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

NCT00418821 | Terminated Phase 4 Results

• 3 other identifiers: OBS128742007-007003-33ALID01803
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie

Outcome Measures

Primary Outcomes (31)

• Number of Lactating Women With Serum IgG Antibodies to Laronidase

  Up to 18 months

• Amount of IgG Antibody Titers to Laronidase in Lactating Women

  Baseline and Week 12

• Number of Women Who Breastfed

  Up to 18 months

• Number of Women Who Were Successful at Breastfeeding

  Up to 18 months

• Number of Women Whose Breast Milk Contains Laronidase

  Up to 18 months

• Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease

  Up to 18 months

• Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels

  Up to 18 months

• Amount of uGAG in the Urine of Women

  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

  Baseline

• Physical Examination Findings of the Mother

  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

  Baseline and Week 12

• Heart Rate of the Mother

  Baseline and Week 12

• Respiratory Rate of the Mother

  Baseline and Week 12

• Blood Pressure of the Mother

  Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.

  Baseline and Week 12

• Weight of the Mother

  Baseline and Week 12

• Height of the Mother

  Baseline

• Number of Infants With Abnormal uGAG Levels

  Up to 72 weeks

• Amount of uGAG in the Urine of Infants

  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

  Baseline

• Number of Infants With Abnormal Physical Finding

  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

  Heart rate was measured at specified timepoints.

  Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

• Respiratory Rate of the Infant

  Respiratory rate was measured at specified timepoints.

  Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

• Blood Pressure of the Infant

  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

  Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

• Height of the Infant

  Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

• Temperature of the Infant

  Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72

• Head Circumference of the Infant

  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

  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

  Baseline, Week 12, Week 24, Week 36, and Week 72

• Time to Development of IgM and IgG Antibodies to Laronidase

  up to Week 72

• Amount of IgG and IgM Antibody Titers to Laronidase

  Baseline, Week 12, Week 24, Week 36, and Week 72

Study Arms (1)

Laronidase

EXPERIMENTAL

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

Interventions

Laronidase BIOLOGICAL

dose of 0.58mg/kg body weight intravenously (IV) every week

Also known as: Aldurazyme, Recombinant human alpha L iduronidase

Laronidase

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead
• BioMarin/Genzyme LLC: collaborator

Study Sites (1)

Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo

Rome, Italy

Location

Related Publications (1)

• Castorina M, Antuzzi D, Richards SM, Cox GF, Xue Y. Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement. therapy. Clin Exp Obstet Gynecol. 2015;42(1):108-13.

  PMID: 25864295 DERIVED

Related Links

• Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement therapy

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Limitations and Caveats

Study was conducted to fulfill a post marketing commitment (PMC). Upon acknowledgement of closure of PMC by FDA, the study was terminated by the Sponsor.

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

800-633-1610

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 3, 2007
• First Posted: January 5, 2007
• Study Start: October 22, 2010
• Primary Completion: December 21, 2022
• Study Completion: December 21, 2022
• Last Updated: February 20, 2024
• Results First Posted: February 20, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will share

Locations

IT (1)