[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hereditary-spastic-paraparesis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hereditary-spastic-paraparesis":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,53,101],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":36,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":41,"lastUpdatePostDateStruct":42,"startDateStruct":45,"completionDateStruct":47,"leadSponsor":49,"locationsCount":52},"100599866","the-effect-of-targeting-the-plantaris-muscle-tendon-in-surgical-correction-of-ankle-equinus-in-children-100599866",false,"NCT07090057","The Effect of Targeting the Plantaris Muscle-tendon in Surgical Correction of Ankle Equinus in Children","The Effect of Targeting the Plantaris Muscle-tendon Unit in Surgical Correction of Ankle Equinus in Children","Plantaris Sx","Inclusion Criteria (All of the following criteria must be met):\n\n* Ability to provided informed consent\u002Fassent in English.\n* Pediatric patients (4-17 years) who have consented for surgery for the management of equinus contracture \\* (either TA lengthening or GN recession) at the Stollery Children's Hospital\n* Known underlying diagnosis of any of the following: idiopathic toe walking, cerebral palsy, hereditary spastic paraparesis, traumatic brain injury, spinal cord injury\u002Ftethering, hereditary sensory-motor neuropathy, stroke\n* Ability to maintain hindfoot and midfoot neutral during assessment\n* Passive plantarflexion on affected side greater than 20° and greater than degree of equinus contracture.\n\n  * Note: may be isolated or in conjunction with other orthopaedic procedures; in bilateral ankle equinus procedures, data will be collected bilaterally, but included as a single participant (i.e., single randomization).\n\nExclusion Criteria (Any one or more of the following):\n\n* Unable to provide informed consent\u002Fassent in English.\n* Previous surgery for equinus\n* Limb deficiency on affected side\n* Knee flexion contracture of greater than 5°\n* Surgical intervention of the lower extremities below the affected knee in the last twelve months\n* BoNTA injections below the affected knee within the last six months\n* Known or suspected arthrofibrosis.","ALL","4 Years","17 Years",{"count":21,"type":22},42,"ESTIMATED","INTERVENTIONAL",[25],"NA","Tight ankle muscles can produce ankle equinus (limited ability to pull the foot upward) and occur often in children, significantly impacting their ability to walk. If not treated, children with ankle equinus frequently experience reduced function and long-term foot problems, such as pain. Currently, treatment options include surgery or Botulinum toxin (BoNTA) injection into the large calf muscles that point the foot downwards, aiming to reduce their tightness. However, these treatments can be less effective over time, can create prolonged calf weakness, and may require long-term bracing. Another small muscle in the leg, the plantaris, is believed to have some contribution to equinus in many children. It is sometimes included in treatment plans for equinus but its contribution is poorly understood. It is unclear whether targeting the plantaris alone could lead to better treatment of ankle equinus. Understanding the effect of treatments targeting the plantaris could help clinicians improve the management of ankle equinus.\n\nIn this study, the investigators will look at the impact of surgical treatment to the plantaris in ankle equinus. The investigators hypothesize that the plantaris is a significant contributor to equinus.\n\nIn this study, data will be collected from children undergoing surgical correction of ankle equinus, including lengthening of the plantaris and lengthening of the larger muscles producing equinus (the gastrocsoleus mechanism). Children will be randomly assigned to have either their plantaris or the gastrocsoleus lengthening be done first during surgery. All children will have both structures lengthened during surgery, only the order will be varied and all surgical procedures for each patient will be completed in a single setting. In both groups, maximum passive ankle dorsiflexion (upwards bend of the ankle with the knee straight) will be measured before and after each structure is lengthened. The outcome is maximum passive ankle dorsiflexion (upwards bend of the ankle) with the knee straight.