Clinical Trials Logo

Alpha-Mannosidosis clinical trials

View clinical trials related to Alpha-Mannosidosis.

Filter by:

NCT ID: NCT06184503 Not yet recruiting - Alpha-Mannosidosis Clinical Trials

Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3

LAMPO(0-3)
Start date: May 2024
Phase:
Study type: Observational

The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis. The main questions it aims to answer are: - study the effect of velmanase alfa on a marker of the disease called GlcNAc(Man)2 after one year of therapy - explore how the child's body reacts to velmanase alfa during the therapy The parents or legal guardians of participants will be asked to provide the results of analyses performed in the routine clinical setting related to the participant's general health and the administration of velmanase alfa. Additional data will be extracted from other observational sponsored studies/registries, compassionate use programs, investigator-initiated studies (IIS), and published case reports (presented in the literature) if existing.

NCT ID: NCT04959240 Available - Alpha-Mannosidosis Clinical Trials

Expanded Access to Velmanase Alfa

Start date: n/a
Phase:
Study type: Expanded Access

Individual patient expanded access requests may be considered for patients who have no other treatment options.

NCT ID: NCT04031066 Withdrawn - Alpha-Mannosidosis Clinical Trials

Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis

SHAMAN
Start date: January 11, 2021
Phase: Phase 3
Study type: Interventional

Randomized, double-blind, placebo-controlled, parallel group study where subjects will receive velmanase alfa or placebo for 24 weeks. Each subject undergoes to 8 complete visits at the clinic for clinical, laboratory and functional assessments. Study treatment is administered weekly through i.v. infusions

NCT ID: NCT03651245 Terminated - Deafness Clinical Trials

European Alpha-Mannosidosis Participant

EUMAP
Start date: August 20, 2018
Phase:
Study type: Observational

International, multicenter, observational, longitudinal monitoring study to investigate the prevalence of Alpha-Mannosidosis in participants at risk for Alpha-Mannosidosis.

NCT ID: NCT03333200 Recruiting - Gaucher Disease Clinical Trials

Longitudinal Study of Neurodegenerative Disorders

Start date: January 11, 2012
Phase:
Study type: Observational

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

NCT ID: NCT03264040 Withdrawn - Clinical trials for Alpha-Mannosidase Deficiency

Biomarker for Mannosidosis Disease (BioMannosidosis)

BioMannosido
Start date: August 20, 2018
Phase:
Study type: Observational

Development of a new MS-based biomarker for the early and sensitive diagnosis of Mannosidosis disease from blood (plasma)

NCT ID: NCT02998879 Completed - Alpha-Mannosidosis Clinical Trials

Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis

rhLaman-08
Start date: December 2016
Phase: Phase 2
Study type: Interventional

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

NCT ID: NCT02478840 Completed - Alpha-Mannosidosis Clinical Trials

Evaluation of Long-term Efficacy of Treatment With Lamazym

rhLAMAN-10
Start date: February 2015
Phase: Phase 3
Study type: Interventional

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 Program.

NCT ID: NCT02254863 Recruiting - Clinical trials for Mucopolysaccharidosis II

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

DUOC-01
Start date: September 2014
Phase: Phase 1
Study type: Interventional

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

NCT ID: NCT02171104 Recruiting - Hunter Syndrome Clinical Trials

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Start date: July 10, 2014
Phase: Phase 2
Study type: Interventional

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 conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).