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L'essai clinique NCT07347990 pour Microangiopathie thrombotique, Hematopoietic Stem Cell Transplantation (HSCT) est pas encore en recrutement. Consultez la vue en carte du Radar des Essais Cliniques et les outils de découverte par IA pour tous les détails, ou posez vos questions ici.
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Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy 30 Ouvert Survie globale

Pas encore en recrutement
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L'essai clinique NCT07347990 est une étude interventionnel pour Microangiopathie thrombotique, Hematopoietic Stem Cell Transplantation (HSCT). Son statut actuel est : pas encore en recrutement. Le recrutement est prévu pour commencer le 1 janvier 2026, avec un objectif de 30 participants. Dirigée par First Affiliated Hospital of Zhejiang University, l'étude devrait être terminée d'ici le 31 décembre 2029. Les données du site ClinicalTrials.gov ont été mises à jour pour la dernière fois le 16 janvier 2026.
Résumé succinct
The goal of this clinical trial is to evaluate the efficacy and safety of Iptacopan as a second-line treatment for high-risk hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA). Iptacopan is a selective oral small-molecule complement factor B inhibitor. It acts by inhibiting factor B, blocking the formation of C3 convertase, reducing C3b deposition, thereby suppressing C5 convertase...Afficher plus
Titre officiel

A Prospective, Multicenter, Single-Arm Study: Safety and Efficacy of Iptacopan in the Treatment of High-Risk Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (TA-TMA)

Pathologies
Microangiopathie thrombotiqueHematopoietic Stem Cell Transplantation (HSCT)
Publications
Articles scientifiques et travaux de recherche publiés sur cet essai clinique:
Autres identifiants de l'étude
  • IPTA-TMA-001
Numéro NCT
NCT07347990
Date de début (réel)
2026-01-01
Dernière mise à jour publiée
2026-01-16
Date de fin (estimée)
2029-12-31
Inscription (estimée)
30
Type d'étude
Interventionnel
PHASE
N/A
Statut
Pas encore en recrutement
Mots clés
hematopoietic stem cell transplantation
transplantation-associated thrombotic microangiopathy
Iptacopan
Objectif principal
Traitement
Méthode d'allocation
N/A
Modèle d'intervention
Groupe unique
Masquage
Aucun (ouvert)
Bras / Interventions
Groupe de participants/BrasIntervention/Traitement
ExpérimentalIptacopan group
This experimental arm includes patients diagnosed with transplantation-associated thrombotic microangiopathy (TA-TMA) who have failed first-line therapy; all participants will receive Iptacopan as second-line treatment.
Iptacopan
Iptacopan will be administered under the supervision of hospital staff during inpatient stays or self-managed by patients in an outpatient setting. The induction phase lasts 4 weeks at a dosage of 200 mg twice daily (BID). Starting from Day 29, patients will enter the maintenance phase at a dosage of 200 mg once daily (QD), continuing until treatment completion at Week 12.
Critère principal d'évaluation
Critères d'évaluationDescription de la mesurePériode
Six-month Overall Survival Rate Following TA-TMA Diagnosis
The primary endpoint is defined as the proportion of patients who remain alive at 6 months after the initial diagnosis of transplantation-associated thrombotic microangiopathy (TA-TMA). Survival status will be systematically assessed through follow-up visits, medical record review, or direct patient contact at the 6-month timepoint.
From the date of TA-TMA diagnosis until 6 months post-diagnosis.
Critère secondaire d'évaluation
Critères d'évaluationDescription de la mesurePériode
TA-TMA Complete Response Rate by Week 12 (Jodele Criteria)
Proportion of patients achieving complete response of TA-TMA according to Jodele criteria within 12 weeks of treatment initiation.
12 weeks from start of treatment.
TA-TMA Partial Response Rate by Week 12 (Jodele Criteria)
Proportion of patients achieving partial response of TA-TMA (defined as response between complete response and no response) within 12 weeks of treatment initiation.
12 weeks from start of treatment.
Overall Survival (OS)
Time from TA-TMA diagnosis to death from any cause.
Up to 24 months from diagnosis.
Non-Relapse Mortality (NRM)
Time from TA-TMA diagnosis to death not attributable to hematologic disease relapse or progression.
Up to 24 months from diagnosis.
Cumulative Incidence of Relapse (CIR)
Time from TA-TMA diagnosis to hematologic disease relapse or progression.
Up to 24 months from diagnosis.
Mean Hemoglobin Change from Baseline
Change in mean hemoglobin level (g/dL) from baseline to specified time points
Baseline to 12 weeks.
Exploratory Biomarker Analysis
Exploratory analysis of complement pathway biomarkers including C5b-9 (ng/mL) and Factor B (ng/mL) levels .
Baseline, 2 weeks, 4 weeks, 8 weeks, 12 weeks after treatment .
Failure-Free Survival (FFS)
Time (in days) from treatment initiation to first occurrence of TA-TMA response among responders.
Up to 12 weeks from treatment initiation.
Incidence of Acute and Chronic GVHD
Incidence of acute and chronic graft-versus-host disease.
Up to 24 months from treatment initiation.
Multiple Organ Dysfunction Syndrome (MODS) Involvement and Resolution
Assessment of MODS organ involvement and resolution status during treatment.
Baseline, 2 weeks, 4 weeks, 8 weeks, and 12 weeks after treatment; thereafter, every three months until study completion
Safety and Tolerability Assessment
Frequency, duration, and severity of adverse events monitored through physical examinations and laboratory assessments, including infections and secondary primary malignancies. Adverse events will be graded according to CTCAE v4.03.
From treatment initiation to 30 days after last dose.
Assistant à la participation
Critères d'éligibilité

