Clinical Trials for Mucopolysaccharidoses
Clinical trials are research studies conducted in an effort to improve overall patient health and care.
Each trial involves running supervised tests to determine the effectiveness and safety of new drugs, procedures and/or devices with the aim of answering scientific questions about a disease or condition.
Risks and side-effects are also evaluated during these trials with results ranging from being unpleasant, with side effects such as headaches, to more serious or even life-threatening risks.
A clinical trial may be separated into phases, or steps, with each step designed to answer a separate research question. This maybe reflected in the trial name.
A brief description of each phase:
- Phase I: Usually designed to evaluate safety, determine a safe dosage range, and identify side effects on a small group of patients.
- Phase II: If Phase I is successful, the trial is then repeated with a larger group to further evaluate its effect and safety.
- Phase III: Trials are conducted on an ever larger group and are compared with the best current treatment while gathering more information on effect and safety.
- Phase IV: These studies monitor long-term side effects after the treatment has been marketed.
Clinical Trials data for Mucopolysaccharidoses is grouped into the following categories:-
The format for each of the following is:
- The name of the trial,
- followed by whether the trial is recruiting patients,
- The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
- Note**: Please follow the link to determine the current trial status.
- followed by a list of drug/s, if any, used in the trial
ClinicalTrials.gov has listed the following trials for Mucopolysaccharidoses:
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation - This study is no longer recruiting patients (Current: 23 Nov 2006)
- Stem Cell Transplantation for Hurler - This study is currently recruiting patients (Current: 23 Nov 2006) - Busulfan,Cyclophosphamide,ATG
- Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) - This study has been completed (Current: 23 Nov 2006)
- Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Mucopolysaccharidosis I (Hurler Disease) - This study is currently recruiting patients (Current: 23 Nov 2006) - anti-thymocyte globulin,busulfan,cyclophosphamide,cyclosporine,filgrastim,methylprednisolone
- Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I - This study is currently recruiting patients (Current: 23 Nov 2006) - laronidase
- Iduronate-2-Sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) - This study is no longer recruiting patients (Current: 23 Nov 2006) - Iduronate-2-sulfatase enzyme replacement therapy
- Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI - This study is no longer recruiting patients (Current: 23 Nov 2006) - N-acetylgalactosamine 4-sulfatase
- Open-Label Study of Efficacy and Safety of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI - This study is no longer recruiting patients (Current: 23 Nov 2006) - N-acetylgalactosamine 4-sulfatase
- Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase (rhASB) in Patients With MPS VI - This study has been completed (Current: 23 Nov 2006) - N-acetylgalactosamine 4-sulfatase
- Study of rhASB in Patients With Mucopolysaccharidosis VI - This study is no longer recruiting patients (Current: 23 Nov 2006) - N-acetylgalactosamine 4-sulfatase
- A Phase 4 Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme (Laronidase) Treated Patients. - This study is currently recruiting patients (Current: 23 Nov 2006) - Recombinant Human Alpha-L-Iduronidase, Aldurazyme® (laronidase)
- A Dose-Optimization Study of Aldurazyme (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Mucopolysaccharidosis I (MPS I) Registry - This study is currently recruiting patients (Current: 23 Nov 2006)
- Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients - This study is no longer recruiting patients (Current: 23 Nov 2006) - Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Stem Cell Transplant w/Laronidase for Hurler - This study is currently recruiting patients (Current: 23 Nov 2006) - Laronidase ERT
- A Safety Study of Aldurazyme (JC0498 Laronidase) Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - Recombinant Human Alpha-L-Iduronidase, Aldurazyme® (laronidase)
- A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI - This study is currently recruiting patients (Current: 23 Nov 2006) - galsulfase
- MPS VI Clinical Surveillance Program (CSP) - This study is currently recruiting patients (Current: 23 Nov 2006) - galsulfase
- A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme (Laronidase) in MPS I Patients Less Than 5 Years Old - This study has been completed (Current: 23 Nov 2006) - Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006) - anti-thymocyte globulin,cyclophosphamide,cyclosporine,filgrastim,methylprednisolone
- Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Mucopolysaccharidosis I (Hurler Disease) - Mucopolysaccharidosis I
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation - Mucopolysaccharidosis I
- A Safety Study of Aldurazyme (JC0498 Laronidase) Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease - Mucopolysaccharidosis I
- A Phase 4 Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme (Laronidase) Treated Patients. - Mucopolysaccharidosis I
- A Dose-Optimization Study of Aldurazyme (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease - Mucopolysaccharidosis I
- Mucopolysaccharidosis I (MPS I) Registry - Mucopolysaccharidosis I
- Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients - Mucopolysaccharidosis I
- Stem Cell Transplant w/Laronidase for Hurler - Mucopolysaccharidosis I
- Stem Cell Transplantation for Hurler - Mucopolysaccharidosis I
- Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I - Mucopolysaccharidosis I
- A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme (Laronidase) in MPS I Patients Less Than 5 Years Old - Mucopolysaccharidosis I
- Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism - Mucopolysaccharidosis I
The format for each of the following is:
- The drug,
- followed by the name of the trial,
- followed by whether the trial is recruiting patients,
- The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
- Note**: Please follow the link to determine the current trial status.
ClinicalTrials.gov has listed the following drugs in trials for Mucopolysaccharidoses:
The trial data on this page has been sourced from
