Treating Congestive HF With hiPSC-CMs Through Endocardial Injection

NCT04982081 | Unknown Phase 1

• 1 other identifier: LTao
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Heart failure is the primary cause of morbidity and mortality worldwide. Currently drug treatments for heart failure manage the symptoms, but not restore the loss cardiomyocytes due to the very limited regenerative capability in the adult heart. Novel reparative therapies that replace the cardiomyocytes loss are highly demanded to restore the cardiac function. The main purposes of this explanatory study is to investigate the safety and efficacy of the catheter-based endocardial delivery of human iPSC-derived cardiomyocytes in patients with congestive heart failure.

Conditions

Cardiovascular Diseases; Congestive Heart Failure; Dilated Cardiomyopathy

Keywords

induced pluripotent stem cell; Congestive heart failure; Regenerative medicine; dilated cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• Incidence of major serious adverse events (SAEs)

  Incidence of SAEs is defined as the composite of: death, fatal myocardial infarction, stroke, tamponade, cardiac perforation, ventricular arrhythmias affecting hemodynamics (\> 15s), and tumorigenicity related to the hiPSC-CM .

  First month post-catheterization

Secondary Outcomes (9)

• Incidence of severe arrhythmia

  1-6 months post-catheterization

• Incidence of newly formed tumors

  Baseline, 1,3,6 and 12 months post-catheterization

• Changes in penal reactive antibodies (PRA)

  Baseline, 1, 3 and 6 months post-catheterization

• Changes in donor specific antibodies (DSA)

  Baseline, 1, 3 and 6 months post-catheterization

• Overall Left Ventricular systolic performance as assessed by MRI

  Baseline, 1, 3, 6 and 12 months post-catheterization

Study Arms (2)

hiPSC-CM therapy low dosage

EXPERIMENTAL

Biological: hiPSC-CM therapy

hiPSC-CM therapy high dosage

EXPERIMENTAL

Biological: hiPSC-CM therapy

Interventions

hiPSC-CM therapy BIOLOGICAL

20 patients with congestive heart failure who met the inclusion and exclusion criteria will be recruited. After being fully informed and signed the informed consent, the patients will be randomly divided into two dosage groups: 100 million cells (10 patients) and 400 million cells (10 patients). Human iPSC-derived cardiomyocytes will be injected into the myocardium through a transcatheter endocardial injection system.

hiPSC-CM therapy high dosage; hiPSC-CM therapy low dosage

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients aged 18-75 years (including 18 and 75).
• Signed the informed consent.
• Patients with congestive heart failure who have received regular treatment for heart failure.
• New York Heart Association (NYHA) Class III or IV despite optimal standard of care
• Left Ventricular Ejection Fraction (LVEF)\<40% as assessed by echocardiography ( measure in the 3 months of recruit is included, excluding the measured values within 1 month of myocardial infarction ）
• The thickness of left ventricular ≥8mm
• Female patient who is not pregnant or nursing during the clinical trial

You may not qualify if:

• PRA ≥ 20% or DSA positive.
• Patients received treatments such as pacemakers, ICD or CRT device.
• Patient with severe valvular disease or presence of a mechanical valve replacement, such as PCI implantation, or patients requiring simultaneous radiofrequency ablation of atrial fibrillation.
• Patient with any therapeutic traumatic heart surgery within 30 days.
• Hemodynamic instability or cardiogenic shock.
• Right heart failure.
• Restrictive cardiomyopathy such as amyloidosis, sarcoidosis or hematochromia, constrictive pericarditis.
• Myocardial infarction occurred within 30 days or stroke occurred within 60 days before enrollment.
• Thickness at left ventricular free wall infarction \< 6 mm.
• Severe ventricular arrhythmias (persistent ventricular tachycardia or other conditions that the investigator considers necessary to exclude).
• Baseline glomerular filtration rate \< 30 ml/min / 1.73 m2.
• Abnormal liver function: ALT or AST 3 times higher than the normal value.
• Have a hematologic abnormality as evidenced by hematocrit \< 25%, white blood cells count \<2,500/ul or platelet count \<100000 / ul.
• Known allergies to penicillin, streptomycin or radiocontrast agent.
• Abnormal coagulation function, INR \> 1.3, which cannot be corrected.

Sponsors & Collaborators

• Help Therapeutics: lead
• Xijing Hospital: collaborator

Study Sites (1)

Help Therapeutics

Nanjing, Jiangsu, 210000, China

RECRUITING

MeSH Terms

Conditions

Cardiovascular Diseases; Heart Failure; Cardiomyopathy, Dilated

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiomegaly; Cardiomyopathies; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Ling Tao, MD, PhD

  Xijing Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jiaxian Wang, MD, PhD

CONTACT

+86-18565616060; wangjx@helptx.com.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2021
• First Posted: July 29, 2021
• Study Start: September 21, 2021
• Primary Completion: December 30, 2022
• Study Completion: July 31, 2023
• Last Updated: September 23, 2022

Record last verified: 2022-09

Locations

CN (1)