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A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease (CRSP-AID-502)

3 septembre 2026 mis à jour par: CRISPR Therapeutics

A Phase 1/2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Participants With Refractory Neurologic Autoimmune Disease.

This is a single-arm, open-label, multicenter, ascending dose Phase 1/2 trial evaluating the safety and preliminary efficacy of CTX112 in adult participants with neurological autoimmune diseases (AIDs), including Progressive Multiple Sclerosis, relapsing Neuromyelitis Optica Spectrum Disorder, relapsing Myelin Oligodendrocyte Glycoprotein Antiody-Associated Disease, refractory AutoImmune Encephalitis, and refractory Stiff Person Syndrome

Aperçu de l'étude

Statut

Pas encore de recrutement

Les conditions

Intervention / Traitement

Description détaillée

This trial will evaluate the safety and preliminary efficacy of CTX112 in adult participants with Progressive Multiple Sclerosis (PMS), relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD), relapsing Myelin Oligodendrocyte Glycoprotein Antiody-Associated Disease (MOGAD), refractory AutoImmune Encephalitis (AIE), and refractory Stiff Person Syndrome (SPS). PMS, NMOSD, MOGAD, AIE, and SPS are neurological AIDs where B cells play a significant role. Compartmentalized central nervous system (CNS) inflammation can also occur, leaving a proportion of patients unresponsive to biologics, including clusters of differentiation 19 and 20 (CD19 and CD20)-monoclonal antibodies (mAbs). Both anti-CD19 and anti-CD20 mAbs penetrate the blood-brain barrier (BBB) poorly, limiting the degree of B-cell depletion attainable within the CNS compartment. No curative treatment exists for these conditions, and despite the best available therapies a substantial proportion of patients continue to experience relapses and/or accruing disability and suffer from low quality of life combined with high morbidity and mortality. Current options focus largely on treating or preventing relapses, typically with peripherally acting agents that require repeated parenteral administration. This underscores an urgent need for novel therapies able to cross the BBB and target the CNS B-cell compartment, thereby potentially eliciting a deep immune reset and durable remission in patients who have failed multiple lines of standard therapy. Preliminary evidence suggests that chimeric antigen receptor (CAR) T cells may offer long-lasting benefits, with extended treatment-free remissions and a manageable safety and tolerability profile.CTX112 is a CD19 directed CAR-T cell immunotherapy comprised of allogeneic T cells prepared for the treatment of refractory autoimmune diseases. The cells are collected from healthy adult volunteer donors and are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA and Cas9 nuclease). As with autologous CD19-targeting CAR T cell therapy, CTX112 has the potential to generate clinical responses in patients with PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE and SPS following a one-time treatment, but with the benefit of immediate availability and without the need for the patient to undergo apheresis for collection of T cells.

This trial may enroll up to 220 participants. The total duration of trial intervention for each participant is up to 5 years.

Type d'étude

Interventionnel

Inscription (Estimé)

220

Phase

  • Phase 2
  • La phase 1

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

La description

Inclusion Criteria:

  1. Age ≥18 years and <70 years at screening.
  2. Body weight >40 kg.
  3. Participants must voluntarily sign a written informed consent and be willing and able to comply with all trial requirements. Legal authorized representative, additional assent and witness may be used as appropriate.
  4. Adequate hematologic, renal, liver, cardiac and pulmonary function.
  5. Participants must agree to use acceptable methods of contraception.
  6. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other trial procedures.
  7. Confirmed diagnosis of PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE, and refractory SPS

Exclusion Criteria:

  1. Prior treatment with any gene therapy or genetically modified cell therapy.
  2. Prior solid organ (e.g., heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
  3. Presence of another clinically significant central nervous (CNS) pathology or another condition that in the opinion of the investigator may increase CART cell-related toxicities or confound disease assessments for the main indication.
  4. Presence of other active autoimmune disease or other conditions that are likely to pose increased safety risks and/or confound disease assessments, or pose significant risk to those receiving CART cell therapy.
  5. Presence or history of certain bacterial, viral or fungal infection.
  6. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).
  7. History or current diagnosis that requires uninterrupted, ongoing anticoagulation.
  8. Pregnant or lactating.
  9. Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol.
  10. History of suicide attempts within 3 years prior to screening.
  11. Any contraindications to lumbar puncture or MRI scans

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: N / A
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: CTX112
Administré par perfusion IV après la chimiothérapie lymphodepleting
Biologique: CTX112
CTX112 is a CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components
Autres noms:
  • Zugocabtagene geleucel
  • zugo-cel

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
To evaluate the safety of CTX112 in adult participants with neurological autoimmue disorders, including PMS, relapsing NMOSD, relapsing MOGAD, refractory AIE, and refractory SPS.
Délai: From CTX112 infusion up to 28 days post infusion
Incidence of dose-limiting toxicities.
From CTX112 infusion up to 28 days post infusion

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
To assess the pharmacodynamics response to CTX112 in adults with neurological autoimmune disorders
Délai: From CTX112 infusion up to 24 months post-infusion
Number of B cells in blood over time.
From CTX112 infusion up to 24 months post-infusion
To assess the pharmacokinetics (PK) of CTX112 in adults with neurological autoimmune disorders.
Délai: From CTX112 infusion up to 24 months post-infusion.
Levels of CTX112 in blood over time.
From CTX112 infusion up to 24 months post-infusion.
To assess the preliminary efficacy of CTX112 in adults with NMOSD.
Délai: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
For participants with NMOSD, outcomes include but are not limited to, annualized rate of relapses
From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with MOGAD
Délai: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
For participants with MOGAD, outcomes include but are not limited to annualized rate of relapses
From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with PMS.
Délai: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
For participants with PMS, outcomes include but are not limited to, change from baseline in Expanded Disability Status Scale (EDSS) score
From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with AIE
Délai: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
For participants with AIE, outcomes include but are not limited to, change from baseline in the Modified Rankin Score (mRS) score
From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
To assess the preliminary efficacy of CTX112 in adults with SPS
Délai: From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24
For participants with SPS, outcomes include but are not limited to, change from baseline in Modified Rankin Score(mRS) score
From CTX112 infusion up to Month 6, Month 12, Month 18, and Month 24

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

CRISPR Therapeutics

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Estimé)

15 septembre 2026

Achèvement primaire (Estimé)

30 décembre 2029

Achèvement de l'étude (Estimé)

30 décembre 2029

Dates d'inscription aux études

Première soumission

12 août 2026

Première soumission répondant aux critères de contrôle qualité

31 août 2026

Première publication (Réel)

4 septembre 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

9 septembre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

3 septembre 2026

Dernière vérification

1 septembre 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • CRSP-AID-502

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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