Clinical trials: Alpha Mannosidosis
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Alpha-Mannosidosis
Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years
- Trial name
- rhLaman-08 (NCT02998879)
- Testing
- Velmanase Alfa (e.g. Lamazym)
- Where
- 6 sites in Austria, Denmark and 3 more
- Volunteers
- 5 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Alpha-Mannosidosis
Evaluation of Long-term Efficacy of Treatment With Lamazym
The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently receiving the treatment according to the AfterCare…
- Trial name
- rhLAMAN-10 (NCT02478840)
- Testing
- Lamazym
- Where
- Denmark
- Volunteers
- 18 people
- Sponsor
- Zymenex A/S
Alpha-Mannosidosis
Expanded Access to Velmanase Alfa
Individual patient expanded access requests may be considered for patients who have no other treatment options.
- Trial name
- NCT04959240
- Testing
- Velmanase Alfa
- Sponsor
- Chiesi Farmaceutici S.p.A.
Adrenoleukodystrophy, Batten Disease
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard treatment with umbilical cord blood transplant (UCBT) for inborn…
- Conditions
- Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell
- Trial name
- DUOC-01 (NCT02254863)
- Testing
- DUOC-01
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Joanne Kurtzberg, MD
Alpha-Mannosidase B Deficiency, Lysosomal Alpha B Mannosidosis
Biomarker for Mannosidosis Disease (BioMannosidosis)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Mannosidosis disease from blood (plasma)
- Conditions
- Alpha-Mannosidase B Deficiency, Lysosomal Alpha B Mannosidosis, Alpha-Mannosidase Deficiency
- Trial name
- BioMannosido (NCT03264040)
- Where
- 3 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Alpha Mannosidosis
The Natural History of Alpha-Mannosidosis
The natural history study of the rare lysosomal disease alpha-mannosidosis will answer the question; why the rare disease develops as it does?
- Trial name
- HUE-MAN (NCT00498420)
- Where
- 4 sites in Czechia, Germany and 2 more
- Volunteers
- 45 people
- Sponsor
- Zymenex A/S
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Mucopolysaccharidosis, Hurler Syndrome
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome
- Trial name
- NCT01043640
- Testing
- Campath-1H, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
I Cell Disease, Fucosidosis
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory…
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy
- Trial name
- NCT00005900
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Fairview University Medical Center
Alpha Mannosidosis
Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis
This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym.
- Trial name
- NCT01268358
- Testing
- Lamazym
- Volunteers
- 10 people
- Sponsor
- Zymenex A/S
Alpha-Mannosidosis
Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamzede i.v. treatment of subjects with alpha-Mannosidosis.
- Trial name
- rhLAMAN-07 (NCT01908712)
- Testing
- Lamazym
- Where
- France
- Volunteers
- 13 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Alpha-Mannosidosis
A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis
- Trial name
- NCT01681953
- Testing
- Lamazym
- Where
- 6 sites in Denmark, France and 3 more
- Volunteers
- 25 people
- Sponsor
- Zymenex A/S
Immunodeficiency, Skeletal Abnormalities
European Alpha-Mannosidosis Participant
International, multicenter, observational, longitudinal monitoring study to investigate the prevalence of Alpha-Mannosidosis in participants at risk for Alpha-Mannosidosis.
- Conditions
- Immunodeficiency, Skeletal Abnormalities, Deafness, Mental Retardation
- Trial name
- EUMAP (NCT03651245)
- Where
- 19 sites in Germany, Switzerland
- Volunteers
- 667 people
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidosis Disorders, Hurler Syndrome
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based…
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome
- Trial name
- NCT02171104
- Testing
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen
- Where
- United States
- Volunteers
- 149 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Alpha-Mannosidosis
Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
- Trial name
- NCT01681940
- Testing
- Lamazym
- Where
- 4 sites in Belgium, Denmark and 2 more
- Volunteers
- 10 people
- Sponsor
- Zymenex A/S
Alpha-Mannosidosis
Lamazym Aftercare Study
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
- Trial name
- rhLAMAN-09 (NCT01908725)
- Testing
- Lamazym
- Where
- Denmark
- Volunteers
- 8 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Alpha-Mannosidosis
Clinical Biomarkers in Alpha-mannosidosis
Background: Alpha-mannosidosis is a rare inherited disorder. It causes problems in many organs and tissues of the body. It can occur in children and adults.
- Trial name
- NCT02141503
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)