Huntington DiseasePhase 1/2NCT07339514
A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.
A Phase 1/2 study testing ER2001 intravenous injection in people with Huntington Disease, run by ExoRNA Bioscience at 3 sites in China.
Trial 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. 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 intravenous injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 3 sitesChina
- Study dates
- 11 Feb 2025to 30 May 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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.
- 2.25 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.
- 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.
- 8.Other concomitant medications have been stable for 1 month prior to Screening.
- 9.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.
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) > 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.
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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
3 sites in 1 country.
China3 sites
West China Hospital of Sichuan University
Chengdu, Sichuan, 610041
First Affiliated Hospital of Guangzhou Medical University
Guangzhou, Guangdong, 510080
First Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guangdong, 510080
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Phase I Clinical Study of ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease.
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).
Read the detailed description
Two dose levels will be included in the dose escalation phase, and subjects will be in cohorts (Cohort 1, and Cohort 2) in sequence to receive the investigational drug. Either 3 or 6 subjects will be enrolled in each cohort. Dose escalation from the current cohort to the next cohort will be determined through joint review by the sponsor and the IDMC, typically based on the number of DLT(dose-limiting toxicity)s observed in subjects. If one DLT occurs in a cohort, additional 3 subjects will be enrolled in that cohort, with a total of 6 subjects. If one DLT occurs in the low-dose cohort while the administration is ongoing in the high-dose cohort, recruitment of subjects in the high-dose cohort should be held until all enrolled subjects have completed their dosing regimen and a safety assessment of at least 7 days after completion of the treatment. In addition, the IDMC will hold a meeting to decide whether further actions are necessary, e.g., dose interruption or reduction for subjects in the high-dose cohort. If two DLTs occur in a cohort, the dose will be discontinued in that cohort and any active higher dose cohorts. No more than one subject within the same cohort should initiate the treatment on the same day.
Based on review of safety, tolerability, PK and exploratory PD results in the dose escalation phase, dose expansion will be performed in 1 or 2 cohort(s) at the recommended dose levels. For the dose expansion phase, 9 subjects (including 3 subjects in the placebo control group) will be enrolled for each dose level cohort.
- Conditions
- Huntington Disease
- Being tested
- ER2001 intravenous injection, Placebo
- 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 intravenous injectionDrug
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.
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs).
To evaluate the safety and tolerability of ER2001 injection or placebo.
Measured: Approximately 7.5 months
Secondary outcomes (4)
Peak Plasma Concentration (Cmax)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Measured: Approximately 7.5 months
Terminal half-life (t1/2)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Measured: Approximately 7.5 months
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 7.5 months
Maximum concentration (Cmax) in cerebrospinal fluid (CSF)
To assess the Pharmacokinetics (PK) of ER2001 injection.
Measured: Approximately 7.5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2026
- Study start11 Feb 2025
- Primary completion1 Apr 2026 (estimated)
- Study completion30 May 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Biological Product.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ExoRNA Bioscience
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- jinsheng zeng, Ph.D, Study chair, First Affiliated Hospital of Sun Yat-sen Universitypingyi xu, Ph.D, Principal investigator, First Affiliated Hospital of Guangzhou Medical Universityhuifang shang, Ph.D, Principal investigator, West China Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07339514
- Sponsor's protocol ID
- ER2001-102
- Other IDs
- CTR20243121
- Status last verified
- Feb 2025
- First submitted
- 5 Jan 2026
- Last updated
- 14 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Phase I Clinical Study of ER2001 Intravenous Injection in Adults With Early Manifest Huntington's Disease.
- ClinicalTrials.gov ID
- NCT07339514
- Acronym / sponsor ID
- ER2001-102
- Conditions
- Huntington Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- ExoRNA BioscienceIndustry (pharma, biotech, devices)
- Study start
- 11 Feb 2025 (actual)
- Primary completion
- 1 Apr 2026 (estimated)
- Study completion
- 30 May 2026 (estimated)
- First posted
- 14 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Two dose levels will be included in the dose escalation phase, and subjects will be in cohorts (Cohort 1, and Cohort 2) in sequence to receive the investigational drug. Either 3 or 6 subjects will be enrolled in each cohort. Dose escalation from the current cohort to the next cohort will be determined through joint review by the sponsor and the IDMC, typically based on the number of DLT(dose-limiting toxicity)s observed in subjects. If one DLT occurs in a cohort, additional 3 subjects will be enrolled in that cohort, with a total of 6 subjects. If one DLT occurs in the low-dose cohort while the administration is ongoing in the high-dose cohort, recruitment of subjects in the high-dose cohort should be held until all enrolled subjects have completed their dosing regimen and a safety assessment of at least 7 days after completion of the treatment. In addition, the IDMC will hold a meeting to decide whether further actions are necessary, e.g., dose interruption or reduction for subjects in the high-dose cohort. If two DLTs occur in a cohort, the dose will be discontinued in that cohort and any active higher dose cohorts. No more than one subject within the same cohort should initiate the treatment on the same day.
Based on review of safety, tolerability, PK and exploratory PD results in the dose escalation phase, dose expansion will be performed in 1 or 2 cohort(s) at the recommended dose levels. For the dose expansion phase, 9 subjects (including 3 subjects in the placebo control group) will be enrolled for each dose level cohort.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- Model description
- Biological Product.
| Arm | Type | Receives |
|---|---|---|
| 1.ER2001 - 0.08mg/kg | Active comparator | Drug: ER2001 intravenous injectionThe 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. |
| 2.ER2001 - 0.32mg/kg | Active comparator | Drug: ER2001 intravenous injectionThe 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. |
| 3.Placebo Intravenous Injection | Placebo comparator | Drug: PlaceboThe 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. |
Who may qualify
- Ages
- 25 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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.
- 2.25 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.
- 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.
- 8.Other concomitant medications have been stable for 1 month prior to Screening.
- 9.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.
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ER2001 intravenous injectionThe 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. | Drug | 1.ER2001 - 0.08mg/kg, 2.ER2001 - 0.32mg/kg |
| PlaceboPlacebo 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. | Drug | 3.Placebo Intravenous Injection |
Primary outcome
Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs).
Time frame: Approximately 7.5 months
To evaluate the safety and tolerability of ER2001 injection or placebo.
4 secondary outcomes
Peak Plasma Concentration (Cmax)
Time frame: Approximately 7.5 months
Terminal half-life (t1/2)
Time frame: Approximately 7.5 months
Area under the plasma concentration versus time curve from time 0 to the last quantifiable concentration (AUC0-t).
Time frame: Approximately 7.5 months
Maximum concentration (Cmax) in cerebrospinal fluid (CSF)
Time frame: Approximately 7.5 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
jinsheng zeng, Ph.D study chair
First Affiliated Hospital of Sun Yat-sen University
pingyi xu, Ph.D principal investigator
First Affiliated Hospital of Guangzhou Medical University
huifang shang, Ph.D principal investigator
West China Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ER2001 intravenous injection in people with Huntington Disease, run by ExoRNA Bioscience at 3 sites 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 5 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: under way, not looking for participants, as of the sponsor's last update on 14 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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 7.5 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 Jan 2026.