DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Mucopolysaccharidosis I; Hurler Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome

Intervention: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological); Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological)

Phase: Phase 2

Status: Completed

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company

Summary

The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.

Clinical Details

Official title: A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Safety Evaluation

Pharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞)

Pharmacokinetics - Elimination Half Life (t1/2)

Pharmacokinetics - Total Plasma Clearance (CL)

Pharmacokinetics - Volume of Distribution (Vz)

Eligibility

Minimum age: N/A. Maximum age: 5 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Written informed consent is required from the parent(s) or legal guardian(s) prior to

any protocol-related procedures being performed. (A separate informed consent will be requested from the parent(s) for their genotyping, which is independent of the inclusion.)

- Be less than 5 years of age at the time of enrollment.

- Have confirmed iduronidase deficiency with a fibroblast or leukocyte

alpha-L-iduronidase enzyme activity level of less than 10. 0 % of the lower limit of the normal range, or below the detection range of the measuring laboratory.

- Have a clinical diagnosis of MPS I based on genotyping.

- Documentation in his/her medical record that the parent(s) or legal guardian(s) have

had counseling or a consultation regarding HSCT in order to assure that the parent(s) or legal guardian(s) are fully informed regarding the risks and benefits of this alternative treatment for patients eligible for the trial and with the severe manifestations of MPS I with neurodegeneration. Exclusion Criteria:

- The patient is under consideration for or has undergone hematopoietic stem cell

transplantation (HSCT).

- The patient has acute hydrocephalus at the time of enrollment.

- The patient has a clinically significant organic disease (with the exception of

symptoms relating to MPS I) including: cardiovascular, hepatic, pulmonary, neurologic, or renal disease, other serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival.

- The patient has received any investigational product within 30 days prior to trial

enrollment.

- The patient has known severe hypersensitivity to Aldurazyme® (laronidase) or

components of the delivery solution.

Locations and Contacts

Hôpital E. Herriot, Lyon, France

Sophia Children's Hospital, Rotterdam, Netherlands

Willink Biochemical Genetics Unit Royal Hospital for Children, Manchester, United Kingdom

Johannes Gutenberg Universität, Kinderklinik, Mainz, Germany

Additional Information

Link to Results Synopsis for ALID-014-02

Starting date: October 2002
Last updated: March 17, 2015

Page last updated: August 20, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017