Thérapie par cellules souches pour la maladie de Parkinson à début précoce
human allogeneic induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell injection
Synucléinopathies+6
+ Maladies des ganglions de la base
+ Maladies du cerveau
Étude thérapeutique
Résumé
Date de début de l'étude : 22 septembre 2025
Date à laquelle le premier participant a commencé l'étude.Cette étude clinique vise à explorer la sécurité et l'efficacité d'un nouveau traitement appelé XS411 pour les personnes atteintes de la maladie de Parkinson à début précoce (EOPD). La maladie de Parkinson est une affection qui affecte le mouvement, et cette étude cible les personnes qui développent des symptômes à un plus jeune âge. Le traitement consiste à utiliser des cellules spéciales dérivées de cellules souches pluripotentes, qui ont le potentiel de se développer en cellules productrices de dopamine dans le cerveau, améliorant potentiellement les symptômes. Comprendre la sécurité et les bénéfices potentiels de ce traitement est crucial, car il pourrait offrir de nouveaux espoirs aux personnes atteintes de EOPD. L'étude est menée en deux phases. Dans la Phase I, 6 à 12 participants atteints de EOPD reçoivent une seule injection de XS411 à doses croissantes pour déterminer sa sécurité et la dose la plus efficace, chaque participant étant surveillé pendant au moins 28 jours. Dans la Phase II, 81 participants sont impliqués dans une évaluation plus approfondie, où ils sont répartis aléatoirement pour recevoir soit le traitement, soit un placebo, ceux du groupe de traitement recevant également un immunosuppresseur pour empêcher le corps de rejeter les cellules injectées. Les résultats aideront à évaluer l'efficacité du traitement et sa sécurité pour un usage plus large. Ce plan d'étude aide à garantir que les risques potentiels sont soigneusement gérés tout en cherchant à découvrir les éventuels bénéfices du traitement.
Protocole
Cette section fournit des détails sur le plan de l'étude, y compris la manière dont l'étude est conçue et ce qu'elle évalue.90 participants à inclure
Nombre total de participants que l'essai clinique vise à recruter.Traitement
Éligibilité
Les chercheurs recherchent des patients correspondant à une certaine description appelée critères d'éligibilité : état de santé général ou traitements antérieurs du patient.Tout sexe
Le sexe biologique des participants éligibles à s'inscrire.De 18 à 70 ans
Tranche d'âge des participants éligibles à participer.Volontaires sains non autorisés
Indique si les individus en bonne santé et ne présentant pas la condition étudiée peuvent participer.Conditions
Pathologie
Critères
Inclusion Criteria: * 18 years old ≤ age of onset ≤ 50 years old, diagnosed with EOPD (meeting the MDS 2015 clinical diagnostic criteria for Parkinson's disease ); * 18 years old ≤ age at enrollment ≤ 70 years old , male/female; * Disease duration ≥ 5 years; * Phase I: the modified Hoehn-Yahr grade in the off-phase is 3-4 (including the critical value) , and the modified Hoehn-Yahr grade in the on-phase is ≤3 ; Phase II: the modified Hoehn-Yahr grade in the off-phase is 2-4 (including the critical value) , and the modified Hoehn-Yahr grade in the on-phase is ≤3; * Off-period MDS -UPDRS-III score \>30; * Positive L-dopa stress test; * The patient is unable to adequately control motor fluctuations even with a stable dose of medication recommended in the "Guidelines for the Treatment of Parkinson's Disease in China (Fourth Edition)" ; * Patients received stable doses of anti-PD drugs for at least 4 weeks before administration; * Able to accept surgical anesthesia, suitable for neurosurgery under anesthesia, and able to undergo brain CT /MRI examination; * Participants agree to postpone any other elective neurosurgery until the completion of the 24-month follow-up study; * Participants agreed not to participate in any other clinical studies within 24 months after dosing; * Participants or their legal representatives understand and comply with the research procedures, voluntarily participate and sign the ICF Exclusion Criteria: * Non-primary PD or Parkinson's superimposed syndromes ; * Patients are in the late stages of PD and are experiencing severe, disabling peak-dose dyskinesia or biphasic dyskinesia and/or unpredictable or widely fluctuating symptoms; * Have previously undergone neuronucleotomy, deep brain stimulation (DBS), striatal surgery, extrapyramidal surgery, stereotactic brain surgery, or other brain surgery; or