Trial Outcomes & Findings for Efficacy and Safety, Long-Term Study of NPC-01 to Treat Dysmenorrhea Associated With Endometriosis (NCT NCT00902746)
NCT ID: NCT00902746
Last Updated: 2025-06-15
Results Overview
The detail of dysmenorrhea score that was used in this study is the following. These subscales summed for a total dysmenorrhea score (minimum 0 to maximum 6). Pain score None 0 : None Mild 1 : There are some troubles for work Moderate 2 : Needing to rest in bed and/or affecting work Severe 3 : Morre than 1 day in bed and not possible to work Drug score (during a menstrual period) None 0 : None Mild 1 : taking analgesics for 1 days Moderate 2 : taking analgesics for 2 days Severe 3 : taking analgesics more than 3 days
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
149 participants
Primary outcome timeframe
52 weeks
Results posted on
2025-06-15
Participant Flow
Total 168 patients were consented and registered at minus 2 cycles of study drug administration as provisional registration. After provisional registration, investigators confirmed their eligibility of patients by test gynecological examination, transvaginal ultrasound etc.) and 149 patients were formally registered for this study.
Participant milestones
| Measure |
NPC-01
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
|
|---|---|
|
Overall Study
STARTED
|
149
|
|
Overall Study
Initiation of Study Drug Administration
|
148
|
|
Overall Study
Transfer to Step 2
|
133
|
|
Overall Study
COMPLETED
|
113
|
|
Overall Study
NOT COMPLETED
|
36
|
Reasons for withdrawal
| Measure |
NPC-01
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
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|---|---|
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Overall Study
Protocol Violation
|
7
|
|
Overall Study
Withdrawal by Subject
|
16
|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Lost to Follow-up
|
3
|
|
Overall Study
Physician Decision
|
2
|
Baseline Characteristics
Efficacy and Safety, Long-Term Study of NPC-01 to Treat Dysmenorrhea Associated With Endometriosis
Baseline characteristics by cohort
| Measure |
NPC-01
n=147 Participants
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg, or Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
|
|---|---|
|
Age, Continuous
|
33.2 years
STANDARD_DEVIATION 7.12 • n=5 Participants
|
|
Age, Customized
Under 30 years of age
|
51 participants
n=5 Participants
|
|
Age, Customized
30 year of age and older
|
96 participants
n=5 Participants
|
|
Sex/Gender, Customized
Female
|
147 participants
n=5 Participants
|
|
Total Dysmenorrhea Score (VRS; Verbal Rating Scale)
|
4.1 units on a scale
STANDARD_DEVIATION 0.99 • n=5 Participants
|
|
Visual Analogue Scale
|
53.1 units on a scale
STANDARD_DEVIATION 21.49 • n=5 Participants
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: This analysis was carried out for 112 patients whom end of study data was available on FAS.
The detail of dysmenorrhea score that was used in this study is the following. These subscales summed for a total dysmenorrhea score (minimum 0 to maximum 6). Pain score None 0 : None Mild 1 : There are some troubles for work Moderate 2 : Needing to rest in bed and/or affecting work Severe 3 : Morre than 1 day in bed and not possible to work Drug score (during a menstrual period) None 0 : None Mild 1 : taking analgesics for 1 days Moderate 2 : taking analgesics for 2 days Severe 3 : taking analgesics more than 3 days
Outcome measures
| Measure |
NPC-01
n=112 Participants
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
|
|---|---|
|
Patient Response to Treatment for Dysmenorrhea, as Evaluated by Difference of Total Dysmenorrhea Score (Baseline/Pretreatment-End of Treatment)
|
2.4 units on a scale
Standard Deviation 1.80
|
SECONDARY outcome
Timeframe: 52 weeksPopulation: This analysis was carried out for 112 patients whom end of study data was available on FAS.
VAS stands for Visual Analogue Scale of pain. The scale was rated as a graphic rating scale. as a 100mm baseline from 0:No pain to 100:Worst possible pain.
