Replagal Enzyme Replacement Therapy for Children With Fabry Disease
This study has been completed.
Sponsor:
Shire Human Genetic Therapies, Inc.
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT00084084
First received: June 5, 2004
Last updated: February 7, 2012
Last verified: February 2012
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Purpose
Primary Objective(s):
- To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease
- To assess the effect of Replagal on heart rate variability in patients 7 to 17 years of age
Secondary Objective(s):
- To determine the pharmacokinetics of Replagal at baseline and after the initiation of enzyme replacement therapy (ERT)
- To determine exploratory measurements of efficacy including renal function (ie, estimated glomerular filtration rate [eGFR] and creatinine clearance), clinical outcomes, sweating, and left ventricular mass index (LVMI)
| Condition | Intervention | Phase |
|---|---|---|
|
Fabry Disease |
Drug: agalsidase alfa |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Fabry disease
Farber lipogranulomatosis
Schindler disease
succinic semialdehyde dehydrogenase deficiency
Drug Information available for:
Agalsidase alfa
U.S. FDA Resources
Further study details as provided by Shire Human Genetic Therapies, Inc.:
Primary Outcome Measures:
- Assess safety [ Time Frame: 3+ years ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Determine pharmacokinetics [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
| Enrollment: | 17 |
| Study Start Date: | June 2004 |
| Study Completion Date: | December 2011 |
| Primary Completion Date: | June 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: agalsidase alfa -patients currently on treatment |
Drug: agalsidase alfa
0.2 mg/kg infused by IV over 40 minutes every other week for 52 weeks
Other Name: Replagal
|
|
Experimental: agalsidase alfa -naive patients
0.2 mg/kg infused by IV over 40 minutes every other week for 52 weeks
|
Drug: agalsidase alfa
0.2 mg/kg infused by IV over 40 minutes every other week for 52 weeks
Other Name: Replagal
|
Detailed Description:
TKT029 is an open label multi-center study to assess the safety of enzyme replacement therapy with agalsidase alfa in children with Fabry disease, who have completed 6 months of agalsidase alfa therapy in study TKT023 (Cohort 1) or are treatment-naïve (Cohort 2) and meet all inclusion/exclusion criteria of this study. The study will consist of treatment with agalsidase alfa for 52 weeks, with periodic reassessments by Shire HGT for continuation beyond the 52 weeks. A decision on the part of the study sponsor to terminate the study may be made at any time.
Study tests performed during the Baseline visit in TKT023 will serve as the baseline evaluation for the TKT029 study for patients in Cohort 1 and at Week -1 for patients in Cohort 2. The subjects will receive 0.2 mg/kg IV agalsidase alfa every other week. A number of safety assessments will be performed at Week 13, Week 25, Week 55 and thereafter, every 26 weeks.
The final study visit will follow 30 days after the last study drug infusion for patients who withdraw early from the study, at which time a final safety evaluation will be performed. Patients who complete the study will be telephone interviewed 30 days after their last study infusion for resolution of any outstanding AEs or concomitant medication changes.
Eligibility| Ages Eligible for Study: | 7 Years to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
1a. For Cohort 1 (both phases):
Patients must have completed all study requirements and assessments for Study TKT023 less than 30 (+/-7) days prior to enrolling in Study TKT029 and must have no safety or medical issues that contraindicate participation.
OR
1b. For Cohort 2:
- The patient is between 7 and 17 years of age at the time of informed consent, inclusive.
- The patient must be ERT-naive.
- The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alpha-galactosidase A activity measured in serum, leukocytes, or fibroblasts. Male patients who do not already have a documented deficiency of alpha-galactosidase A activity will provide a blood sample during screening for determination of alpha-galactosidase A activity level in their serum.
OR
- The patient is a heterozygous female or hemizygous male with Fabry disease as confirmed by a mutation of the alpha-galactosidase A gene. Patients who do not already have a documented mutation of the alpha-galactosidase A gene will provide a blood sample during screening for genotyping.
- Adequate general health (as determined by the Investigators) to undergo the specified phlebotomy regimen and protocol-related procedures and no safety or medical contraindications for participation.
