
  • Investigational Site Number 840003
    Fairfax, Virginia 22030, United States
  • Investigational Site Number 1028
    Sofia, 1113, Bulgaria
  • Investigational Site Number 356001
    New Delhi, 110 029, India
  • Investigational Site Number 356002
    Vellore, 632004, India
  • Investigational Site Number 643001
    Moscow, 125367, Russian Federation
  • Investigational Site Number 643002
    Moscow, 125412, Russian Federation
  • Investigational Site Number 804001
    Kiev, 01135, Ukraine
  • Investigational Site Number 826003
    Birmingham, B4 6NH, United Kingdom
  • Investigational Site Number 826002
    Salford, M6 8HD, United Kingdom
09

References and documents

Study documents

  • Study protocol · Jul 18, 2016
  • Statistical analysis plan · Dec 23, 2016

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 28, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01410890
Lead sponsor
Genzyme, a Sanofi Company
Responsible party
Sponsor
First posted
Aug 5, 2011
Start date
Nov 3, 2014
Primary completion
Nov 20, 2020
Completion
Nov 20, 2020
Results posted
Jun 11, 2021
Last update
Mar 28, 2022

Study contacts

Medical Monitor
study director · Genzyme, a Sanofi Company

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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