Phase 2
N=78
GV1001 Subcutaneous(SC) for the Treatment of Progressive Supranuclear Palsy (PSP)
Progressive Supranuclear Palsy
Bottom Line
View on ClinicalTrials.gov: NCT05819658 ↗Enrolled (actual)
78
Serious AEs
13.2%
Results posted
Feb 2026
Primary outcome: Primary: Change From the Baseline in the Total Score of PSP-rating Scale — 4.10; 2.14; 6.46 scores on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- GV1001 Placebo (Drug); GV1001 0.56mg (Drug); GV1001 1.12mg (Drug)
- Age
- Adult, Older Adult · 41+ yrs
- Sex
- All
- Sponsor
- GemVax & Kael
- Primary completion
- Oct 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From the Baseline in the Total Score of PSP-rating Scale |
1.42; -0.47; 2.75 | — |
| SECONDARY Change From the Baseline in the Total Score of PSP-rating Scale |
1.42; -0.47; 2.75 | — |
| SECONDARY Change From the Baseline in the Montreal Cognitive Assessment - Korea (MoCAK) |
0.29; 0.35; -1.46 | — |
| SECONDARY Change From the Baseline in the Montreal Cognitive Assessment - Korea (MoCAK) |
0.29; 0.35; -1.46 | — |
| SECONDARY Change From the Baseline in the Korean Frontal Assessment Battery (K-FAB) |
-0.20; -0.89; -1.42 | — |
| SECONDARY Change From the Baseline in the Korean Frontal Assessment Battery (K-FAB) |
-0.20; -0.89; -1.42 | — |
| SECONDARY Change From the Baseline in the England & Schwab Activity of Daily Living (ES ADL) Scale |
-9.45; -8.35; -17.12 | — |
| SECONDARY Change From the Baseline in the England & Schwab Activity of Daily Living (ES ADL) Scale |
-9.45; -8.35; -17.12 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (History) |
0.42; 0.90; 1.36 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (History) |
0.42; 0.90; 1.36 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Mentation) |
0.26; -0.07; 0.66 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Mentation) |
0.26; -0.07; 0.66 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Bulbar) |
0.49; 0.04; 0.68 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Bulbar) |
0.49; 0.04; 0.68 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Ocular Motor) |
1.36; 0.25; 1.30 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Ocular Motor) |
1.36; 0.25; 1.30 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Limb Motor) |
0.45; -0.07; 0.51 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Limb Motor) |
0.45; -0.07; 0.51 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Midline/Gait) |
0.58; 1.28; 1.79 | — |
| SECONDARY Change From the Baseline in the Score of Each Domain of the PSP-rating Scale (Midline/Gait) |
0.58; 1.28; 1.79 | — |
| SECONDARY Change From the Baseline in the Score of Each Item of the PSP-rating Scale(Item 12_Dysarthria) |
0.44; 0.01; 0.44 | — |
| SECONDARY Change From the Baseline in the Score of Each Item of the PSP-rating Scale(Item 12_Dysarthria) |
0.44; 0.01; 0.44 | — |
Summary
The study will be conducted by the Sponsor to evaluate the efficacy and safety of GV1001 (0.56 mg and 1.12 mg) administered subcutaneously as a treatment for Progressive Supranuclear Palsy, (PSP). In 75 patients diagnosed with PSPR Richardson(PSP-RS) or PSP-Parkinsonism (PSP-P) at five hospitals in Korea, subcutaneous administration of GV1001 0.56 or 1.12 mg/day will be conducted with multicenter, randomized, double-blind, placebo-controlled, parallel design, prospective phase 2a.
Eligibility Criteria
Inclusion Criteria
- Patients aged ≥41 years to ≤ 85 years.
- Clinical diagnosis of probable progressive supranuclear palsy (PSP).
- Patient is on a stable therapy for a neurological drug for at least 1 month prior to screening visit.
- Patients who are able to walk 3 meters or more independently or with assistive devices.
- Score 15 points ≥ on the Korean Mini-Mental Status Exam (K-MMSE) at the screening visit.
- Have reliable caregiver to accompany participant to all study visits.
- Patients and/or their representatives who have voluntarily provided a written consent for participation in this clinical study.
Exclusion Criteria
- Patients who have Presence of structural lesions or Suspected concurrent onset of central nervous system diseases based on the CT/MRI scan results and neurological examinations performed within 12 months of screening or at screening.
- Patients with a history of known or suspected seizures.
- Patients with a recent unexplained loss of consciousness within 3 months prior to screening or a history of significant head trauma with loss of consciousness.
- Patients with acute or unstable cardiovascular disease, uncontrolled hypertension, uncontrolled diabetes, or any other medical condition that can interfere with completing the clinical study.
- Patients with hypersensitivity reactions to the ingredients of the investigational product.
- Patients with a history of cancer within 5 years prior to screening.
- Patients with abnormal renal function.
- Patients with severe liver function abnormalities.
- Patients weighing ≤35 kg.
- Among the female subjects who does not agree to use proper contraception.
- Pregnant or breastfeeding women.
- Patients who participated in another clinical study within 4 weeks prior to screening and were administered investigational products or were applied investigational medical devices.
- Patients who were administered the study drug (GV1001) of this clinical study within 12 months prior to screening.
- Patients who participated in a clinical study for progressive supranuclear palsy within 6 months prior to screening.
- Other patients judged by the investigator as ineligible to participate in this clinical study.
Data sourced from ClinicalTrials.gov (NCT05819658). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.