Trial Outcomes & Findings for PGL4001 Versus Placebo in Uterine Myomas (NCT NCT00755755)
NCT ID: NCT00755755
Last Updated: 2012-12-13
Results Overview
Uterine bleeding was assessed with the use of the PBAC, a validated self-reporting method to estimate menstrual blood loss. Patients recorded daily the number of tampons and towels used and the degree to which individual items were soiled with blood (plus small or large clots). Monthly scores range from 0 (amenorrhea) to more than 500, with higher numbers indicating more bleeding. A slightly stained tampon/towel scores 1, a partially stained tampon/towel scores 5, a completely saturated tampon scores 10 and a completely saturated towel scores 20. Small clots/flooding (2cm) score 1. Large clots/flooding (3cm) score 5. Menorrhagia is defined as a PBAC \> 100 during one menstrual period which approximates to a blood loss of \> 80 mL. A PBAC of 400 corresponds to a blood loss of around 300 mL or approximately 80 tampons/towels used. The week 13 PBAC score was calculated using the last 28 days of treatment.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
241 participants
Primary outcome timeframe
Week 13 visit
Results posted on
2012-12-13
Participant Flow
Participant milestones
| Measure |
A (PGL4001 5mg)
PGL4001 5 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
B (PGL4001 10mg)
PGL4001 10 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
C (Placebo)
PGL4001 matching placebo (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
|---|---|---|---|
|
Overall Study
STARTED
|
95
|
98
|
48
|
|
Overall Study
COMPLETED
|
89
|
90
|
45
|
|
Overall Study
NOT COMPLETED
|
6
|
8
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
PGL4001 Versus Placebo in Uterine Myomas
Baseline characteristics by cohort
| Measure |
A (PGL4001 5mg)
n=95 Participants
PGL4001 5 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
B (PGL4001 10mg)
n=98 Participants
PGL4001 10 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
C (Placebo)
n=48 Participants
PGL4001 matching placebo (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
Total
n=241 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
95 Participants
n=5 Participants
|
98 Participants
n=7 Participants
|
48 Participants
n=5 Participants
|
241 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age Continuous
|
41.2 years
STANDARD_DEVIATION 5.9 • n=5 Participants
|
42.0 years
STANDARD_DEVIATION 5.5 • n=7 Participants
|
41.6 years
STANDARD_DEVIATION 5.6 • n=5 Participants
|
41.6 years
STANDARD_DEVIATION 5.7 • n=4 Participants
|
|
Sex: Female, Male
Female
|
95 Participants
n=5 Participants
|
98 Participants
n=7 Participants
|
48 Participants
n=5 Participants
|
241 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
Czech Republic
|
7 participants
n=5 Participants
|
6 participants
n=7 Participants
|
3 participants
n=5 Participants
|
16 participants
n=4 Participants
|
|
Region of Enrollment
Hungary
|
7 participants
n=5 Participants
|
11 participants
n=7 Participants
|
9 participants
n=5 Participants
|
27 participants
n=4 Participants
|
|
Region of Enrollment
India
|
10 participants
n=5 Participants
|
11 participants
n=7 Participants
|
7 participants
n=5 Participants
|
28 participants
n=4 Participants
|
|
Region of Enrollment
Romania
|
17 participants
n=5 Participants
|
17 participants
n=7 Participants
|
8 participants
n=5 Participants
|
42 participants
n=4 Participants
|
|
Region of Enrollment
Russian Federation
|
28 participants
n=5 Participants
|
19 participants
n=7 Participants
|
13 participants
n=5 Participants
|
60 participants
n=4 Participants
|
|
Region of Enrollment
Ukraine
|
26 participants
n=5 Participants
|
34 participants
n=7 Participants
|
8 participants
n=5 Participants
|
68 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Week 13 visitPopulation: Intent-to-treat
Uterine bleeding was assessed with the use of the PBAC, a validated self-reporting method to estimate menstrual blood loss. Patients recorded daily the number of tampons and towels used and the degree to which individual items were soiled with blood (plus small or large clots). Monthly scores range from 0 (amenorrhea) to more than 500, with higher numbers indicating more bleeding. A slightly stained tampon/towel scores 1, a partially stained tampon/towel scores 5, a completely saturated tampon scores 10 and a completely saturated towel scores 20. Small clots/flooding (2cm) score 1. Large clots/flooding (3cm) score 5. Menorrhagia is defined as a PBAC \> 100 during one menstrual period which approximates to a blood loss of \> 80 mL. A PBAC of 400 corresponds to a blood loss of around 300 mL or approximately 80 tampons/towels used. The week 13 PBAC score was calculated using the last 28 days of treatment.
