Clinical trials: Intrathecal Drug Delivery
AMN, AMN Gene Mutation
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy…
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- PROPEL (NCT05394064)
- Testing
- SBT101 Treatment
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 9 people
- Sponsor
- SwanBio Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
- Trial name
- ASCEND (NCT06817382)
- Testing
- INS1201
- Where
- 10 sites in United States
- Volunteers
- 12 people
- Sponsor
- Insmed Gene Therapy LLC
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
- United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Huntingtons Disease
A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042) compared with placebo in participants with manifest Huntington's disease (HD)
- Trial name
- NCT03761849
- Testing
- RO7234292
- Where
- 96 sites in United States, Argentina and 16 more
- Volunteers
- 899 people
- Sponsor
- Hoffmann-La Roche
Huntingtons Disease
A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease
Study BP40410 is an open-label, adaptive multiple-dose clinical study designed to characterize the PK of RO7234292 (RG6042) in plasma and CSF as well as the acute time course and recovery profile of CSF mHTT lowering in response…
- Trial name
- NCT04000594
- Testing
- RO7234292 (RG6042)
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Hoffmann-La Roche
Pain
ARCHIMEDES Compassionate Use Supplement
The purpose of this Compassionate Use Supplement to the IDE Protocol is to assure and monitor the safety of six Subjects (in the United States) implanted with ARCHIMEDES until all remaining implanted pumps are explanted.
- Trial name
- NCT00660556
- Testing
- Archimedes Constant Flow Pump & System
- Where
- United States
- Sponsor
- Cerenovus, Part of DePuy Synthes Products, Inc.
Chronic Pain, Cancer Pain
Prometra Post-Approval Study
The Prometra Pump is approved by the FDA for use in the United States. The purpose of this study is to collect long-term safety data on the Prometra Pump.
- Conditions
- Chronic Pain, Cancer Pain, Intractable Pain, Back Pain
- Trial name
- NCT01854229
- Testing
- Prometra Programmable Intrathecal Infusion Pump
- Where
- 26 sites in United States
- Volunteers
- 400 people
- Sponsor
- Flowonix Medical
Spinal Cord Injuries
Phase I/II Study of KP-100IT in Acute Spinal Cord Injury
This study is randomized, double-blind, placebo-controlled Phase I/II study designed to evaluate safety and efficacy of KP-100IT, code of Hepatocyte Growth Factor (HGF) formulation for intrathecal injection, as a treatment for…
- Trial name
- NCT02193334
- Testing
- KP-100IT
- Where
- 2 sites in Japan
- Volunteers
- 45 people
- Sponsor
- Kringle Pharma, Inc.
Hunter Syndrome
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter…
- Trial name
- AIM-IT (NCT02055118)
- Testing
- idursulfase-IT, No IT treatment
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 58 people
- Sponsor
- Shire
Sanfilippo Syndrome
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase).
- Trial name
- NCT02060526
- Testing
- Recombinant human heparan N-sulfatase [rhHNS]
- Where
- 10 sites in United States, Argentina and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Management of Chronic Pain
Safety and Efficacy Study of Hydromorphone Hydrochloride by Intrathecal Administration
The purpose of this study is to determine the safety and efficacy of hydromorphone hydrochloride by intrathecal administration using a programmable implantable pump.
- Trial name
- NCT01709721
- Testing
- Hydromorphone Hydrochloride, Programmable Implantable pump
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- Piramal Critical Care, Ltd.
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
Hunter Syndrome
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies.
- Trial name
- NCT06031259
- Testing
- Idursulfase-IT, Elaprase
- Where
- 4 sites in Canada, France
- Volunteers
- 6 people
- Sponsor
- Takeda
Amyotrophic Lateral Sclerosis
FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS
The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS.
- Trial name
- FUNCtion ALS (NCT07674667)
- Testing
- TRCN-1023
- Where
- 6 sites in Germany, Netherlands and 1 more
- Volunteers
- 30 people
- Sponsor
- Trace Neuroscience, Inc.
Spasticity, Muscle, Microcephaly
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss.
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation
- Trial name
- NCT05518188
- Testing
- MELPIDA
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Elpida Therapeutics SPC
Spinal Cord Injury
Intrathecal Transplantation Of Autologous Adipose Tissue Derived MSC in the Patients With Spinal Cord Injury
The effect of intrathecal transplantation of autologous adipose tissue derived mesenchymal stem cells in the patients with spinal cord injury, Phase I Clinical study.
- Trial name
- NCT01624779
- Testing
- autologous adipose tissue derived mesenchymal stem cells
- Where
- South Korea
- Volunteers
- 15 people
- Sponsor
- Bukwang Pharmaceutical
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 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
Progressive Supranuclear Palsy, Corticobasal Degeneration
A Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Primary Tauopathies
This is a phase II study intended to evaluate the safety, tolerability and pharmacodynamics of NIO752 in participants with primary tauopathies.
- Conditions
- Progressive Supranuclear Palsy, Corticobasal Degeneration, Frontotemporal Lobar Degeneration, Tauopathies
- Trial name
- NCT07868328
- Testing
- NIO752
- Volunteers
- 48 people
- Sponsor
- Novartis Pharmaceuticals
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