\n\nThe investigators expect that maximum passive ankle dorsiflexion will increase after lengthening of the plantaris. Understanding the contribution of the plantaris muscle in ankle equinus could lead to significant improvements in the treatment of children with tight ankles.",[28,29,30,31,32,33,34,35],"Idiopathic Toe Walking","Cerebral Palsy","Hereditary Spastic Paraparesis","Traumatic Brain Injury","Spinal Cord Injury","Spinal Cord Tethering","Stroke","Hereditary Sensory-motor Neuropathy",[37,38,39],"tendoachilles lengthening","gastrocnemius recession","Plantaris lengthening","RECRUITING","2026-03-11",{"date":43,"type":44},"2026-03-12","ACTUAL",{"date":46,"type":44},"2023-03-13",{"date":48,"type":22},"2026-12-31",{"name":50,"class":51},"University of Alberta","OTHER",1,{"id":54,"slug":55,"hasResults":11,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":4,"eligibilityCriteria":59,"healthyVolunteers":11,"sex":17,"minAge":60,"maxAge":61,"enrollmentInfo":62,"targetDuration":4,"studyType":23,"phases":64,"briefSummary":67,"conditions":68,"keywords":81,"overallStatus":90,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":52},"100588946","phase-1-safety-and-efficacy-of-aav9ap4b1-bfb-101-for-patients-with-ap4b1-related-hereditary-spastic-paraplegia-type-47-spg47-100588946","NCT06948019","Safety and Efficacy of AAV9\u002FAP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)","Safety and Efficacy of AAV9\u002FAP4B1 For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1\u002F2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration","Inclusion Criteria:\n\n1. Male and females between the ages of 12 months - 5 years at the time of treatment\n2. A molecularly confirmed diagnosis of SPG47 (confirmed by a CLIA certified, CE-marked, or equivalent lab): Genomic DNA mutation analysis demonstrating bi-allelic pathogenic variants in the AP4B1 gene.\n3. Proband must have features of neurologic dysfunction by clinical history and physical examination.\n4. Stable doses of concomitant medications such as anti-spasticity medications, anti-epileptic medications, behavioral management medications, sleep medications, and special diets, supplements or nutritional support for at least 3 months prior to Screening. If recent changes (\\\u003C 3 months) in medications, the participant may be allowed per Investigator judgement.\n5. Proband must be fully vaccinated per Centers for Disease Control recommendations for childhood vaccinations.\n6. Two competent custodial parents\u002Fguardians with legal capacity (legally acceptable representatives) to execute an Institutional Review Board\u002FIndependent Ethics Committee (IRB\u002FIEC) approved consent for medical research must be able to participate in the consent process. If only one parent has sole custody to consent for medical research, then that parent must be able to actively participate in the consent process.\n7. Legally acceptable representatives must be able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol.\n8. Legally acceptable representatives agree not to post any of the participant's personal medical data or information related to the study on any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed.\n9. Proband and the proband's family must demonstrate ability to travel to the study center. For the first 30 days post treatment probands will need to stay within a 100-mile radius from the treatment center.\n\nExclusion Criteria:\n\n1. Inability to participate in the clinical evaluation as determined by the principal investigator.\n2. Clinically significant abnormal laboratory values (hemoglobin \\\u003C 8 or \\> 20 g\u002FdL; white blood cell \\> 20,000 per cmm, platelets count \\\u003C 100,000 per cmm; international normalized ratio \\[INR\\] \\> upper limit of normal \\[ULN\\]; gamma-glutamyl transferase \\[GGT\\], alanine aminotransferase \\[ALT\\], and aspartate aminotransferase \\[AST\\] or total bilirubin \\> 1.5 × ULN, creatinine\n\n   ≥ 1.5 mg\u002FdL) prior to gene replacement therapy.\n3. Presence of a concomitant medical condition that precludes a cisterna magna or lumbar puncture or use of anesthetics for sedated procedures.\n4. Bleeding disorder or any other medical condition or circumstance in which a cisterna magna or lumbar puncture is contraindicated according to local institutional policy.\n5. Documented cardiomyopathy or significant congenital heart abnormalities.\n6. Inability to be safely sedated in the opinion of the clinical anesthesiologist.\n7. History of severe\u002Flife-threatening allergic reaction to sirolimus, tacrolimus, corticosteroids, or gadolinium.\n8. Any known history and\u002For family history of hemophagocytic lymphohistiocytosis (HLH) or multisystem inflammatory syndrome (MIS)\n9. Concomitant illness or requirement for chronic drug treatment that in the opinion of the investigator creates unnecessary risks for gene transfer.\n10. Concomitant chronic drug treatment that would cause clinically significant interactions with immunosuppressive agents used in the study.\n11. Any item which would exclude the participant from being able to undergo magnetic resonance imaging (MRI) according to local institutional policy.