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NCT07539662ENROLLING_BY_INVITATIONanonymous

A Single-Arm, Open-Label, Single-Center Clinical Study on Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Sponsor

Source record

Institute of Hematology & Blood Diseases Hospital, China

Phase

Source record

NA

Modality

AI-normalized

cell therapy

Target

AI-normalized

Mobilization Regimen, Conditioning Regimen, GVHD Prophylaxis, umbilical cord blood, Rescue

Indication / condition

AI-normalized

ALS (Amyotrophic Lateral Sclerosis)

Intervention

Source record

Mobilization Regimen, Conditioning Regimen, GVHD Prophylaxis, umbilical cord blood, Rescue

Source & freshness

Source record

NCT ID

NCT07539662

Original source

ClinicalTrials.gov

Source last updated

Apr 20, 2026

Ingested at

Jun 16, 2026

Internal sync

Jun 16, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

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NCT ID

NCT07539662

Title

A Single-Arm, Open-Label, Single-Center Clinical Study on Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Status

ENROLLING_BY_INVITATION

Phase

NA

Condition raw

ALS (Amyotrophic Lateral Sclerosis)

Condition normalized

ALS (Amyotrophic Lateral Sclerosis)

Modality raw

cell therapy

Modality normalized

cell therapy

Target raw

Mobilization Regimen, Conditioning Regimen, GVHD Prophylaxis, umbilical cord blood, Rescue

Target normalized

Mobilization Regimen, Conditioning Regimen, GVHD Prophylaxis, umbilical cord blood, Rescue

Interventions

Mobilization Regimen, Conditioning Regimen, GVHD Prophylaxis, umbilical cord blood, Rescue

Public preview

Source record

Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive and fatal neurodegenerative disorder. Its global prevalence is approximately 0.73-1.89 per 100,000 individuals. In China, there are about 200,000 ALS patients, with approximately 25,000 new cases diagnosed annually. Microglia, the resident immune cells of the central nervous system (CNS), rapidly transition from a resting state to a pro-inflammatory phenotype (M1) in ALS. This activation leads to the release of a large number of inflammatory factors (such as TNF-α, IL-1β, IL-6, NO, ROS) and chemokines (such as MCP-1/CCL2), and triggers the NLRP3 inflammasome. Furthermore, systemic immune dysregulation plays a significant role in the pathogenesis of ALS. ALS patients exhibit reduced numbers of regulatory T cells (Tregs), alterations of activated CD8+ T cell infiltrates, and a shift in the helper T cell (Th1/Th2) balance towards the pro-inflammatory Th1 phenotype. In recent years, therapeutic strategies targeting novel pathways such as neuroinflammation, immune dysregulation, and energy metabolism have emerged, including the infusion of Tregs, mesenchymal stem cells (MSCs), and neural stem cells. However, these approaches have still failed to halt disease progression \[NCT05695521, NCT03280056, NCT06973629, NCT02290886\]. Recent research suggests that hematopoietic stem cell transplantation (HSCT) may disrupt the activation cycle between astrocytes and microglia, alleviate chronic inflammatory states in the CNS, partially mitigate mitochondrial dysfunction, and thereby slow neurodegeneration. Unrelated umbilical cord blood transplantation offers advantages such as low HLA-matching requirements, a lower risk of graft-versus-host disease (GVHD), a potent graft-versus-leukemia (GVL) effect, and immediate availability. Investigators plan to conduct an exploratory clinical trial to evaluate the safety and efficacy of umbilical cord blood transplantation for ALS patients. The preliminary plan is to enroll 8 adult subjects. Following successful neutrophil engraftment (defined as an absolute neutrophil count ≥0.5×10⁹/L for three consecutive days) and confirmation of complete donor chimerism. The trial will focus on assessing transplantation-related complications and patient tolerance. A 3-month post-transplantation follow-up will be conducted for a comprehensive evaluation of safety and efficacy for ALS patients.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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