Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study is enrolling participants by invitation only.
Sponsor:
Shire Human Genetic Therapies, Inc.
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT01124643
First received: April 23, 2010
Last updated: June 25, 2012
Last verified: June 2012
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Purpose
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult patients with Fabry disease who have completed Study TKT028.
| Condition | Intervention | Phase |
|---|---|---|
|
Fabry Disease |
Biological: Replagal |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease |
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:
- To evaluate the safety and effect of continued dosing with Replagal [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]To evaluate the long term safety and effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week)following 53 weeks of treatment in Study TKT028 on the reduction from baseline in left ventricular mass (LVM) as measured by echocardiography
Secondary Outcome Measures:
- Effect on exercise tolerance as measured by maximal oxygen consumption and distance walked in meters [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]Secondary Outcome Measures: Excercise tolerance, as measured by maximal oxygen consumption (VO2 max) using the standard exponential exercise protocol (STEEP) and by distance walked in meters (m) using the 6-Minute Walk Test (6MWT)
| Estimated Enrollment: | 43 |
| Study Start Date: | April 2010 |
| Estimated Study Completion Date: | June 2013 |
| Estimated Primary Completion Date: | June 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Replagal 0.2 mg/kg EOW |
Biological: Replagal
0.2 mg/kg administered intravenously [IV] every other week
Other Name: Replagal, algalsidase alfa, alpha-Galactosidase
|
Detailed Description:
The objective of the study is to evaluate the long term safety and effect of continued dosing with Replagal (0.2 mg/kg administered intravenously [IV] every other week) following 53 weeks of treatment in Study TKT028 on the reduction from baseline in left ventricular mass (LVM) as measured by echocardiography
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Complete all study requirements and assessments for Study TKT028 less than 30 days (+/- 7 days) prior to the first dose in this extension protocol
- Voluntarily signed an Institutional Review Board/Independent Ethics Committee- approved informed consent form after all relevant aspects of the study have been explained and discussed.
- Has received and tolerated at least 80% of the total planned Replagal infusions in Study TKT028
- Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have a negative pregnancy test at the time of study entry and as required throughout participation in study
Exclusion Criteria:
- Has received treatment with any investigational drug (other than Replagal) or device within 30 days prior to study entry.
- Is unable to comply with the protocol, (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator
- Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C(HCV) antibody, or human immunodeficiency virus (HIV) antibody
- Is pregnant or lactating
- Is morbidly obese, defined as body mass index (BMI) >39 kg/m2
- Has any safety or medical issues, as assessed by the Investigator, that contraindicate participation in the study (eg, has experienced an adverse reaction to treatment with Replagal or has a known hypersensitivity to any of the components of Replagal
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01124643
Locations
| United States, Iowa | |
| University of Iowa Hospitals and Clinics | |
| Iowa City, Iowa, United States, 52242 | |
| United States, Virginia | |
| O&O Alpan LLC | |
| Fairfax, Virginia, United States, 22030 | |
| Australia | |
| The Royal Melbourne Hospital | |
| Parkville, Victoria, Australia, 3050 | |
| Czech Republic | |
| 1st School of Medicine Charles University | |
| Prague, Czech Republic | |
| Finland | |
| Turku University Central Hospital | |
| Turku, Finland, FI-20520 | |
| Poland | |
| Szpital Uniwersytecki w Krakowie | |
| Krakow, Poland, 31-066 | |
| Instytut Kardiologii, I Klinika Choroby Wiencowej | |
| Warsaw, Poland | |
| Slovenia | |
| General Hospital Slovenj Gradec | |
| Slovenj Gradec, Slovenia, 2380 | |
| United Kingdom | |
| Salford Royal NHS Foundation Trust | |
| Salford, England, United Kingdom, M6 8HD | |
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
| Principal Investigator: | Lubor Golan | 1st School of Medicine, Charles University, Prague |
| Principal Investigator: | Lidia Chojinowska | Instytut Kardiologii |
| Principal Investigator: | Kathleen Nicholls, MB.BS, M.D. | Melbourne Health |
| Principal Investigator: | Jacek Musial, MD, PhD | Szpital Uniwersytecki w Krakowie |
| Principal Investigator: | Ilkka Kantola, MD, PhD | University of Turku |
| Principal Investigator: | Reena Sharma, MD | Salford Royal NHS Foundation Trust |
| Principal Investigator: | Derlis Emilio Gonzalez Rodriguez, MD | Instituto Privado de Hematologia e Investigacion Clinica (IPHIC) |
| Principal Investigator: | Ozlem Goker-Alpan, MD | O & O Alpan LLC |
| Principal Investigator: | Bojan Vujkovac, MD | General Hospital Slovenj Gradec |
| Principal Investigator: | Myrl Holida, PA-C | University of Iowa |
More Information
No publications provided
| Responsible Party: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT01124643 History of Changes |
| Other Study ID Numbers: | HGT-REP-060 |
| Study First Received: | April 23, 2010 |
| Last Updated: | June 25, 2012 |
| Health Authority: | United States: Food and Drug Administration Poland: Ministry of Health Czech Republic: State Institute for Drug Control Australia: Department of Health and Ageing Therapeutic Goods Administration United Kingdom: Medicines and Healthcare Products Regulatory Agency Paraguay: Ministerio de Salud Pública y Bienestar Social |
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 16, 2013