Âges éligibles
Enfant, Adulte, Adulte âgé
Âge minimum
12 Years
Sexes éligibles
Tous
  1. Age ≥12 years at the time of ICF signature.
  2. Previous recipient of autologous or allogeneic HSCT.
  3. Persistent TA-TMA despite initial management of potential triggers (e.g., CNI/mTOR inhibitor reduction, infection or GVHD treatment), with TMA activity sustained for ≥72 hours post-intervention.
  4. TA-TMA diagnosis, confirmed ≤14 days prior to or during screening by either biopsy-proven microthrombi or ≥4 of the following:

(1) LDH > ULN (2) Proteinuria (rUPCR ≥1 mg/mg) (3) Hypertension (age-adjusted) (4) New-onset thrombocytopenia (platelet decrease ≥50%, count ≤50,000/mm³, or transfusion-refractory) (5) New-onset anemia or increased transfusion need (6) Microangiopathy on blood smear (schistocytes ≥1%) or biopsy (7) Elevated terminal complement complex (C5b-9) 5. High-risk TMA features (per 2023 consensus), meeting ≥1 criterion:

  1. LDH ≥2× ULN
  2. Elevated sC5b-9
  3. Proteinuria (rUPCR ≥1 mg/mg)
  4. Multi-organ dysfunction syndrome (MODS)
  5. Concurrent Grade II-IV acute GVHD
  6. Active systemic infection 6. Able to receive oral medication. 7. Failure of first-line therapy (e.g., CNI/mTOR inhibitor adjustment, plasma exchange, rituximab, defibrotide), excluding prior complement inhibitors.

8. Life expectancy >8 weeks. 9. Required vaccination against encapsulated bacteria (meningococcal, pneumococcal) per local guidelines, administered ≥2 weeks prior to first dose. If vaccination is delayed, antimicrobial prophylaxis is required.

10. For subjects unable to receive meningococcal vaccines, antibiotic prophylaxis must be continued throughout treatment and for 8 months post-last dose.

11. For subjects of reproductive potential: agreement to use effective contraception and, for females, a negative pregnancy test at screening.

12. Provision of signed informed consent and compliance with study procedures.

  1. Known familial or acquired ADAMTS13 deficiency (activity <5%).
  2. Known Shiga toxin-associated HUS (positive Shiga toxin assay or culture).
  3. Positive direct Coombs test with clinically significant immune-mediated hemolysis per investigator.
  4. Clinically overt disseminated intravascular coagulation (DIC) according to ISTH criteria.
  5. Bone marrow/graft failure.
  6. Known HIV infection (confirmed by testing within 6 months prior to screening).
  7. Active meningococcal disease.
  8. Septic shock requiring vasopressor support within 7 days prior to enrollment.
  9. Pregnant or breastfeeding.
  10. Any concurrent or prior medical condition unrelated to TA-TMA that, in the opinion of the investigator or sponsor, could increase risk or confound study outcomes (e.g., significant cardiac, pulmonary, renal, endocrine, or hepatic disease).
  11. All-cause respiratory failure requiring mechanical ventilation within 72 hours prior to enrollment.
  12. Acute/chronic heart failure with left ventricular ejection fraction ≤40%.
  13. Prior treatment with iptacopan, eculizumab, or other complement inhibitors within 60 days before first study dose.
  14. Use of any investigational agent within 30 days or 5 half-lives (whichever is longer) prior to screening.
  15. Recurrent primary malignancy or post-transplant lymphoproliferative disorder (PTLD).
First Affiliated Hospital of Zhejiang University logoFirst Affiliated Hospital of Zhejiang University
  • 🏥Ruijin Hospital
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Partie responsable de l'étude
Yanmin Zhao, Investigateur principal, Vice Director, Bone Marrow Transplantation Center, the First Affiliated Hospital, School of Medicine, Zhejiang University, First Affiliated Hospital of Zhejiang University
Contact central de l'étude
Contact: Fei Gao, Attending, MD, +86 19857035073, [email protected]
1 Centres de l'étude dans 1 pays

China

The First Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China, 310003, China
Contact, [email protected]
Contact, [email protected]