Evaluate the Efficacy and Safety of GV1001 in Patients With Benign Prostatic Hyperplasia (BPH)

A Randomized, Active-Controlled, Double-Blind, Parallel Design, Multi-Center, Phase III Clinical Trial to Evaluate the Efficacy and Safety of GV1001 in Patients With Benign Prostatic Hyperplasia (BPH)

The current study is being conducted by the Sponsor to evaluate the efficacy and safety of GV1001 (0.56 mg and 1.12 mg) administered as a treatment for Benign prostate hyperplasia(BPH). The investigational drug, GV1001, was first developed as a cancer vaccine for use as active immunotherapy of cancer forms expressing telomerase (eg, pancreatic cancer, prostate cancer, etc.). Subsequently, it was found that GV1001 showed efficacy in alleviating BPH symptoms during in vivo studies by reducing the size of the prostate gland. Based on the result, the effectiveness of GV1001 as a treatment for BPH has been assessed in experimental animals that are designed to develop BPH.

It is considered that GV1001 acts to alleviate BPH and the results obtained from previous phase II study indicate that GV1001 may provide potential beneficial effects in BPH patients.

So this study is to verify the efficacy and safety of GV1001 on BPH population, large-scale clinical study than phase II.

This was a multi-center, randomized, Double-blind, Active-controlled, parallel design, Phase 3 study in patients with BPH. The study consisted of a Screening period, a 4-weeks Run-in/Washout period, a 24-week Treatment period, an Evaluation and Close-out Visit at Week 24.

There are a total of 3 groups in this study, which contained 2 study groups (GV1001) and 1 placebo group (0.9% normal saline). Approximately 417 patients are planned to be randomly assigned into the study in a 1:1:1 ratio. All patients are randomized into 1 of 3 treatment groups to ensure completion of patients.

The Screening period have a time period of 4 weeks before the beginning of Run-in/Washout period. Eligible patients entered into a 4-week Run-in/Washout period and receive placebo treatment, which will be completed before randomization on Week 0. All randomized patients will receive the investigational drug or a placebo via intradermal injection 12 times with a 2-week interval at Weeks 0, 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22 and 24. Efficacy evaluation will be conducted at Weeks 4, 8, 12, 16, 20 and 24, and safety evaluation will be conducted throughout the 24-weeks period.

Inclusion Criteria:

1. A male at 50 years of age and older

2. Clinical signs and symptoms of benign prostatic hyperplasia

   1. A volume of prostate gland (TRUS) > 30 cc
   2. Moderate to severe lower urinary tract symptoms with IPSS ≥ 13
   3. 5-15 mL/sec of maximum flow rate (Qmax) measured when urine volume was at least 125 mL

3. PSA level < 10 ng/mL (however, if 4 ng/mL < PSA < 10 ng/mL, a person with a biopsy result, confirming that he does not have prostate cancer)

4. Residual urine volume ≤ 200 Ml

5. Consent not to participate in other clinical trials as a subject during this clinical trial period.

6. Consent of patient and patient's partner a. Patient

   • Consent to avoid pregnancy by using condoms for 90 days after the end of study participation period and treatment. (Not applied if the patient had vasectomy.) b. Patient's partner (Consent should be obtained before visit 4, when necessary.)
   • Consent to avoid pregnancy by using contraceptive devices or oral contraceptives during the patient's participation in clinical trial and for 90 days after the end of treatment, except if the partner reaches menopause or is surgically sterilized.

Exclusion Criteria:

1. Hypersensitivity reactions to ingredients of this drug.
2. Taking drugs that affect the results of clinical trials. ex) 5-alpha reductase inhibitors, drugs similar to LHRH, alpha blockers, alpha-beta blockers, anticholinergics, antidiuretic hormones, diuretics, PDE-5 inhibitors, beta-3 adrenoceptor antagonists, steroids, immune suppressants, saw palmetto, etc.
3. Taking drugs of an unapproved study drug in the past or the study drug for this clinical trial
4. Diagnosis with prostate cancer in the past or at present
5. Diagnosis by an investigator to have an influence to an evaluation on urine flow symptoms due to other previous or current diseases besides benign prostatic hyperplasia
6. Surgeries or radiation therapies for prostate gland, bladder or pelvis, or who had invasive treatments for benign prostatic hyperplasia
7. Severe medical condition which may be cause problem to conduct the clinical trial (e.g., chronic heart failure (CHF), difficult-to-control diabetes (HbA1c > 7%), mental disorder, drug, or alcohol abuse, etc.)
8. Moderate to severe liver hypofunction and severe kidney hypofunction (less than 30 mL/min of creatinine clearance)
9. Any other subjects who are considered to be ineligible for this study by an investigator

[Inclusion Criteria for Randomization]

1. Clinical signs and symptoms of benign prostatic hyperplasia

   1. Volume of prostate gland (TRUS) > 30 cc *
   2. moderate to severe lower urinary tract symptoms with IPSS ≥ 13
   3. 5-15 mL/sec of maximum flow rate (Qmax) measured when urine volume was at least 125 mL

2. Residual urine volume ≤ 200 mL

3. Patient's partner (Consent should be obtained before visit 4, when necessary.) - Consent to avoid pregnancy by using contraceptive devices or oral contraceptives during the patient's participation in clinical trial and for 90 days after the end of treatment, except if the partner reaches menopause or is surgically sterilized.

(* In case that additional TRUS examination has been performed after screening, a decision should be made based on the latest result.)