Outcome measures
| Measure |
NPC-01
n=112 Participants
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
|
|---|---|
|
Difference in the Visual Analog Scale (VAS) of Dysmenorrhea (Baseline/Pretreatment-dnd of Treatment)
|
28.4 units on a scale
Standard Deviation 25.13
|
Adverse Events
NPC-01
Serious events: 5 serious events
Other events: 147 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
NPC-01
n=147 participants at risk
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
|
|---|---|
|
Nervous system disorders
Depressed level of consciousness
|
0.68%
1/147 • Number of events 1 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Endometriosis
|
0.68%
1/147 • Number of events 1 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Infections and infestations
Cellulitis orbital
|
0.68%
1/147 • Number of events 1 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Vascular disorders
Haemorrhagic ovrian cyst
|
1.4%
2/147 • Number of events 2 • From study administration to 15 cycles of menstruation, an average of 14 months
|
Other adverse events
| Measure |
NPC-01
n=147 participants at risk
Norethisterone, Ethinyl Estradiol
NPC-01: This study consist of the following steps.
Step 1(Norethisterone 0.6mg, Ethinyl Estradiol 0.035mg):From first study medication to 3th menstrual cycles.
Step 2(Norethisterone 1mg, Ethinyl Estradiol 0.035mg):
After interim analysis of the results of step 1, in the case of the efficacy results meet prespecified criterion, same dose level will be continued to 13th menstrual cycles including step 1. If the results do not meet prespecified criterion, the dose will be increased to Norethisterone 1mg, Ethinyl Estradiol 0.035mg and be continued newly until 13th menstrual cycle from this point.
|
|---|---|
|
Gastrointestinal disorders
Abdominal discomfort
|
7.5%
11/147 • Number of events 20 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Gastrointestinal disorders
Abdominal pain upper
|
11.6%
17/147 • Number of events 29 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Gastrointestinal disorders
Constipation
|
6.8%
10/147 • Number of events 10 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Gastrointestinal disorders
Dental caries
|
8.2%
12/147 • Number of events 13 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Gastrointestinal disorders
Diarrhoea
|
9.5%
14/147 • Number of events 34 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Gastrointestinal disorders
Gastritis
|
6.1%
9/147 • Number of events 9 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Gastrointestinal disorders
Nausea
|
17.0%
25/147 • Number of events 42 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
General disorders
Malaise
|
8.8%
13/147 • Number of events 17 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Infections and infestations
Cystitis
|
6.1%
9/147 • Number of events 9 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Infections and infestations
Gastroenteritis
|
8.8%
13/147 • Number of events 14 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Infections and infestations
Influenza
|
8.2%
12/147 • Number of events 12 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Infections and infestations
Nasopharyngitis
|
64.6%
95/147 • Number of events 242 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Investigations
Blood triglycerides increased
|
6.1%
9/147 • Number of events 11 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Investigations
Weight increased
|
6.8%
10/147 • Number of events 10 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Investigations
Protein urine present
|
5.4%
8/147 • Number of events 8 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.5%
11/147 • Number of events 13 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Nervous system disorders
Headache
|
36.1%
53/147 • Number of events 220 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Amenorrhoea
|
6.8%
10/147 • Number of events 11 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Hypomenorrhoea
|
15.0%
22/147 • Number of events 27 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Menorrhagia
|
16.3%
24/147 • Number of events 42 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Metrorrhagia
|
89.8%
132/147 • Number of events 916 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Oligomenorrhoea
|
45.6%
67/147 • Number of events 135 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Polymenorrhoea
|
12.9%
19/147 • Number of events 29 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Breast discomfort
|
5.4%
8/147 • Number of events 11 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Reproductive system and breast disorders
Haemorrhagic ovarian cyst
|
9.5%
14/147 • Number of events 14 • From study administration to 15 cycles of menstruation, an average of 14 months
|
|
Skin and subcutaneous tissue disorders
Acnes
|
5.4%
8/147 • Number of events 9 • From study administration to 15 cycles of menstruation, an average of 14 months
|
Additional Information
Department director of clinical development department 1
Nobelpharma
Phone: +81-3-5651-1177
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee There is an agreement between the sponsor and PI that no restricts the PI's right but need to discuss the timing of publish date of trial results after the trial is completed.
- Publication restrictions are in place
Restriction type: OTHER