A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Dis… (NCT07339514) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.
China27 participantsStarted 2025-02-11
Plain-language 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).
Who can participate
Age range25 Years – 55 Years
SexALL
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Inclusion criteria
✓. 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. 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.
Exclusion criteria
What they're measuring
1
Incidence and Severity of adverse events (AEs) and serious adverse events (SAEs).
. 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.