A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

February 16, 2024 updated by: Genzyme, a Sanofi Company

A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme® (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic response of their breastfed infants.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
2

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Italy
      • Rome, Italy
        • Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria (Mothers):

  • The patient must have a documented laronidase deficiency with a fibroblast, plasma, serum, leukocyte, or dried blood spot laronidase enzyme activity assay.
  • Be pregnant, planning to breastfeed post-partum, and receiving Aldurazyme (laronidase) therapy while breastfeeding.
  • Provide signed, written informed consent prior to any protocol-related procedures. Consent of a legally authorized guardian(s) is (are) required for mothers younger than 18 years of age. If a mother is under 18 years old and can understand the consent, written informed consent is required from both the mother and the authorized guardian(s).
  • Provide signed, written informed consent for their infants to participate as study patients. If a mother is younger than 18 years of age, consent for mother and infant will be obtained from the legal guardian.

Exclusion Criteria (Mothers and Infants):

  • Have a medical condition, serious intercurrent illness, or other extenuating circumstance that may interfere with study compliance, including all prescribed evaluations and follow-up activities.
  • Have received an investigational drug within 30 days prior to study enrollment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Laronidase
Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase and their infants who were breastfeed while the mothers were receiving laronidase.
Biological: Laronidase
dose of 0.58mg/kg body weight intravenously (IV) every week
Other Names:
  • Aldurazyme
  • Recombinant human alpha L iduronidase

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Lactating Women With Serum IgG Antibodies to Laronidase
Time Frame: Up to 18 months
Up to 18 months
Amount of IgG Antibody Titers to Laronidase in Lactating Women
Time Frame: Baseline and Week 12
Baseline and Week 12
Number of Women Who Breastfed
Time Frame: Up to 18 months
Up to 18 months
Number of Women Who Were Successful at Breastfeeding
Time Frame: Up to 18 months
Up to 18 months
Number of Women Whose Breast Milk Contains Laronidase
Time Frame: Up to 18 months
Up to 18 months
Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
Time Frame: Up to 18 months
Up to 18 months
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
Time Frame: Up to 18 months
Up to 18 months
Amount of uGAG in the Urine of Women
Time Frame: Baseline and Week 12
Urine samples were collected at specified intervals to measure uGAG in the urine of women. Reference range of uGAG between 2.64 - 37.65 was considered as normal. mg/g creatinine = milligram per gram of creatinine.
Baseline and Week 12
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Time Frame: Baseline
Baseline
Physical Examination Findings of the Mother
Time Frame: Up to 18 months
Physical examination was performed at specified intervals. Physical examination included the following physical observations: general appearance, skin, head, ears, eyes, nose, and throat, lymph nodes, abdomen, extremities/joints, neurological, mental status, and the following, if appropriate, breasts, external genitalia, pelvic, and rectal.
Up to 18 months
Temperature of the Mother
Time Frame: Baseline and Week 12
Baseline and Week 12
Heart Rate of the Mother
Time Frame: Baseline and Week 12
Baseline and Week 12
Respiratory Rate of the Mother
Time Frame: Baseline and Week 12
Baseline and Week 12
Blood Pressure of the Mother
Time Frame: Baseline and Week 12
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Baseline and Week 12
Weight of the Mother
Time Frame: Baseline and Week 12
Baseline and Week 12
Height of the Mother
Time Frame: Baseline
Baseline
Number of Infants With Abnormal uGAG Levels
Time Frame: Up to 72 weeks
Up to 72 weeks
Amount of uGAG in the Urine of Infants
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
Urine samples were collected at specified intervals to measure uGAG in the urine of infant. Reference range of uGAG between 30 - 300 was considered as normal.
Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
Time Frame: Baseline
Baseline
Number of Infants With Abnormal Physical Finding
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Physical examination included the following physical observations: general appearance, skin, lymph nodes, heart, lungs, abdomen, extremities/joints, neurological, mental status, breasts, external genitalia, pelvic, rectal, and Heent.
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Heart Rate of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Heart rate was measured at specified timepoints.
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Respiratory Rate of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Respiratory rate was measured at specified timepoints.
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Blood Pressure of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Weight of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Height of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Temperature of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Head Circumference of the Infant
Time Frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Time Frame: Week 12, Week 24, Week 36, Week 48, and Week 72
Infant development was assessed with the Denver II Developmental Screening Test. It consisted of 5 areas/subscore i.e., test behavior, personal-social, fine motor, language, and gross motor. The number of participants with normal overall assessment measured using Denver II Developmental Screening Scores have been reported by visit.
Week 12, Week 24, Week 36, Week 48, and Week 72
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
Time Frame: Baseline, Week 12, Week 24, Week 36, and Week 72
Baseline, Week 12, Week 24, Week 36, and Week 72
Time to Development of IgM and IgG Antibodies to Laronidase
Time Frame: up to Week 72
up to Week 72
Amount of IgG and IgM Antibody Titers to Laronidase
Time Frame: Baseline, Week 12, Week 24, Week 36, and Week 72
Baseline, Week 12, Week 24, Week 36, and Week 72

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Genzyme, a Sanofi Company

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
BioMarin/Genzyme LLC

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Sciences & Operations, Sanofi

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 22, 2010

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 21, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 21, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 3, 2007

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 4, 2007

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 5, 2007

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 20, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 16, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • OBS12874
  • 2007-007003-33 (EudraCT Number)
  • ALID01803 (Other Identifier: company study code)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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