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Huntington DiseaseEarly Phase 1NCT06024265

The Safety and Tolerability Study With ER2001 Intravenous Injection 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 of ER2001 intravenous injection to evaluate safety, tolerability and pharmacokinetics of ascending single and multiple doses of intravenously administered ER2001 in patients with early manifest Huntington's Disease. Read more about this study

Who is it for?
Ages 25 to 55 years
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
10 participants
Where
1 siteChina
Study dates
4 Apr 2023to 4 Sep 2025
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 to 55 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • 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.
  • 25 Years to 55 Years. Gender is not limited.
  • Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4.
  • 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.
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  • 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.
  • 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.
  • 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.
  • 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: An Open-Label, Dose Escalation Early Phase 1 Study of ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease

This is an open label, dose escalation clinic trial of ER2001 intravenous injection to evaluate safety, tolerability and pharmacokinetics of ascending single and multiple doses of intravenously administered ER2001 in patients with early manifest Huntington's Disease. Furthermore, pharmacodynamics in particular target engagement, and early clinical signs of efficacy will be assessed. This study will evaluate increasing doses of ER2001 in sequential cohorts. ER2001 was escalated over 4 dose levels . The planned duration of this treatment is 14 weeks.

Read the detailed description

Participants will receive totally 8 intravenous injections of ER2001 on the first day of the study, after 2 weeks they will receive 6 injections at weekly intervals, followed by 1 injections at 6 weeks interval. The treatment period of 14 weeks will be followed by an observation phase of 84 days . A modified 3 plus 3 dose-escalation design will be employed with escalating rules set according to the incidence of dose-limiting toxicity (DLT). The first cohort of 1-6 participants will receive a low dose. After safety analysis the next cohort of 3-6 will received a higher dose. Planned doses for increase in subsequent cohorts are 0.04, 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

    Also called ER2001 intravenous injection solution

    The minimum initial dose is 0.04mg/kg, then escalate to 0.08mg/kg, 0.16mg/kg and 0.32mg/kg. The planned duration of the treatment is 14 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 3, 4, 5, 6, 7, 8, and 14.

Study groups in NCT06024265
Study groupRoleWhat they receive
ER2001 InjectionExperimentalThe minimum initial dose is 0.04mg/kg, then escalate to 0.08mg/kg, 0.16mg/kg and 0.32mg/kg. The planned duration of the treatment is 14 weeks, and ER2001 will be administrated intravenously at the first day of weeks 1, 3, 4, 5, 6, 7, 8, and 14.
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 6.5 months

Secondary outcomes (4)

  1. Peak Plasma Concentration (Cmax)

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 3.5 months

  2. Terminal half-life (t1/2)

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 3.5 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 3.5 months

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

    To assess the Pharmacokinetics (PK) of ER2001 injection.

    Measured: Approximately 3.5 months

Timeline

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

  1. First posted6 Sep 2023
  2. Study start4 Apr 2023
  3. Primary completion31 Jan 2025
  4. Study completion4 Sep 2025

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 four cohorts. The first cohort of 1-6 participants will receive an initial dose, followed by a safety review of the included participants. If the safety is confirmed and decision made to proceed to the next higher dose, the next cohort of 3-6 participants will receive a higher dose. The planned increasing doses are 0.04, 0.08, 0.16, and 0.32 mg/kg, respectively in subsequent.
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06024265
Sponsor's protocol ID
ER2001-001
Status last verified
Oct 2025
First submitted
25 May 2023
Last updated
19 Dec 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Zhang L, Wu T, Shan Y, Li G, Ni X, Chen X, Hu X, Lin L, Li Y, Guan Y, Gao J, Chen D, Zhang Y, Pei Z, Chen X. Therapeutic reversal of Huntington's disease by in vivo self-assembled siRNAs. Brain. 2021 Dec 16;144(11):3421-3435. doi: 10.1093/brain/awab354.

    PubMed 34918046

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 10 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 19 Dec 2025. 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 6.5 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 NCT06024265, which also lists 1 linked publication.

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 19 Dec 2025.

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