Clinical trials: Demyelinating Disease
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.
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
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
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.
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
Metachromatic Leukodystrophy (MLD)
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
- Trial name
- NCT00418561
- Testing
- rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
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
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Chronic Inflammatory Demyelinating Polyneuropathy
Non-invasive Vagus Nerve Stimulation (nVNS) in Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Participants will be requested to deliver non-invasive vagus nerve stimulation (nVNS) two times per day, at least five days per week.
- Trial name
- NCT03772717
- Testing
- Non-invasive vagus nerve stimulation (nVNS), Standard of care treatment
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Emory University
Multiple Sclerosis, Relapsing-Remitting, Clinically Isolated Syndrome
Open Label Extension of Prior TERMS Study to Treat Relapsing Forms of Multiple Sclerosis
The purpose of this study is to further evaluate the safety and efficacy of Tovaxin in the treatment of relapsing forms of multiple sclerosis.
- Trial name
- OLTERMS (NCT00595920)
- Testing
- Tovaxin
- Where
- 27 sites in United States
- Volunteers
- 116 people
- Sponsor
- Opexa Therapeutics, Inc.
Multiple Sclerosis, Relapsing-Remitting
Study of the Effect of BG00012 on MRI Lesions and Pharmacokinetics in Pediatric Subjects With RRMS
The primary objective of this study is to evaluate the effect of BG00012 (dimethyl fumarate) on brain magnetic resonance imaging (MRI) lesions in pediatric participants with relapsing-remitting multiple sclerosis (RRMS).
- Trial name
- FOCUS (NCT02410200)
- Testing
- dimethyl fumarate
- Where
- 12 sites in United States, Belgium and 8 more
- Volunteers
- 22 people
- Sponsor
- Biogen
Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE)
COVID-19 Booster Vaccine in Autoimmune Disease Non-Responders
This is a randomized, multi-site, adaptive, open-label clinical trial comparing the immune response to different additional doses of COVID-19 vaccine in participants with autoimmune disease requiring IS medications.
- Conditions
- Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE), Pemphigus Vulgaris, Multiple Sclerosis (MS)
- Trial name
- NCT05000216
- Testing
- Moderna mRNA-1273, BNT162b2, Ad26.COV2.S
- Where
- 29 sites in United States
- Volunteers
- 258 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
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
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Multiple Sclerosis
A Study to Evaluate Efficacy, Safety, and Tolerability of Alemtuzumab in Pediatric Patients With RRMS With Disease Activity on Prior DMT
Primary Objective: To evaluate the efficacy, safety, and tolerability of alemtuzumab intravenously (IV) in pediatric participants from 10 to less than (<) 18 years of age with Relapsing Remitting Multiple Sclerosis (RRMS) who…
- Trial name
- LemKids (NCT03368664)
- Testing
- Alemtuzumab GZ402673, Glatiramer acetate, Beta-Interferon
- Where
- 13 sites in France, Italy and 4 more
- Volunteers
- 16 people
- Sponsor
- Genzyme, a Sanofi Company
Disability Physical, Brain Injuries
Impact of Custom Assistive and Adaptive Technology in Rehabilitation
Madonna's Rehabilitation Engineering Center of Excellence (REC) is continually developing custom devices for persons with disabilities.
- Conditions
- Disability Physical, Brain Injuries, Stroke, Spinal Cord Injuries
- Trial name
- NCT03567239
- Testing
- Using adaptive device.
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Madonna Rehabilitation Hospital
Multiple Sclerosis, Relapsing-Remitting
Extension Study of BG00012 in Pediatric Subjects With Relapsing Remitting Multiple Sclerosis (RRMS)
The primary objective of the study is to evaluate the long-term safety of BG00012 in subjects who completed Study 109MS202 (NCT02410200).
- Trial name
- NCT02555215
- Testing
- dimethyl fumarate
- Where
- 12 sites in United States, Belgium and 8 more
- Volunteers
- 20 people
- Sponsor
- Biogen
Adrenoleukodystrophy
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II.
- Trial name
- NCT00004418
- Testing
- glyceryl trierucate/glyceryl trioleate
- Where
- 2 sites in United States
- Volunteers
- 126 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.