A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.

NCT07339514 | Active Not Recruiting Phase 1 DMC

• 2 other identifiers: ER2001-102CTR20243121
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 3

Brief Summary

This is a dose escalation and expansion clinical study to evaluate the safety, tolerability, PK profile and preliminary efficacy of ER2001 Injection vs. placebo in subjects with definitive diagnosis of early manifest HD. The study consists of a dose escalation phase (Part A, an open-label without placebo, which will be carried out firstly) and a dose expansion phase (Part B,randomized, blinded, placebo-controlled), both of which include a screening period (4 week prior to the first administration), a treatment period (for 6 consecutive weeks, once a week \[QW\] for 6 weeks), and a safety follow-up period (24 weeks).

Conditions

Huntington Disease

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs).

  To evaluate the safety and tolerability of ER2001 injection or placebo.

  Approximately 7.5 months

Secondary Outcomes (4)

• Peak Plasma Concentration (Cmax)

  Approximately 7.5 months

• Terminal half-life (t1/2)

  Approximately 7.5 months

• Area under the plasma concentration versus time curve from time 0 to the last quantifiable concentration (AUC0-t).

  Approximately 7.5 months

• Maximum concentration (Cmax) in cerebrospinal fluid (CSF)

  Approximately 7.5 months

Other Outcomes (5)

• Change from baseline in the concentration of mutant huntingtin (mHTT) protein in cerebrospinal fluid (CSF)

  Approximately 7.5 months

• Immunogenicity

  Approximately 7.5 months

• change in multicytokine levels from baseline

  Approximately 7.5 months

Study Arms (3)

1.ER2001 - 0.08mg/kg

ACTIVE COMPARATOR

The planned duration of the treatment is 6 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

Drug: ER2001 intravenous injection

2.ER2001 - 0.32mg/kg

ACTIVE COMPARATOR

The planned duration of the treatment is 6 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

Drug: ER2001 intravenous injection

3.Placebo Intravenous Injection

PLACEBO COMPARATOR

The planned duration of the treatment is 6 weeks, and Placebo will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

Drug: Placebo

Interventions

Placebo DRUG

Placebo Injection, The planned duration of the treatment is 6 weeks, and Placebo will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

3.Placebo Intravenous Injection

ER2001 intravenous injection DRUG

The drug dose is 0.08mg/kg, or 0.32mg/kg. The planned duration of each patient's treatment is 6 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 2, 3, 4, 5, and 6.

1.ER2001 - 0.08mg/kg; 2.ER2001 - 0.32mg/kg

Eligibility Criteria

• Age: 25 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Patient has documented ability to understand the written study informed consent forms (ICFs) at the time of screening and has provided signed written informed consent prior to any study procedures.
• Years to 55 Years. Gender is not limited. 3.Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 10 to 13 and a diagnostic classification level (DCL) of 4.
• HTT gene expansion testing with the presence of ≥40 CAG repeats. 5.Ability to undergo and tolerate MRI scans. 6.Ability to undergo and tolerate lumbar puncture. 7.All HD medications given for motor, behavioral, and cognitive symptoms have been stable for 3 months prior to Screening.
• Other concomitant medications have been stable for 1 month prior to Screening.
• organ function measured prior to administration of study treatment. 10.Postmenopausal or evidence of non-childbearing status for women of childbearing potential. Male patients must use a condom during treatment and for 6 months after the last dose of ER2001 when having sexual intercourse with a pregnant woman or with a woman of childbearing potential. Female partners of male patients should also use a highly effective form of contraception if they are of childbearing potential.

You may not qualify if:

• History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening.
• Current active psychosis, confusional state, or violent behavior.
• Bleeding tendency or history of coagulation disorder; As long as the investigator confirms that there is no evidence of bleeding tendency or coagulation dysfunction at present.
• ECG with corrected QT interval (QTc) \> 450 ms and/or indication of uncontrolled cardiac conditions, as judged by the investigator (e.g. unstable ischemia, uncontrolled ventricular arrhythmia, recent (within 3 months) myocardial infarction,congestive heart failure, electrolyte disturbances, etc.)
• Patients with HIV, Treponema pallidum, Hepatitis B, or Hepatitis C infection.
• Need to take antiretroviral drugs, including antiretroviral drugs as preventive treatment.
• Current or recurrent disease, infection, or other significant concurrent medical condition or medications that could confound clinical and laboratory evaluations or could affect a subject's safety or their ability to undergo the neurosurgical procedure or comply with the procedures and study visit schedule.
• Clinical diagnosis of chronic migraines.
• Presence of an implanted deep brain stimulation device, ventriculoperitoneal or other CSF shunt, or other implanted catheter.
• Preexisting structural brain lesions (such as tumor, arteriovenous malformation) as assessed by a centrally read MRI scan during the screening period.
• Any history of gene therapy, RNA or DNA investigational agents, such as antisense oligonucleotides (ASO), cell transplantation or any other experimental brain surgery.
• Treatment with investigational therapy within 4 weeks prior to screening or 5 drug elimination half-lives of investigational therapy, whichever is longer.
• Unable or unsafe to perform lumbar puncture on the patient.
• In the Investigator's judgment, that Parkinson's disease, multiple system atrophy and other dystonia diseases may be combined.
• Patients who are hypersensitive to any ingredients in the formulation of ER2001.

Sponsors & Collaborators

• ExoRNA Bioscience: lead

Study Sites (3)

First Affiliated Hospital of Guangzhou Medical University

Guangzhou, Guangdong, 510080, China

Location

First Affiliated Hospital of Sun Yat-sen University

Guangzhou, Guangdong, 510080, China

Location

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Officials

• jinsheng zeng, Ph.D

  First Affiliated Hospital of Sun Yat-sen University

  STUDY CHAIR

• pingyi xu, Ph.D

  First Affiliated Hospital of Guangzhou Medical University

  PRINCIPAL INVESTIGATOR

• huifang shang, Ph.D

  West China Hospital

  PRINCIPAL INVESTIGATOR

Study Design

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

Model Details: Biological Product.

Study Record Dates

• First Submitted: January 5, 2026
• First Posted: January 14, 2026
• Study Start: February 11, 2025
• Primary Completion: April 1, 2026
• Study Completion (Estimated): May 30, 2026
• Last Updated: January 14, 2026

Record last verified: 2025-02

Locations

CN (3)