A First-in-Human Study to Evaluate the Safety and Efficacy of Left Ventricular Assist Device Combined With Exosome Therapy for Patients With Heart Failure（RESTORATION）

NCT07883317 | Not Yet Recruiting Early Phase 1 DMC

• 1 other identifier: RESTORATION
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

Brief Summary

A single-center, randomized, double-blind, placebo-controlled, dose-escalation clinical study to evaluate the safety and tolerability of intravenous infusion of different doses of the investigational exosome preparation in heart failure subjects receiving left ventricular assist device therapy. Subjects will receive exosome or placebo once daily for 3 consecutive days, in addition to optimal medical therapy (OMT). The drug category, dosage and administration of OMT will be recorded as concomitant medications and entered into the Case Report Form (CRF). The OMT regimen (including drug type, dosage, route of administration, treatment duration, etc.) will be documented throughout the study. During the study, subjects will undergo efficacy and safety assessments and/or sample collection. After completion of treatment, follow-up visits will be conducted at Day 28, 6 months and 12 months.

Conditions

Heart Failure

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-related serious adverse events within Day 28 after administration of the investigational exosome preparation.

  within Day 28 after administration of the investigational exosome preparation

Secondary Outcomes (15)

• Incidence of all adverse events (AEs) and serious adverse events (SAEs) within Day 28 after administration of the investigational exosome preparation.

  within Day 28 after administration of the investigational exosome preparation.

• Incidence and severity of infusion-related reactions within 24 hours after administration of the investigational exosome preparation, including fever, chills, hypotension, hypersensitivity reactions, and cytokine release syndrome (CRS).

  within 24 hours after administration of the investigational exosome preparation

• Occurrence of clinically significant electrocardiographic abnormalities before and after administration of the investigational exosome preparation

  Baseline (prior to study drug infusion), up to Day 28 post-administration

• Occurrence of clinically significant echocardiographic abnormalities before and after administration of the investigational exosome preparation

  Baseline (prior to study drug infusion), up to Day 28 post-administration

• Laboratory abnormalities occurring before and after administration of the investigational exosome preparation

  Baseline (prior to study drug infusion), up to Day 28 post-administration

Study Arms (2)

Intervention group

EXPERIMENTAL

Biological: Exosome Preparation

Control group

PLACEBO COMPARATOR

Other: Placebo

Interventions

Exosome Preparation BIOLOGICAL

Subjects will receive exosome preparation once daily for 3 consecutive days

Intervention group

Placebo OTHER

Subjects will receive placebo once daily for 3 consecutive days

Control group

Eligibility Criteria

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

You may qualify if:

• Subject or legal representative has signed the Informed Consent Form (ICF).
• Age ≥ 18 years.
• Body Surface Area (BSA) ≥ 1.2 m².
• NYHA Class III B or NYHA Class IV.
• Left ventricular ejection fraction (LVEF) ≤ 25%.
• a) Inotrope-dependent; OR b) Cardiac Index (CI) \< 2.2 L/min/m² in the absence of inotropes, and the subject must also meet one of the following:
• Receiving optimal medical management per current heart failure guidelines for at least 45 days out of the preceding 60 days, with evidence of treatment failure;
• Advanced heart failure for at least 14 days AND dependent on intra-aortic balloon pump (IABP) support for at least 7 days.
• Females of childbearing potential must agree to use adequate contraception throughout the study.

You may not qualify if:

• Heart failure caused by thyroid disease, hypertrophic obstructive cardiomyopathy, pericardial disease, amyloidosis or restrictive cardiomyopathy.
• History of any solid organ transplantation (including heart transplantation).
• Moderate to severe aortic regurgitation that was not corrected concomitantly at the time of LVAD implantation.
• Irreversible severe right heart failure that cannot be corrected by medications or right ventricular assist device.
• Pre-existing technical barriers at the time of LVAD implantation conferring an abnormally high surgical risk as judged by the heart team.
• Planned biventricular assist device (Bi-VAD) support prior to LVAD implantation.
• Presence of an aortic mechanical valve that was not replaced with a bioprosthesis or sutured closed during LVAD implantation.
• Presence of any risk factor indicating severe end-organ dysfunction or failure:
• INR ≥2.0 not attributable to anticoagulation therapy
• Total bilirubin\>43 μmol/L (2.5 mg/dL), shock liver, or biopsy-proven liver cirrhosis
• History of severe chronic obstructive pulmonary disease (COPD): FEV1/FVC\<0.7 and FEV1\<50% of predicted value
• Fixed pulmonary hypertension with latest pulmonary vascular resistance (PVR) ≥8 Wood units and no response to pharmacotherapy
• History of stroke within 90 days prior to enrollment; or cerebrovascular disease with severe (\>80%) uncorrected carotid artery stenosis
• Serum creatinine ≥221 μmol/L (2.5 mg/dL) or requirement for chronic renal replacement therapy
• Severe peripheral vascular disease (PVD) with rest pain or limb ulceration

Sponsors & Collaborators

• Yining Yang: lead

MeSH Terms

Conditions

Heart Failure

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Study Officials

• Yining Yang, MD, PhD

  People's Hospital of Xinjiang Uygur Autonomous Region

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jing Tao, MD, PhD

CONTACT

8618935903292; taojing_xj@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: President

Study Record Dates

• First Submitted: October 2, 2026
• First Posted: October 20, 2026
• Study Start (Estimated): January 15, 2027
• Primary Completion (Estimated): February 15, 2028
• Study Completion (Estimated): January 15, 2029
• Last Updated: October 20, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP