Clinical trials: Intrathecal Drug Delivery
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Cerebral Palsy
Intrathecal Stem Cells for Cerebral Palsy Phase II
The purpose of this study is to determine whether the infusion of intrathecal autologous bone marrow total nucleated cells would improve the neurologic evolution of pediatric patients with quadriparetic cerebral palsy.
- Trial name
- ISCII (NCT02231242)
- Testing
- Autologous Stem Cell Transplantation
- Where
- Mexico
- Volunteers
- 60 people
- Sponsor
- Hospital Universitario Dr. Jose E. Gonzalez
Spastic/Dystonic Quadriplegia, Cerebral Palsy (CP)
Outcomes of Neurosurgical Tone Management
Patients with spastic/dystonic quadriplegia (GMFCS IV/V) are often severely disabled by their tone, and require surgical tone management to improve quality of life, allow for better function, improve comfort, and range of motion.
- Trial name
- NCT07817745
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Boston Children's Hospital
Cerebral Palsy (CP)
Intrathecal Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells for Children With Cerebral Palsy
This is a single-center, open-label, single-arm Phase I pilot study designed to evaluate the feasibility and short-term safety of a single intrathecal administration of allogeneic umbilical cord tissue-derived mesenchymal stromal…
- Trial name
- NCT07763028
- Testing
- allogeneic umbilical cord tissue-derived mesenchymal stromal cells (UCT-MSCs)
- Where
- Indonesia
- Volunteers
- 7 people
- Sponsor
- Gadjah Mada University
Hereditary Spastic Paraplegia Type 50
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).
- Trial name
- SPG50 (NCT06692712)
- Testing
- MELPIDA
- Where
- 2 sites in United States, Spain
- Volunteers
- 24 people
- Sponsor
- Elpida Therapeutics SPC
Neoplastic Meningitis
Phase 1 Intrathecal Topotecan for Neoplastic Meningitis
1. To find the optimal dose of topotecan that can safely be given directly into the spinal fluid (called intrathecal administration) of children whose cancer has spread to the lining of the brain and/or spinal cord. 2.
- Trial name
- PBTC-019 (NCT00674674)
- Testing
- Topotecan
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Baylor College of Medicine
Brain and Central Nervous System Tumors, Extragonadal Germ Cell Tumor
Melphalan in Patients With Neoplastic Meningitis
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of melphalan in patients with persistent or recurrent…
- Conditions
- Brain and Central Nervous System Tumors, Extragonadal Germ Cell Tumor, Leukemia, Lymphoma
- Trial name
- NCT00002750
- Testing
- melphalan
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Duke University
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
Cerebral Palsy, Intrathecal Baclofen
Predictive Factors for Succes of ITB in CP
For children with severe cerebral palsy (CP) in whom other options are insufficient, intrathecal baclofen (ITB) treatment could be an option.
- Trial name
- NCT05817409
- Testing
- Intrathecal Baclofen Pump
- Volunteers
- 75 people
- Sponsor
- Amsterdam UMC, location VUmc
Infantile GM2 Gangliosidosis (Disorder)
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of…
- Trial name
- NCT04798235
- Testing
- TSHA-101
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Dr. Anupam Sehgal
Leukemia, Lymphoid Malignancies
Intrathecal Rituximab in Lymphoid Malignancies Involving Central Nervous System
The goal of this clinical research study is to learn about the safety and effectiveness of rituximab given by spinal tap in patients with lymphoid malignancies involving the central nervous system.
- Conditions
- Leukemia, Lymphoid Malignancies, Metastatic Malignant Neoplasm to the Leptomeninges
- Trial name
- NCT01596127
- Testing
- Intrathecal Rituximab
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- M.D. Anderson Cancer Center
Spinal Cord Injuries
Safety of Cultured Allogeneic Adult Umbilical-cord or Placental Derived Mesenchymal Stem Cell Intrathecal Injection for SCI
This trial will study the safety and efficacy of intrathecal injection of cultured allogeneic adult umbilical-cord or placental derived mesenchymal stem cells for the treatment of spinal cord injury
- Trial name
- NCT05018793
- Testing
- Cultured Allogeneic Adult Umbilical-cord or Placental Derived Mesenchymal Stem Cell Intrathecal injection
- Where
- 2 sites in United States, Greece
- Volunteers
- 15 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
CLN7
Study for the Treatment for CLN7 Disease
This is a phase 1 open-label, single-administration of gene therapy agent AAV9/CLN7, administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN7 Batten disease.
- Trial name
- CLN7 (NCT04737460)
- Testing
- AAV9/CLN7
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Benjamin Greenberg
GM2 Gangliosidosis, Tay Sachs Disease
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
This study is to continue Long-Term Follow-Up of Patients who were previously treated with AXO-AAV-GM2 Gene Therapy as treatment for Tay-Sachs or Sandhoff Disease to follow the subjects through 5 years after their initial gene…
- Conditions
- GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Trial name
- NCT06614569
- Testing
- AXO-AAV-GM2
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Terence Flotte
Metachromatic Leukodystrophy (MLD)
Direct Lentiviral Injection Gene Therapy for MLD
This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect.
- Trial name
- NCT03725670
- Testing
- Intrathecal and intravenous LV gene therapy
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Lymphoma, Non-Hodgkin, Child, Only
Safety and Efficacy of Intrathecal Rituximab in 16 Children of Stage Ⅲ、ⅣNon-Hodgkin Lymphoma
Objective: This study demonstrated that the efficacy and safety of intrathecal(IT) rituximab in the treatment of stage Ⅲ and Ⅳ non-Hodgkin lymphoma(NHL) in children.
- Trial name
- NCT06190457
- Testing
- intrathecal rituximab
- Volunteers
- 16 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Ataxia Telangiectasia
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene.
- Trial name
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy Yu
Brain and Central Nervous System Tumors, Intraocular Melanoma
Monoclonal Antibody Therapy in Treating Patients With Leptomeningeal Cancer
RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Conditions
- Brain and Central Nervous System Tumors, Intraocular Melanoma, Lung Cancer, Melanoma (Skin)
- Trial name
- NCT00003022
- Testing
- iodine I 131 monoclonal antibody 3F8
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Brain and Central Nervous System Tumors, Neuroblastoma
Radiolabeled MAB Therapy in Patients With Refractory, Recurrent, or Advanced CNS or Leptomeningeal Cancer
The purpose of this study is to test the feasibility and toxicity of administering intrathecal immunotherapy for patients with central nervous system/leptomeningeal (CNS/LM) malignancies.
- Conditions
- Brain and Central Nervous System Tumors, Neuroblastoma, Sarcoma
- Trial name
- NCT00089245
- Testing
- Iodine I 131 MOAB 8H9
- Where
- United States
- Volunteers
- 177 people
- Sponsor
- Y-mAbs Therapeutics
Brain and Central Nervous System Tumors
Topotecan in Treating Patients With Recurrent, Progressive, or Refractory Cancer That is Metastatic to the Lining Around the Brain
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of intrathecal or intraventricular topotecan in treating…
- Trial name
- NCT00025311
- Testing
- topotecan hydrochloride
- Where
- 3 sites in United States, France
- Sponsor
- NYU Langone Health