Outcome measures
| Measure |
A (PGL4001 5mg)
n=94 Participants
PGL4001 5 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
B (PGL4001 10mg)
n=93 Participants
PGL4001 10 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
C (Placebo)
n=48 Participants
PGL4001 matching placebo (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
|---|---|---|---|
|
Co-primary Endpoint: Percentage of Subjects With Reduction in Uterine Bleeding Defined as a Pictorial Blood-loss Assessment Chart (PBAC) Score <75 at End-of-treatment Visit (Week 13)
|
91.5 percentage of patients
|
92.5 percentage of patients
|
18.8 percentage of patients
|
PRIMARY outcome
Timeframe: Week 13Population: Intent-to-treat
Percent change in total fibroid volume from screening to end of treatment visit (Week 13 visit) assessed by MRI and read centrally by a radiologist who was unaware of the study-group assignments. The total fibroid volume was the sum of the individual fibroid volumes.
Outcome measures
| Measure |
A (PGL4001 5mg)
n=85 Participants
PGL4001 5 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
B (PGL4001 10mg)
n=80 Participants
PGL4001 10 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
C (Placebo)
n=45 Participants
PGL4001 matching placebo (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
|---|---|---|---|
|
Co-primary Endpoint: Percent Change in Total Myoma Volume Assessed by Magnetic Resonance Imaging (MRI) From Screening to End of Treatment Visit (Week 13 Visit)
|
-21.2 percentage of change
Interval -100.0 to 223.4
|
-12.3 percentage of change
Interval -100.0 to 146.6
|
3 percentage of change
Interval -100.0 to 134.8
|
Adverse Events
A (PGL4001 5mg)
Serious events: 2 serious events
Other events: 47 other events
Deaths: 0 deaths
B (PGL4001 10mg)
Serious events: 1 serious events
Other events: 52 other events
Deaths: 0 deaths
C (Placebo)
Serious events: 2 serious events
Other events: 22 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
A (PGL4001 5mg)
n=95 participants at risk
PGL4001 5 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
B (PGL4001 10mg)
n=98 participants at risk
PGL4001 10 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
C (Placebo)
n=48 participants at risk
PGL4001 matching placebo (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
|---|---|---|---|
|
Reproductive system and breast disorders
Ovarian hemorrhage
|
1.1%
1/95 • Number of events 1 • 3 years 2 months
|
0.00%
0/98 • 3 years 2 months
|
0.00%
0/48 • 3 years 2 months
|
|
Reproductive system and breast disorders
Uterine hemorrhage
|
1.1%
1/95 • Number of events 1 • 3 years 2 months
|
1.0%
1/98 • Number of events 1 • 3 years 2 months
|
0.00%
0/48 • 3 years 2 months
|
|
Reproductive system and breast disorders
New uterine leiomyoma
|
0.00%
0/95 • 3 years 2 months
|
0.00%
0/98 • 3 years 2 months
|
2.1%
1/48 • Number of events 1 • 3 years 2 months
|
|
Reproductive system and breast disorders
breast cancer
|
0.00%
0/95 • 3 years 2 months
|
0.00%
0/98 • 3 years 2 months
|
2.1%
1/48 • Number of events 1 • 3 years 2 months
|
Other adverse events
| Measure |
A (PGL4001 5mg)
n=95 participants at risk
PGL4001 5 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
B (PGL4001 10mg)
n=98 participants at risk
PGL4001 10 mg (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
C (Placebo)
n=48 participants at risk
PGL4001 matching placebo (oral tablets) with concomitant oral administration of 1 tablet containing 80 mg Fe2+
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
2.1%
2/95 • Number of events 3 • 3 years 2 months