who have undergone other surgical procedures that the investigator determined would affect participation in this study; or who have surgical contraindications * Patients currently receiving L-dopa intestinal instillation, apomorphine injection, or continuous daily infusion of anti-PD drugs; * Patients who had used botulinum toxin, phenol, subarachnoid injection of baclofen, or received interventional treatment for dystonia or spasticity within 6 months before medication; * Have used glucocorticoids or immunosuppressive drugs for a long time within 3 months before the screening visit; * Those who have received cell therapy before; * Patients who received electroconvulsive therapy within 30 days before administration; * Those who have received or plan to receive vaccines during the trial within 3 months before screening, such as vaccines for novel coronavirus pneumonia (COVID-19), influenza, herpes zoster, and pneumococcal vaccine; * Those with a history of mental illness who are judged by the researchers to be unsuitable for study participation; or those with severe suicidal ideation currently or within the year before screening or any history of suicide attempts within the past 2 years; * Those with active epilepsy or currently taking anti-epileptic drugs; * Those with a history of dementia or severe cognitive impairment; or those with obvious dementia or cognitive impairment at screening; dementia may affect participants' poor compliance, inability to accurately record diaries, and / or inability to sign the ICF; * Severe anxiety at screening; * Patients whose previous head CT/MRI examinations showed brain injuries such as brain trauma, cerebral vascular malformation, hydrocephalus, brain tumors, or abnormal brain imaging of the striatum and other brain regions, which significantly increased the surgical risk; * Those with uncontrolled autoimmune diseases; * Patients with a history of severe cardiovascular and cerebrovascular diseases; * Patients with other serious systemic diseases; * During the screening period, patients have severe arthritis, limp, severe sequelae of stroke, severe osteoporosis, or a history of severe trauma within 1 month (such as hip or lower limb fractures), which may affect the study evaluation as assessed by the researchers; * Patients with malignant tumors or a history of malignant tumors; * Patients with active disseminated intravascular coagulation and obvious bleeding tendency within 3 months before signing the ICF, or patients who cannot stop taking antiplatelet drugs or other anticoagulants for at least 10 days before surgery; * Previous history of abnormal coagulation function; * Patients with lab result abnormalities during screening include; * Allergic to the drugs used in the study; * Are pregnant or breastfeeding, or plan to become pregnant during the study; * Female participants of fertile potential must have a positive pregnancy test result before dosing; female participants of fertile potential, or male participants who are not sterilized and whose partners are fertile, must not take effective contraceptive measures from the time they sign the ICF until at least 24 months after dosing; female participants must not agree to not donate eggs from the time they sign the ICF until at least 24 months after dosing, and male participants must not agree to not donate sperm from the time they sign the ICF until at least 24 months after dosing; * Participants who are currently participating in other clinical trials, or have participated in other clinical studies and received intervention treatment within 1 month before administration; * Those who are considered by the researchers to have poor compliance; * Any other situation that the researcher deems to jeopardize the safety of the participants or affect the study evaluation.
Plan de l'étude
Découvrez tous les traitements administrés dans cette étude, leur description détaillée et ce qu'ils impliquent.2 groupes d'intervention sont désignés dans cette étude
Cette étude ne comporte pas de groupe placebo.
Groupes de traitement
Groupe I
ExpérimentalGroupe II
ExpérimentalObjectifs de l'étude
Objectifs principaux
Objectifs secondaires
Centres d'étude
Ce sont les hôpitaux, cliniques ou centres de recherche où l'essai est conduit. Vous pouvez trouver le site le plus proche de vous ainsi que son statut.Cette étude comporte 1 site
Huashan Hospital, Fudan University
Shanghai, ChinaOuvrir Huashan Hospital, Fudan University dans Google Maps