- The minor child must assent to participate in the protocol and the parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed concent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legal guardian(s).
Exclusion Criteria:
Patients who meet any of the following criteria are not eligible for this study:
- Patient and/or the patient's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study.
- Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator or the medical monitor.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00084084
Locations
| United States, Arizona | |
| University of Arizona Health Sciences Center | |
| Tucson, Arizona, United States, 85724 | |
| Tucson Access Center of Arizona Kidney Disease Hypertension Center | |
| Tucson, Arizona, United States, 85719 | |
| United States, Florida | |
| Children's Physician Group | |
| Palm Beach Gardens, Florida, United States, 33410 | |
| United States, Louisiana | |
| Christus St. Patrick Hospital | |
| Lake Charles, Louisiana, United States, 70601 | |
| United States, Maryland | |
| Clinical Center, National Institutes of Health | |
| Bethesda, Maryland, United States, 20892 | |
| United States, Missouri | |
| St. Louis Children's Hospital | |
| St. Louis, Missouri, United States, 63110 | |
| United States, New York | |
| NYU School of Medicine | |
| New York, New York, United States, 10016 | |
| United States, Pennsylvania | |
| Sacred Heart Hospital | |
| Allentown, Pennsylvania, United States, 18102 | |
| United States, Tennessee | |
| East Tennessee Children's Hospital | |
| Knoxville, Tennessee, United States, 37916 | |
| United States, Texas | |
| Institute of Metabolic Diseases | |
| Dallas, Texas, United States, 75226 | |
| United States, Virginia | |
| Office of Michael Cohen | |
| Stafford, Virginia, United States, 22556 | |
| Canada, Ontario | |
| The Hospital for Sick Children | |
| Toronto, Ontario, Canada, M5G 1X8 | |
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
| Principal Investigator: | Raphael Schiffman, MD | Institute of Metabolic Disease, Baylor Research Institute |
| Principal Investigator: | Ray Pais, MD, FAAP | East Tennessee Children's Hospital |
| Principal Investigator: | Y. Howard Lien, MD, PhD | Tuscon Access Center of Arizona Kidney Disease Hypertension Center |
| Principal Investigator: | Gregory M. Pastores, MD | New York University School of Medicine |
| Principal Investigator: | Manju Thomas, MD | Sacred Heart Hospital |
| Principal Investigator: | Alison Whelan, MD | St. Louis Children's Hospital |
| Principal Investigator: | Michael E. Cohen, MD | Office of Michael Cohen |
| Principal Investigator: | Lynda Bideau, MD | Children's Physicians Group |
| Principal Investigator: | Yang-Tze Yoko Broussard, MD | Christus St. Patrick Hospital |
| Principal Investigator: | Victoria Castaneda, MD | East Tennessee Children's Hospital |
| Principal Investigator: | Li-Wen Lai, PhD | University of Arizona Health Sciences Center |
| Principal Investigator: | Tanya J. Lehky, MD | Clinical Center, National Institutes of Health |
| Principal Investigator: | Tyler Reimschisel, MD | St. Louis Children's Hospital |
| Principal Investigator: | Brian J. Corden, MD, PhD | Memorial Hospital |
| Principal Investigator: | Karen L. Johnson, MD, MPH | University of Tennessee Health Science Center |
| Principal Investigator: | Joe T. Clarke, MD, PhD | The Hospital for Sick Children |
| Principal Investigator: | Leslie F. Carroll, MD | Sacred Heart Hospital |
| Principal Investigator: | Rick A. Martin, MD | St. Louis Children's Hospital |
More Information
No publications provided
| Responsible Party: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00084084 History of Changes |
| Other Study ID Numbers: | TKT029 |
| Study First Received: | June 5, 2004 |
| Last Updated: | February 7, 2012 |
| Health Authority: | United States: Food and Drug Administration Canada: Health Canada |
Keywords provided by Shire Human Genetic Therapies, Inc.:
|
Lysosomes Storage Glycolipid Fabry disease |
Stroke Children Pediatrics |
Additional relevant MeSH terms:
|
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |
ClinicalTrials.gov processed this record on May 21, 2013