The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adult… (NCT07344519) | Clinical Trial Compass
Active — Not RecruitingEarly Phase 1
The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease.
China9 participantsStarted 2024-06-25
Plain-language 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）. 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.
Who can participate
Age range25 Years
SexALL
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Inclusion criteria
✓. 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.
✓. Other concomitant medications have been stable for 1 month prior to Screening.
✓. organ function measured prior to administration of study treatment.
Exclusion criteria
✕. 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.
What they're measuring
1
Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs)
✕. 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.
✕. 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.