Trial Outcomes & Findings for Phase 2A Clinical Trial Evaluating the Potential Activity and Safety of hMaxi-K Gene for ED (NCT NCT02713789)

NCT ID: NCT02713789

Last Updated: 2019-08-08

Results Overview

Physical examination of the penis included inspection and palpation.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 26 participants
• Primary outcome timeframe: up to Week 24 ± 3 days
• Results posted on: 2019-08-08

Participant Flow

Participant milestones

Measure	hMaxi-K 8000 µg Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Overall Study STARTED	8	10	8
Overall Study COMPLETED	8	9	7
Overall Study NOT COMPLETED	0	1	1

Reasons for withdrawal

Measure	hMaxi-K 8000 µg Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Overall Study Lost to Follow-up	0	1	1

Baseline Characteristics

Phase 2A Clinical Trial Evaluating the Potential Activity and Safety of hMaxi-K Gene for ED

Baseline characteristics by cohort

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.	Total n=26 Participants Total of all reporting groups
Age, Continuous	55.8 years STANDARD_DEVIATION 7.32 • n=5 Participants	59.7 years STANDARD_DEVIATION 9.2 • n=7 Participants	59.1 years STANDARD_DEVIATION 6.31 • n=5 Participants	58.3 years STANDARD_DEVIATION 7.73 • n=4 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	10 Participants n=7 Participants	8 Participants n=5 Participants	26 Participants n=4 Participants
Race/Ethnicity, Customized Middle Eastern	7 Participants n=5 Participants	10 Participants n=7 Participants	8 Participants n=5 Participants	25 Participants n=4 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants

PRIMARY outcome

Timeframe: up to Week 24 ± 3 days

Population: Safety Population: all participants who were randomized to any of the treatment groups and who have received at least one dose of study treatment

Physical examination of the penis included inspection and palpation.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Number of Participants With Adverse Experiences as Measured by Changes in Physical Examination of the Penis	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: up to Week 24 ± 3 days

Population: Safety Population

Clinical significance was determined by the Investigator using central laboratory values.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Number of Participants With Significant Changes in Clinical Laboratory Parameters as Measured on Interval Blood and Urine Tests	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: up to Week 24 ± 3 days

Population: Safety Population

Clinical significance was determined by the Investigator using central laboratory values.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Number of Participants With Significant Changes on the Cardiogram as Measured by Significant Prolongation of QT Intervals and Cardiac Rhythm	0 Participants	2 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: Modified Intent-to-Treat (mITT) Population: all participants in the Intent-to-Treat (ITT) Population (all participants who were randomized to either of the treatment groups and who received at least one dose of study treatment) who had at least one post-dose efficacy assessment

SEP is a diary in which participants record each sexual attempt made throughout the study and is composed of 5 questions assessing sexual function. Two questions from the SEP deal with the ability to achieve vaginal penetration (Question 2: Were you able to insert your penis into your partner's vagina?), and the ability to maintain an erection (Question 3: Did your erection last long enough for you to have successful intercourse?). The number of "yes" response to each question in the SEP was computed as (Sum of all "yes" responses to respective SEP question /Total number of responses to that SEP question)*100. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Mean Change From Baseline in the Percentage of "Yes" Responses to Questions 2 and 3 of the Sexual Encounter Profile (SEP) at Week 24 SEP2	-18.8 percentage of responses Interval -47.78 to 10.28	-16.7 percentage of responses Interval -57.45 to 24.12	-28.6 percentage of responses Interval -73.7 to 16.56
Mean Change From Baseline in the Percentage of "Yes" Responses to Questions 2 and 3 of the Sexual Encounter Profile (SEP) at Week 24 SEP3	-3.1 percentage of responses Interval -10.51 to 4.26	0 percentage of responses Interval 0.0 to 0.0	0 percentage of responses Interval -53.4 to 53.4

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: mITT Population. Only those participants with data available were analyzed.

The IIEF is a validated, self-administered questionnaire that is a valid measure of male erectile dysfunction. The test contains 15 questions in 5 domains: (1) Erectile duration/function (questions [Q] 01 to 05 and 15); (2) Orgasmic function (questions 09 and 10); (3) Sexual desire (questions 11 and 12); (4) Intercourse satisfaction (questions 06, 07, and 08); (5) Overall sexual satisfaction (questions 13 and 14). The EF domain has been validated to assess erectile changes only. The domain-specific scores were calculated as the sum of the scores of the questions in the respective domain; thus, the total score of the EF domain was the sum of the 6 questions. Score range: 0 to 5 (Q01 to Q05), 1 to 5 (Q15). Total score: 1 to 30. Higher scores reflect better erectile function. Change from Baseline was calculated as the post-Baseline score minus the Baseline score.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=9 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=7 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Change From Baseline in the Erectile Function (EF) Domain of the International Index of Erectile Function (IIEF) at Week 24	-0.9 score on a scale Standard Error 2.86	-2.1 score on a scale Standard Error 1.85	-5.3 score on a scale Standard Error 2.08

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: mITT Population. Only those participants with data available were analyzed.

