Clinical trials: Demyelinating Disorder
- Condition Demyelinating Disorder
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Pelizaeus-Merzbacher Disease, PMD
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01391637
- Testing
- HuCNS-SC transplant in the lead-in phase
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Multiple Sclerosis
Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)
The aim of this retrospective study is to review and describe safety, tolerability and efficacy of Rebif® (subcutaneous interferon [IFN]-beta-1a) in children and adolescents, using information already recorded in medical records.
- Trial name
- REPLAY (NCT01207648)
- Testing
- Rebif®
- Where
- 18 sites in United States, Argentina and 6 more
- Volunteers
- 307 people
- Sponsor
- EMD Serono
Multiple Sclerosis (MS)
MS PATHS COVID-19 Questionnaire Data Linkage Sub-Study
The primary objective of this sub-study is to supplement the Multiple Sclerosis Partners Advancing Technology and Health Solutions (MS PATHS) dataset with coronavirus disease 2019 (COVID-19)-related health information obtained…
- Trial name
- NCT05058729
- Where
- 6 sites in United States
- Volunteers
- 1,507 people
- Sponsor
- Biogen
Multiple Sclerosis
An Observational Study on the Progression of Clinically Isolated Syndrome to Multiple Sclerosis Over a 2-year Period
This is a prospective, multicentric, observational study with a 2 years recruitment period. The purpose of the study is to observe the multiple sclerosis (MS) progression of subjects since their first episode of neurological…
- Trial name
- NEO (NCT01112657)
- Where
- 6 sites in Taiwan
- Volunteers
- 154 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
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.
Guillain-Barre Syndrome
An Open Label Clinical Study to Evaluate Tanruprubart (Also Commonly Known as ANX005) in Participants With Guillain-Barré Syndrome (FORWARD Study)
The goal of this open label study is to measure pharmacokinetics, pharmacodynamics, early efficacy, and safety of tanruprubart in adult and pediatric participants, in the United States, Canada, and Europe.
- Trial name
- NCT07020819
- Testing
- Tanruprubart
- Where
- 30 sites in United States, Denmark and 3 more
- Volunteers
- 30 people
- Sponsor
- Annexon, Inc.
Multiple Sclerosis, Exposure During Pregnancy
Biogen Multiple Sclerosis Pregnancy Exposure Registry
The primary objective of the study is to prospectively evaluate pregnancy outcomes in women with multiple sclerosis who were exposed to a Registry-specified Biogen Multiple Sclerosis product during the eligibility window for that…
- Trial name
- NCT01911767
- Testing
- Dimethyl fumarate, Peginterferon beta-1a
- Where
- 15 sites in United States, Australia and 8 more
- Volunteers
- 408 people
- Sponsor
- Biogen
Multiple Sclerosis
MATRIX: Measuring Neutralizing Antibodies in the Patients Treated With Interferon Beta 1a IM, in Mexico and Colombia
This is a cross sectional Phase 4, multicenter, study of AVONEX® and JUMTAB® to determine the frequency of IFN induced Neutralizing Antibodies (Nabs).
- Trial name
- MATRIX (NCT01556685)
- Testing
- clinical and neurological evaluation, Blood Sample
- Where
- 2 sites in Colombia, Mexico
- Volunteers
- 180 people
- Sponsor
- Biogen
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
Crohn's Disease, Prenatal Exposure
Pregnancy Exposure Registry for Tysabri®
The primary objective of the Registry was to evaluate the outcomes of pregnancy in women with Multiple Sclerosis (MS) or Crohn's Disease (CD) who were exposed to TYSABRI® at any time within 90 days prior to first day of Last…
- Conditions
- Crohn's Disease, Prenatal Exposure, Multiple Sclerosis, Pregnancy
- Trial name
- NCT00472992
- Where
- United States
- Volunteers
- 376 people
- Sponsor
- Biogen
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
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
Multiple Sclerosis
Post-Authorization Safety Study for Assessment of Pregnancy Outcomes in Patients Treated With Mayzent
This study will utilize a prospective, observational, exposure cohort design to examine pregnancy and infant outcomes in women and infants who are exposed to siponimod during pregnancy to treat MS.
- Trial name
- NCT04933552
- Testing
- Siponimod
- Where
- United States
- Volunteers
- 867 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
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
Metachromatic Leukodystrophy (MLD)
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
The purpose of this study is to determine the safety of ascending doses of HGT-1110 administered by intrathecal (IT) injection for 38 weeks (20 injections) in children with metachromatic leukodystrophy (MLD).
- Trial name
- IDEAMLD (NCT01510028)
- Testing
- Recombinant human arylsulfatase A
- Where
- 5 sites in Australia, Denmark and 3 more
- Volunteers
- 24 people
- Sponsor
- Shire
Prenatal Exposure Delayed Effects, Multiple Sclerosis
Pregnancy Exposure Registry for Avonex (Interferon Beta-1a)
The primary objectives of the study were to prospectively record and analyze birth defects and spontaneous fetal losses in women with multiple sclerosis (MS) exposed to Avonex within approximately 1 week of conception or during…
- Conditions
- Prenatal Exposure Delayed Effects, Multiple Sclerosis, Pregnancy
- Trial name
- NCT00168714
- Testing
- BG9418 (interferon beta-1a)
- Where
- United States
- Volunteers
- 329 people
- Sponsor
- Biogen
Multiple Sclerosis
Noninterventional Study Assessing Cognitive Function and Physical Activity in People With Multiple Sclerosis
Study assessing cognitive function and physical activity in people with relapsing remitting multiple sclerosis.
- Trial name
- CogniPlus (NCT01491100)
- Testing
- Interferon beta-1b (Betaseron, BAY86-5046)
- Where
- 17 sites in Albania, Algeria and 15 more
- Volunteers
- 1,085 people
- Sponsor
- Bayer
Multiple Sclerosis
A Retrospective Study to Understand the Clinical Management of Subjects With Multiple Sclerosis at a Site in Taiwan Over the Last 10 Years
This is an observational, non comparative, non-randomised, open-label, retrospective, single centre study planned to collect the data of subjects diagnosed with multiple sclerosis (MS) as per Poser or McDonald criteria between…
- Trial name
- NCT01084798
- Where
- Taiwan
- Volunteers
- 123 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
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
Relapsing-remitting Multiple Sclerosis, Active Secondary Progressive Multiple Sclerosis
Special Drug-use Surveillance for Kesimpta for s.c. Injection 20 mg Pen
This study was an uncontrolled, central registration system, open-label, multicenter observational study in patients using Kesimpta for the labeled indication.
- Trial name
- NCT04940065
- Testing
- Kesimpta
- Where
- 103 sites in Japan
- Volunteers
- 367 people
- Sponsor
- Novartis Pharmaceuticals