Clinical trials: Demyelinating Diseases
- Condition Demyelinating Diseases
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Multiple Sclerosis
Pregnancy Exposure Registry for Vumerity (Diroximel Fumarate)
The primary objectives of the study are to estimate the risk of major congenital malformations (MCMs) in infants born to women with multiple sclerosis (MS) who were exposed to diroximel fumarate (DRF) at any time from 2 weeks…
- Trial name
- NCT05658497
- Testing
- Diroximel Fumarate, Avonex, Tysabri
- Where
- 8 sites in United States, Australia and 5 more
- Volunteers
- 908 people
- Sponsor
- Biogen
Relapsing-Remitting Multiple Sclerosis
An Observational Study Evaluating Therapy Optimisation Using High-frequency and High-dosage Administration of Interferon-beta (Rebif®) in Multiple Sclerosis (MS) Patients
This was an observational, single arm, multicentric study conducted for the adjustment of treatment strategy and its monitoring using high-frequency and high-dosage administration of interferon-beta (Rebif) in MS subjects.
- Trial name
- NCT01142453
- Testing
- Interferon beta 1a
- Volunteers
- 231 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the…
- Trial name
- NCT01896102
- Testing
- Lenti-D Drug Product (eli-cel)
- Where
- 8 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Genetix Biotherapeutics Inc.
Infantile Globoid Cell Leukodystrophy
The Natural History of Infantile Globoid Cell Leukodystrophy
The purpose of this natural history study is to understand more about the progression of infantile Krabbe disease, a very rare genetic disease. There is very little published longitudinal data with only anecdotal cases.
- Trial name
- NCT00983879
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Zymenex A/S
Multiple Sclerosis
The Gilenya Pregnancy Registry
The purpose of the Multi-National Gilenya Pregnancy Exposure Registry in Multiple Sclerosis (MS) is to continuously monitor, evaluate, and assess for major and minor teratogenic effects in the offspring of women exposed to…
- Trial name
- NCT01285479
- Testing
- Fingolimod
- Where
- 61 sites in United States, Argentina and 32 more
- Volunteers
- 312 people
- Sponsor
- Novartis Pharmaceuticals
Multiple Sclerosis
Efficacy, Safety and Pharmacokinetics of Teriflunomide in Pediatric Patients With Relapsing Forms of Multiple Sclerosis
Primary Objective: To assess the effect of teriflunomide in comparison to placebo on disease activity measured by time to first clinical relapse after randomization in children and adolescents 10 to 17 years of age with relapsing…
- Trial name
- TERIKIDS (NCT02201108)
- Testing
- Teriflunomide
- Where
- 57 sites in United States, Belgium and 20 more
- Volunteers
- 166 people
- Sponsor
- Genzyme, a Sanofi Company
Multiple Sclerosis
Safety Study in Patients With Multiple Sclerosis Treated Fingolimod or Other Approved Disease-modifying Therapies
The purpose of this world-wide prospective parallel-cohort study in patients with relapsing forms of MS, either newly treated with fingolimod or receiving another disease-modifying therapy, is to further explore the incidence of…
- Trial name
- PASSAGE (NCT01442194)
- Testing
- other disease-modifying therapy, Fingolimod
- Where
- 238 sites in United States, Argentina and 5 more
- Volunteers
- 3,076 people
- Sponsor
- Novartis Pharmaceuticals
Multiple Sclerosis, Pregnancy
Fampridine Pregnancy Exposure Registry
This is a global pregnancy registry to evaluate the outcomes of pregnancy in women with multiple sclerosis who have been exposed to prolonged-release fampridine since the first day of their last menstrual period prior to…
- Trial name
- NCT01532154
- Testing
- Fampridine
- Where
- France
- Volunteers
- 1 people
- Sponsor
- Biogen
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Multiple Sclerosis, Progressive Multifocal Leukoencephalopathy
Risk of Progressive Multifocal Leukoencephalopathy Among Patients With Multiple Sclerosis Exposed to Zeposia
This study will evaluate the risk of progressive multifocal leukoencephalopathy (PML), a rare brain infection caused by John Cunningham virus (JCV), among people with multiple sclerosis who have been treated with ozanimod.
- Trial name
- NCT07732907
- Testing
- Ozanimod
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Bristol-Myers Squibb
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.
Alexander Disease
Zilganersen Expanded Access Program for Individuals With Alexander Disease
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
- Trial name
- NCT07487389
- Testing
- zilganersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Multiple Sclerosis, Diabetes
An Observational Post-Marketing Safety Registry of Sativex®
The purpose of this registry is to monitor safety outcomes of patients who are receiving Sativex® for Multiple Sclerosis (MS) spasticity and for off-label indications in the United Kingdom (UK), Germany and Sweden.
- Conditions
- Multiple Sclerosis, Diabetes, Cancer, Neuropathic Pain
- Trial name
- NCT02073474
- Testing
- Sativex®
- Volunteers
- 978 people
- Sponsor
- Jazz Pharmaceuticals
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study…
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Trial name
- NCT02698579
- Testing
- No interventional drug product utilized in this follow-up study
- Where
- 13 sites in United States, Argentina and 7 more
- Volunteers
- 64 people
- Sponsor
- Genetix Biotherapeutics Inc.
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Pelizaeus-Merzbacher Disease
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01005004
- Testing
- HuCNS-SC cells
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Hurler Syndrome, Inherited Metabolic Disorder
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy
- Trial name
- NCT03639844
- Testing
- rivogenlecleucel, rimiducid
- Where
- 2 sites in United States
- Sponsor
- Bellicum Pharmaceuticals
Relapsing-Remitting Multiple Sclerosis
Ireland Natalizumab (TYSABRI) Observational Program
The objectives of this study are to assess the long-term safety and impact on disease activity and progression of natalizumab (Tysabri) in participants with relapsing remitting multiple sclerosis (RRMS) in a clinical practice…
- Trial name
- iTOP (NCT01943526)
- Testing
- natalizumab
- Where
- 5 sites in Ireland
- Volunteers
- 191 people
- Sponsor
- Biogen
Cerebral Adrenoleukodystrophy (CALD)
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and…
- Trial name
- NCT03852498
- Testing
- Lenti-D
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
Relapsing-remitting Multiple Sclerosis
A Study to Evaluate Safety and Efficacy of Ocrelizumab in Comparison With Fingolimod in Children and Adolescents With Relapsing-remitting Multiple Sclerosis (RRMS)
This double-blind, double-dummy study will evaluate the safety and efficacy of ocrelizumab compared with fingolimod in children and adolescents with RRMS aged between 10 and < 18 years over a flexible duration.
- Trial name
- Operetta 2 (NCT05123703)
- Testing
- Ocrelizumab, Fingolimod
- Where
- 71 sites in United States, Argentina and 23 more
- Volunteers
- 188 people
- Sponsor
- Hoffmann-La Roche