\n12. Any other situation that would exclude the participant from undergoing any other procedure required in this study.\n13. Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing.\n14. The presence of significant non-SPG47 related central nervous system (CNS) impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study.\n15. Recent or planned elective surgical procedures that would confound the scientific rigor or interpretation of results of the study, as determined by the Investigator\u002Fstudy team.\n16. Failure to obtain appropriate informed consent.\n17. Reason to believe that the participant or parents\u002Fguardians of the participant will not comply with the study procedures outlined in the study protocol.\n18. Receiving a live vaccine within 30 days prior to gene transfer.\n19. Receiving an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study.\n20. Enrollment and participation in another interventional clinical trial.","12 Months","60 Months",{"count":63,"type":22},5,[65,66],"PHASE1","PHASE2","Safety and Efficacy of AAV9\u002FAP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1\u002F2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.\n\nThe goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are:\n\n* Is the gene therapy safe and well tolerated?\n* Does the gene therapy improve motor function and developmental outcomes?\n\nParticipants will:\n\n* Undergo screening assessments to confirm eligibility\n* Receive a single dose of the gene therapy vector\n* Attend follow-up visits for safety monitoring and developmental assessments over the course of five years",[69,70,30,71,72,73,74,75,76,77,78,79,80],"HSP","Hereditary Spastic Paraplegia","Hereditary Spastic Paraplegia Type 50","Hereditary Spastic Paraplegia Type 47","Hereditary Spastic Paraplegia Type 51","Hereditary Spastic Paraplegia Type 52","SPG47","AP4B1","Neurogenetic Disorders","Neurodevelopmental Conditions","Movement Disorders","Gene Therapy",[69,70,71,72,73,74,30,82,80,83,75,76,84,85,86,87,88,89],"Spasticity","AAV9","AP4M1","AP4E1","AP4S1","movement disorders","neurogenetic conditions","neurodevelopmental conditions","NOT_YET_RECRUITING","2025-04-22",{"date":93,"type":44},"2025-04-28",{"date":95,"type":22},"2025-08",{"date":97,"type":22},"2032-08",{"name":99,"class":100},"BlackfinBio Ltd","INDUSTRY",{"id":102,"slug":103,"hasResults":11,"nctId":104,"briefTitle":105,"officialTitle":105,"acronym":106,"eligibilityCriteria":107,"healthyVolunteers":11,"sex":17,"minAge":108,"maxAge":4,"enrollmentInfo":109,"targetDuration":4,"studyType":23,"phases":111,"briefSummary":112,"conditions":113,"keywords":114,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":52},"100533731","evaluation-of-an-intensive-training-program-for-patients-with-hereditary-spastic-paraparesis-spg4spast-100533731","NCT06229626","Evaluation of an Intensive Training Program for Patients with Hereditary Spastic Paraparesis SPG4\u002FSpast","WALK-up","Inclusion Criteria:\n\n* Patient with molecular diagnosis of hereditary spastic paraparesis based on pathogenic variant of SPAST gene,\n* Walking possible for 6 minutes without human assistance (one or more technical aids are authorized: e.g. cane, walker, orthoses),\n* At least 1 physiotherapy session per week already in place.\n* Understanding of the protocol\n* Possibility of connecting to the Internet from home to access video material provided as part of the protocol.\n\nExclusion Criteria:\n\n* Botulinum toxin injection within 2 months of protocol inclusion\n* Discontinuation of private physiotherapy,\n* Refusal to participate in the protocol,\n* Participation in another interventional clinical trial evaluating a health product or in a randomized clinical trial\n* Pregnant women\n* Not affiliated to a social security scheme or beneficiary of such a scheme\n* Patient under guardianship or trusteeship","18 Years",{"count":110,"type":22},50,[25],"Hereditary spastic paraparesis is a group of inherited neurological diseases. Only symptomatic treatments exist for the moment. The Modifspa study (cf citation) carried out by the team showed that patients perceived a feeling of effectiveness of physiotherapy on lower limb spasticity. The aim of the Walk-up study is to objectivize this feeling of efficacy on gait disorders in these patients.\n\nThis is an interventional study using physical training. The study is prospective, open, randomized in 2 parallel groups, one of which is a control group. Analyses will be comparative between the 2 groups during the course of the study.",[30],[115,116],"physical rehabilitation program","hereditary spastic paraparesis","2024-11-21",{"date":119,"type":44},"2024-11-25",{"date":121,"type":44},"2024-04-04",{"date":123,"type":22},"2026-10",{"name":125,"class":51},"Assistance Publique - Hôpitaux de Paris"]