Clinical trials: Intrathecal Drug Delivery
Spasticity
Continuous Intrathecal Baclofen Infusion for Chronic Spasticity
The purpose of this clinical investigation is to demonstrate product performance of the Prometra Programmable Pump System in the delivery of intrathecal Lioresal® (baclofen) for the management of severe spasticity.
- Trial name
- CIBI (NCT01051128)
- Testing
- Prometra Programmable Implantable Pump System
- Sponsor
- Flowonix Medical
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
Metachromatic Leukodystrophy (MLD)
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to…
- Trial name
- EMBOLDEN (NCT03771898)
- Testing
- SHP611
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 36 people
- Sponsor
- Shire
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
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
Amyotrophic Lateral Sclerosis Als
Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis
The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).
- Trial name
- NCT06493279
- Testing
- RJK002 Intrathecal injection
- Where
- China
- Volunteers
- 9 people
- Sponsor
- RJK Biopharma Ltd
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT01780246
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Biogen
Alzheimer's Disease
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7812653 in Participants With Early Symptomatic Alzheimer's Disease (eAD)
This study aims to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, and pharmacodynamics following administration of RO7812653 in participants with eAD.
- Trial name
- NCT07234942
- Testing
- RO7812653
- Where
- 12 sites in Canada, Netherlands and 3 more
- Volunteers
- 6 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
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 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.
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.
Cerebral Palsy
Stem Cell and Conditioned Medium for Cerebral Palsy
The aim of this study was to compare the effectiveness of umbilical cord mesenchymal stem cells (UCMSCs) and conditioned medium (CM) administration, UCMSCs only and control with standard therapy.
- Trial name
- NCT04314687
- Testing
- Umbilical Cord Mesenchymal Stem Cells, Conditioned Medium, Standard Therapy
- Where
- Indonesia
- Volunteers
- 78 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
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
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
Hunter Syndrome
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT…
- Trial name
- NCT02412787
- Testing
- Idursulfase-IT, Elaprase
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 56 people
- Sponsor
- Shire
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis
The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis.
- Trial name
- NCT07093268
- Testing
- Intrathecal Riluzole
- Where
- 2 sites in Australia
- Volunteers
- 10 people
- Sponsor
- Brain Trust Bio
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
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
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.