The IIEF is a validated, self-administered questionnaire that is a valid measure of male erectile dysfunction. The test contains 15 questions in 5 domains: (1) Erectile duration/function (questions 01 to 05 and 15); (2) Orgasmic function (questions 09 and 10); (3) Sexual desire (questions 11 and 12); (4) Intercourse satisfaction (questions 06, 07, and 08); (5) Overall sexual satisfaction (questions 13 and 14). The domain-specific scores were calculated as the sum of the scores of the questions in the respective domain; thus, the total score of the Orgasmic Function domain was the sum of 2 questions. Score range: 0 to 5 (Q09 and Q10). Total score: 0 to 10. Higher scores reflect better erectile function. Change from Baseline was calculated as the post-Baseline score minus the Baseline score.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=9 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=7 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Change From Baseline in the Orgasmic Function Domain of the International Index of Erectile Function (IIEF) at Week 24	-1.5 score on a scale Standard Error 1.40	1.1 score on a scale Standard Error 1.30	-0.3 score on a scale Standard Error 0.57

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: mITT Population. Only those participants with data available were analyzed.

The IIEF is a validated, self-administered questionnaire that is a valid measure of male erectile dysfunction. The test contains 15 questions in 5 domains: (1) Erectile duration/function (questions 01 to 05 and 15); (2) Orgasmic function (questions 09 and 10); (3) Sexual desire (questions 11 and 12); (4) Intercourse satisfaction (questions 06, 07, and 08); (5) Overall sexual satisfaction (questions 13 and 14). The domain-specific scores were calculated as the sum of the scores of the questions in the respective domain; thus, the total score of the Sexual Desire domain was the sum of 2 questions. Score range: 1 to 5 (Q11 and Q12). Total score: 2 to 10. Higher scores reflect better erectile function. Change from Baseline was calculated as the post-Baseline score minus the Baseline score.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=9 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=7 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Change From Baseline in the Sexual Desire Domain of the International Index of Erectile Function (IIEF) at Week 24	-0.9 score on a scale Standard Error 0.97	-1.0 score on a scale Standard Error 1.14	0.0 score on a scale Standard Error 1.29

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: mITT Population. Only those participants with data available were analyzed.

The IIEF is a validated, self-administered questionnaire that is a valid measure of male erectile dysfunction. The test contains 15 questions in 5 domains: (1) Erectile duration/function (questions 01 to 05 and 15); (2) Orgasmic function (questions 09 and 10); (3) Sexual desire (questions 11 and 12); (4) Intercourse satisfaction (questions 06, 07, and 08); (5) Overall sexual satisfaction (questions 13 and 14). The domain-specific scores were calculated as the sum of the scores of the questions in the respective domain; thus, the total score of the Intercourse Satisfaction domain was the sum of 3 questions. Score range: 0 to 5 (Q06 through Q8). Total score: 0 to 15. Higher scores reflect better erectile function. Change from Baseline was calculated as the post-Baseline score minus the Baseline score.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=9 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=7 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Change From Baseline in the Intercourse Satisfaction Domain of the International Index of Erectile Function (IIEF) at Week 24	-0.1 score on a scale Standard Error 0.93	-0.3 score on a scale Standard Error 1.07	-2.3 score on a scale Standard Error 0.75

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: mITT Population. Only those participants with data available were analyzed.

The IIEF is a validated, self-administered questionnaire that is a valid measure of male erectile dysfunction. The test contains 15 questions in 5 domains: (1) Erectile duration/function (questions 01 to 05 and 15); (2) Orgasmic function (questions 09 and 10); (3) Sexual desire (questions 11 and 12); (4) Intercourse satisfaction (questions 06, 07, and 08); (5) Overall sexual satisfaction (questions 13 and 14). The domain-specific scores were calculated as the sum of the scores of the questions in the respective domain; thus, the total score of the Overall Sexual Satisfaction domain was the sum of 2 questions. Score range: 1 to 5 (Q13 and Q14). Total score: 2 to 10. Higher scores reflect better erectile function. Change from Baseline was calculated as the post-Baseline score minus the Baseline score.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=9 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=7 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Change From Baseline in the Overall Sexual Satisfaction Domain of the International Index of Erectile Function (IIEF) at Week 24	-1.1 score on a scale Standard Error 0.69	0.1 score on a scale Standard Error 1.34	-1.11 score on a scale Standard Error 0.83