|
3.1%
3/98 • Number of events 5 • 3 years 2 months
|
4.2%
2/48 • Number of events 2 • 3 years 2 months
|
|
Gastrointestinal disorders
Constipation
|
4.2%
4/95 • Number of events 6 • 3 years 2 months
|
0.00%
0/98 • 3 years 2 months
|
2.1%
1/48 • Number of events 1 • 3 years 2 months
|
|
Nervous system disorders
Headache
|
4.2%
4/95 • Number of events 6 • 3 years 2 months
|
10.2%
10/98 • Number of events 19 • 3 years 2 months
|
4.2%
2/48 • Number of events 2 • 3 years 2 months
|
|
Reproductive system and breast disorders
Breat pain / tenderness / disconfort
|
2.1%
2/95 • Number of events 2 • 3 years 2 months
|
6.1%
6/98 • Number of events 6 • 3 years 2 months
|
0.00%
0/48 • 3 years 2 months
|
|
Reproductive system and breast disorders
Dysmenorrhea
|
0.00%
0/95 • 3 years 2 months
|
0.00%
0/98 • 3 years 2 months
|
4.2%
2/48 • Number of events 3 • 3 years 2 months
|
|
Infections and infestations
Nasopharyngitis
|
3.2%
3/95 • Number of events 3 • 3 years 2 months
|
0.00%
0/98 • 3 years 2 months
|
0.00%
0/48 • 3 years 2 months
|
|
Endocrine disorders
Hypothyroidism
|
2.1%
2/95 • Number of events 2 • 3 years 2 months
|
4.1%
4/98 • Number of events 4 • 3 years 2 months
|
0.00%
0/48 • 3 years 2 months
|
|
Metabolism and nutrition disorders
Hypercholesterolemia
|
3.2%
3/95 • Number of events 4 • 3 years 2 months
|
2.0%
2/98 • Number of events 2 • 3 years 2 months
|
2.1%
1/48 • Number of events 1 • 3 years 2 months
|
|
General disorders
Pyrexia
|
3.2%
3/95 • Number of events 3 • 3 years 2 months
|
2.0%
2/98 • Number of events 2 • 3 years 2 months
|
4.2%
2/48 • Number of events 2 • 3 years 2 months
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders
|
14.7%
14/95 • Number of events 18 • 3 years 2 months
|
14.3%
14/98 • Number of events 16 • 3 years 2 months
|
12.5%
6/48 • Number of events 10 • 3 years 2 months
|
|
Nervous system disorders
Nervous system disorders
|
4.2%
4/95 • Number of events 7 • 3 years 2 months
|
11.2%
11/98 • Number of events 22 • 3 years 2 months
|
6.2%
3/48 • Number of events 4 • 3 years 2 months
|
|
Infections and infestations
Infections and infestations
|
8.4%
8/95 • Number of events 8 • 3 years 2 months
|
12.2%
12/98 • Number of events 15 • 3 years 2 months
|
6.2%
3/48 • Number of events 4 • 3 years 2 months
|
|
Endocrine disorders
Endocrine disorders
|
5.3%
5/95 • Number of events 8 • 3 years 2 months
|
8.2%
8/98 • Number of events 8 • 3 years 2 months
|
2.1%
1/48 • Number of events 2 • 3 years 2 months
|
|
General disorders
General disorders and administration site conditions
|
5.3%
5/95 • Number of events 5 • 3 years 2 months
|
4.1%
4/98 • Number of events 4 • 3 years 2 months
|
4.2%
2/48 • Number of events 3 • 3 years 2 months
|
|
Nervous system disorders
Diziness
|
1.1%
1/95 • Number of events 1 • 3 years 2 months
|
3.1%
3/98 • Number of events 3 • 3 years 2 months
|
0.00%
0/48 • 3 years 2 months
|
|
Infections and infestations
Influenza
|
1.1%
1/95 • Number of events 1 • 3 years 2 months
|
3.1%
3/98 • Number of events 3 • 3 years 2 months
|
2.1%
1/48 • Number of events 1 • 3 years 2 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders
|
7.4%
7/95 • Number of events 10 • 3 years 2 months
|
4.1%
4/98 • Number of events 4 • 3 years 2 months
|
4.2%
2/48 • Number of events 3 • 3 years 2 months
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
9.5%
9/95 • Number of events 16 • 3 years 2 months
|
10.2%
10/98 • Number of events 13 • 3 years 2 months
|
6.2%
3/48 • Number of events 7 • 3 years 2 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place