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Huntington DiseaseEarly Phase 1NCT07344519

The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease.

A Early Phase 1 study testing ER2001 injection in people with Huntington Disease, run by ExoRNA Bioscience at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is an open label, dose escalation clinic trial to evaluate safety, tolerability and pharmacokinetics with ER2001 Intravenous Injection repetitive treatment in Huntington's Disease patients who Completed Study ER2001-001（NCT06024265）. Read more about this study

Who is it for?
Ages 25 years and older
Conditions the study is for
Huntington Disease
What is being tested?
ER2001 injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
1 siteChina
Study dates
25 Jun 2024to 31 Jan 2026
Phase
Early Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 25 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Huntington's Disease patients who Completed Study ER2001-001（NCT06024265）.
  • 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.
  • HTT gene expansion testing with the presence of ≥40 CAG repeats.
  • Ability to undergo and tolerate MRI scans.
  • Ability to undergo and tolerate lumbar puncture.
  • All HD medications given for motor, behavioral, and cognitive symptoms have been stable for 3 months prior to Screening.
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  • Other concomitant medications have been stable for 1 month prior to Screening.
  • organ function measured prior to administration of study treatment.
  • 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.

Who cannot join

  • 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) > 480 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.
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  • 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.
  • 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.
  • Malignancy within 5 years of screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
  • Concurrent or planned concurrent participation in any interventional clinical study, including explicit pharmacological and non- pharmacological interventions. Observational studies are acceptable.
  • Any serious medical condition or clinically significant laboratory, vital signs, or abnormalities at screening that, in the Investigator's judgment, precludes the patient's safe participation in and completion of the study.

Trial locations and contacts

1 site in 1 country.

China1 site

  • First Affiliated Hospital of Guangzhou Medical University

    Guangzhou, Guangdong, 510080

Other trials in China

About this study

Official title: Open-Label, Dose Escalation Early Phase 1 Study of ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease.

This is an open label, dose escalation clinic trial to evaluate safety, tolerability and pharmacokinetics with ER2001 Intravenous Injection repetitive treatment in Huntington's Disease patients who Completed Study ER2001-001（NCT06024265）. Furthermore, pharmacodynamics in particular target engagement, and clinical signs of efficacy will be assessed. This study will evaluate increasing doses of ER2001 in sequential cohorts. ER2001 was escalated over 3 dose levels . The planned duration of this additional treatment is 6 weeks for one course.

Read the detailed description

The study 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 (12 weeks). Participants will receive 6 injections at the same dose level used in the NCT06024265 study, once a week over a period of 6 consecutive weeks. Planned doses for increase in subsequent cohorts are 0.08, 0.16, and 0.32 mg/kg. Decision to proceed to the next higher dose cohort will be based upon a safety review of the included participants. Treatment will continue until intolerable toxicity or as per patient preference.

Being tested
ER2001 injection
Medical subject headings
Huntington Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ER2001 injectionDrug

    The minimum initial dose is 0.08mg/kg, then escalate to 0.16mg/kg, and 0.32mg/kg. 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.

Study groups in NCT07344519
Study groupRoleWhat they receive
ER2001 InjectionExperimentalThe minimum initial dose is 0.08mg/kg, then escalate to 0.16mg/kg, and 0.32mg/kg. 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.
Receives: Drug: ER2001 injection

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    To evaluate the safety and tolerability of ER2001 injection.

    Measured: Approximately 4.5 months

Secondary outcomes (4)

  1. Peak Plasma Concentration (Cmax)

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 4.5 months

  2. Terminal half-life (t1/2)

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 4.5 months

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

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 4.5 months

  4. Maximum concentration (Cmax) in cerebrospinal fluid (CSF)

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 4.5 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Jan 2026
  2. Study start25 Jun 2024
  3. Primary completion1 Jun 2025
  4. Study completion31 Jan 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
As designed, the single group will be divided into 3 cohorts. The planned increasing doses are 0.08, 0.16, and 0.32 mg/kg, respectively in subsequent. If the safety is confirmed and decision made to proceed to the next higher dose, the next cohort of 3 participants will receive a higher dose.
Masking
None (open label)
Enrolment
8 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07344519
Sponsor's protocol ID
ER2001-002
Status last verified
May 2026
First submitted
7 Jan 2026
Last updated
22 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing ER2001 injection in people with Huntington Disease, run by ExoRNA Bioscience at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs) (time frame: Approximately 4.5 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07344519.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.

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