Clinical trials: Spastic Paraplegia
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, 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
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
Muscle Spasticity, Hypertonia, Muscle
Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals
To assess the safety and tolerability of single and multiple doses of MTR-601 in normal healthy volunteers under fed and fasted conditions. To evaluate the plasma and urine pharmacokinetics (PK) of MTR-601.
- Conditions
- Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis
- Trial name
- NCT06117020
- Testing
- MTR-601
- Where
- United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Motric Bio
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Cerebral Palsy, Spastic Paraplegia and Hemiparesis
Treatment With Xeomin Versus Botox in Children With Spastic Equine and Equinovarus Foot Deformation in Pediatric Cerebral Palsy
1. To assess the clinical and neurophysiological efficacy of Xeomin® vs. Botox® in children with spastic equine and equinovarus foot deformation in pediatric cerebral palsy 2.
- Conditions
- Cerebral Palsy, Spastic Paraplegia and Hemiparesis, Equine and Equinovarus Foot Deformation
- Trial name
- XEBEC (NCT02188277)
- Testing
- Xeomin, Botox®
- Where
- 3 sites in Russia
- Volunteers
- 64 people
- Sponsor
- Merz Pharmaceuticals GmbH
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.
Cerebral Palsy (CP), Spastic Paraplegia
Retrospective Study on the WalkOn AFO in Children or Teenagers With Neurological Pathologies
The goal of this observational study is to assess the interest of prefabricated carbon fiber ankle foot orthoses (AFO) in children and teenagers with central or peripheral neurological pathologies.
- Conditions
- Cerebral Palsy (CP), Spastic Paraplegia, Spinal Cord Injuries (SCI), Spina Bifida
- Trial name
- NCT06740006
- Where
- France
- Volunteers
- 65 people
- Sponsor
- Otto Bock France SNC