Clinical trials: Intrathecal Drug Delivery
Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy…
- Trial name
- NCT02350816
- Testing
- HGT-1410
- Where
- 8 sites in United States, France and 5 more
- Volunteers
- 17 people
- Sponsor
- Shire
Mucopolysaccharidosis (MPS)
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01155778
- Testing
- Recombinant human heparan N-sulfatase (rhHNS)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire
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
Spinal Muscular Atrophy
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Limb Girdle Muscular Dystrophy
Cell Therapy in Limb Girdle Muscular Dystrophy
The purpose of this study was to study the effect of stem cell therapy on Limb Girdle Muscular Dystrophy patients.
- Trial name
- NCT02245711
- Testing
- Stem Cell
- Where
- India
- Sponsor
- Neurogen Brain and Spine Institute
Malignant Meningeal Neoplasms
Intrathecal Mafosfamide
This research study is an investigational treatment with the experimental drug Mafosfamide. This treatment is only for children with cancer that has spread to the meninges (tissues that cover the spinal column and brain) and has…
- Trial name
- NCT00062881
- Testing
- Intrathecal Mafosfamide
- Where
- 10 sites in United States
- Volunteers
- 65 people
- Sponsor
- Baylor College of Medicine
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
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
Leukemia, Lymphoma
Phase I Study of Intrathecal Topotecan
The purpose of this study is to determine the qualitative and quantitative toxicity of intrathecal topotecan, a topoisomerase I inhibitor, in patients with meningeal malignancies refractory to conventional therapy (radiation…
- Conditions
- Leukemia, Lymphoma, Meningeal Neoplasms
- Trial name
- NCT00001333
- Testing
- topotecan
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- National Cancer Institute (NCI)
Spinal Muscular Atrophy
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
- Trial name
- NCT07767890
- Where
- 4 sites in Japan
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Acute Lymphoblastic Leukemia, Childhood Acute Leukemia
The Effect of Mandala on Anxiety and Fear Levels in Children With Leukemia Before Intrathecal Chemotherapy Treatment
This study aims to examine the effect of mandala coloring activity, administered before intrathecal chemotherapy treatment in children with leukemia, on pre-procedure anxiety and fear in children.
- Conditions
- Acute Lymphoblastic Leukemia, Childhood Acute Leukemia, Acute Myeloblastic Leukaemia
- Trial name
- NCT07203742
- Testing
- mandala painting
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Ege 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
Neurotoxicity, Unspecified Adult Solid Tumor, Protocol Specific
Carboxypeptidase-G2 in Treating Nervous System Toxic Effects in Patients Given an Overdose of Intrathecal Methotrexate
RATIONALE: Chemoprotective drugs such as carboxypeptidase-G2 may protect normal cells from the toxic effects of chemotherapy. PURPOSE: Clinical trial to study the effectiveness of carboxypeptidase-G2 in treating nervous system…
- Conditions
- Neurotoxicity, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00018967
- Testing
- glucarpidase
- Where
- 103 sites in United States, Australia and 2 more
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
CNS Embryonal Tumor, CNS, Medulloblastoma
Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation
Pilot study to determine feasibility of adding intrathecal chemotherapy and maintenance therapy after high dose chemotherapy for treatment of newly diagnosed HR-EBTs in patients less than 6 years of age.
- Conditions
- CNS Embryonal Tumor, CNS, Medulloblastoma, Atypical Teratoid Rhabdoid Tumor, Medulloblastoma, Childhood
- Trial name
- NCT06942039
- Testing
- Cytarabine IT, hydrocortisone, Cisplatin
- Where
- 13 sites in Canada
- Volunteers
- 15 people
- Sponsor
- C17 Council
Spinal Muscular Atrophy (SMA)
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .
- Trial name
- NCT06888661
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 4 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
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
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
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
Mucopolysaccharidosis I, Lysosomal Storage Diseases
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
The investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of mucopolysaccharidosis I (MPS I).
- Conditions
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
- Trial name
- NCT00215527
- Testing
- laronidase
- Where
- 2 sites in United States, Finland
- Volunteers
- 4 people
- Sponsor
- Patricia I. Dickson, M.D.