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: mITT Population

SEP is a diary in which participants record each sexual attempt made throughout the study and is composed of 5 questions assessing sexual function. Question 1: Were you able to achieve at least some erection?; Question 4: Were you satisfied with the hardness of your erection?; Question 5: Overall, were you satisfied with the sexual experience? The number of "yes" response to each question in the SEP was computed as (Sum of all "yes" responses to respective SEP question /Total number of responses to that SEP question)*100. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

Outcome measures

Measure	hMaxi-K 8000 µg n=8 Participants Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 Participants Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 Participants Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Mean Change From Baseline in the Percentage of "Yes" Responses to Questions 1, 4, and 5 of the Sexual Encounter Profile (SEP) at Week 24 SEP1	15.6 percentage of responses Interval -35.49 to 66.74	-31.0 percentage of responses Interval -80.04 to 18.14	-50.0 percentage of responses Interval -96.24 to -3.76
Mean Change From Baseline in the Percentage of "Yes" Responses to Questions 1, 4, and 5 of the Sexual Encounter Profile (SEP) at Week 24 SEP4	12.5 percentage of responses Interval -17.06 to 42.06	14.3 percentage of responses Interval -20.67 to 49.24	0 percentage of responses Interval 0.0 to 0.0
Mean Change From Baseline in the Percentage of "Yes" Responses to Questions 1, 4, and 5 of the Sexual Encounter Profile (SEP) at Week 24 SEP5	0 percentage of responses Interval 0.0 to 0.0	28.6 percentage of responses Interval -16.56 to 73.7	0 percentage of responses Interval 0.0 to 0.0

Adverse Events

hMaxi-K 8000 µg

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

hMaxi-K 16000 µg

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	hMaxi-K 8000 µg n=8 participants at risk Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 participants at risk Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 participants at risk Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Eye disorders Eye operation	12.5% 1/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Musculoskeletal and connective tissue disorders Osteoarthropathy	0.00% 0/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)

Other adverse events

Measure	hMaxi-K 8000 µg n=8 participants at risk Participants received a single injection of hMaxi-K 8000 micrograms (µg) into the corpus cavernosum of the penis.	hMaxi-K 16000 µg n=10 participants at risk Participants received a single injection of hMaxi-K 16000 µg into the corpus cavernosum of the penis.	Placebo n=8 participants at risk Participants received a single injection of matching placebo (PBS-20% sucrose) into the corpus cavernosum of the penis.
Gastrointestinal disorders Diarrhoea	0.00% 0/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)
Infections and infestations Nasopharyngitis	0.00% 0/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Injury, poisoning and procedural complications Injury	0.00% 0/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)
Investigations Aspartate aminotransferase increased	25.0% 2/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Investigations Blood alkaline phosphatase increased	12.5% 1/8 • Up to Week 24 (± 3 days)	20.0% 2/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Investigations Blood testosterone decreased	0.00% 0/8 • Up to Week 24 (± 3 days)	20.0% 2/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)
Investigations Electrocardiogram QT prolonged	25.0% 2/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Metabolism and nutrition disorders Hyperglycaemia	100.0% 8/8 • Up to Week 24 (± 3 days)	80.0% 8/10 • Up to Week 24 (± 3 days)	75.0% 6/8 • Up to Week 24 (± 3 days)
Metabolism and nutrition disorders Hypomagnesaemia	37.5% 3/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	25.0% 2/8 • Up to Week 24 (± 3 days)
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)
Nervous system disorders Headache	0.00% 0/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Renal and urinary disorders Proteinuria	37.5% 3/8 • Up to Week 24 (± 3 days)	40.0% 4/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Renal and urinary disorders Haematuria	12.5% 1/8 • Up to Week 24 (± 3 days)	30.0% 3/10 • Up to Week 24 (± 3 days)	25.0% 2/8 • Up to Week 24 (± 3 days)
Reproductive system and breast disorders Benign prostatic hyperplasia	0.00% 0/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)
Reproductive system and breast disorders Penile pain	0.00% 0/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Skin and subcutaneous tissue disorders Skin reaction	0.00% 0/8 • Up to Week 24 (± 3 days)	10.0% 1/10 • Up to Week 24 (± 3 days)	0.00% 0/8 • Up to Week 24 (± 3 days)
Vascular disorders Hypertension	12.5% 1/8 • Up to Week 24 (± 3 days)	0.00% 0/10 • Up to Week 24 (± 3 days)	12.5% 1/8 • Up to Week 24 (± 3 days)

Additional Information

JP Nicandro, PharmD

Urovant Sciences GmbH

Phone: 833-219-5557

Email: clinicaltrials@urovant.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place