Phase 2
N=271
Safety and Efficacy of Elagolix in Pre-Menopausal Women With Heavy Uterine Bleeding and Uterine Fibroids
Heavy Uterine Bleeding · Uterine Fibroids
Bottom Line
View on ClinicalTrials.gov: NCT01441635 ↗Enrolled (actual)
271
Serious AEs
2.9%
Results posted
Jul 2020
Primary outcome: Primary: Mean Change From Baseline to the Last 28 Days of Treatment in Menstrual Blood Loss (MBL) — 213.70; 269.36; 321.73; 335.11 mL — p=< 0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Elagolix (Drug); Placebo (Drug); Estradiol/Norethindrone acetate (E2/NETA) (Drug); Estradiol (Drug); Progesterone (Drug)
- Age
- Adult · 20+ yrs
- Sex
- Female
- Sponsor
- AbbVie (prior sponsor, Abbott)
- Primary completion
- Nov 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change From Baseline to the Last 28 Days of Treatment in Menstrual Blood Loss (MBL) |
213.70; 269.36; 321.73; 335.11; 251.72; 247.70 | < 0.001 sig |
| SECONDARY Percent Change From Baseline to the Last 28 Days of Treatment in Menstrual Blood Loss (MBL) |
-83.83; -71.85; -6.98; -81.03; -11.12; -79.60 | < 0.001 sig |
| SECONDARY Percentage of Participants With MBL < 80 mL and With a ≥ 50% Reduction From Baseline in MBL During the Last 28 Days of Treatment |
84; 74; 13; 85; 17; 85 | < 0.001 sig |
| SECONDARY Percentage of Participants With MBL < 80 mL During the Last 28 Days of Treatment |
84; 74; 13; 85; 22; 88 | < 0.001 sig |
| SECONDARY Percentage of Participants With a ≥ 50% Reduction From Baseline in MBL During the Last 28 Days of Treatment |
84; 74; 13; 91; 28; 85 | < 0.001 sig |
| SECONDARY Percentage of Participants With No Change, Decrease From Baseline, or Increase From Baseline in Hemoglobin at Month 3 |
0; 0; 9; 0; 0; 0 | — |
| SECONDARY Change in Hemoglobin Concentration From Baseline to Month 3 |
1.18; 1.30; -0.43; 1.13; 0.28; 0.92 | < 0.001 sig |
| SECONDARY Change From Baseline to Month 3 in Uterine Bleeding Score |
-0.50; -0.37; -0.19; -0.52; -0.22; -0.24 | 0.011 sig |
| SECONDARY Change From Baseline to Month 3 in Percentage of Days With Any Uterine Bleeding |
-15.22; -11.00; -5.78; -15.82; -6.99; 3.63 | 0.020 sig |
| SECONDARY Change From Baseline to Month 3 in Percentage of Days With Moderate to Very Heavy Bleeding |
-7.22; -5.00; -4.00; -7.03; -3.08; -7.92 | 0.008 sig |
| SECONDARY Percentage of Participants With Any Uterine Bleeding or Moderate to Very Heavy Uterine Bleeding at Month 3 |
37; 57; 93; 47; 94; 78 | — |
| SECONDARY Percentage of Participants With Suppression of Bleeding (Spotting Allowed) or Amenorrhea During the Last 56 Days of Treatment |
66; 45; 0; 66; 0; 31 | — |
| SECONDARY Percent Change From Baseline to Month 3 in Uterine Volume |
-21.01; -21.37; 18.72; -21.68; -8.62; -17.43 | < 0.001 sig |
| SECONDARY Percentage of Participants With ≥ 25% Reduction in Uterine Volume at Month 3 / Final Visit |
53; 43; 7; 48; 11; 42 | 0.003 sig |
| SECONDARY Percent Change From Baseline to Month 3 in Volume of the Largest Fibroid |
14.23; -22.19; -7.26; -38.52; -2.05; -25.77 | 0.161 |
| SECONDARY Percentage of Participants With ≥ 25% Reduction in Volume of Largest Fibroid at Month 3 / Final Visit |
57; 52; 33; 68; 35; 58 | 0.203 |
| SECONDARY Change From Baseline to Month 3 in the Uterine Fibroid Symptom Quality of Life Questionnaire (UFS-QoL) |
-39.0; -33.2; -19.6; -31.6; -21.4; -20.3 | — |
| SECONDARY Change From Baseline to Month 3 in the Uterine Fibroids Daily Symptom Scale Scores |
-1.0; -0.2; -0.3; -0.6; -1.4; -1.1 | — |
| SECONDARY Change From Baseline to Month 3 in the Subject Surgery Intention Questionnaire (SSIQ) Version 2.0 |
-1.2; -3.1; 1.0; -1.8; 2.3; -1.4 | — |
| SECONDARY Change From Baseline to Month 3 in the Physician Surgery Intention Questionnaire (PSIQ) Version 2.0 |
-0.9; -1.3; -2.7; -0.8; 0.7; -0.6 | — |
Summary
The purpose of this proof-of-concept study is to assess the safety and effectiveness of elagolix versus placebo to reduce uterine bleeding associated with uterine fibroids, and to reduce fibroid volume and uterine volume in premenopausal women 20 to 49 years of age with heavy uterine bleeding.
Eligibility Criteria
Inclusion Criteria
- Subject is a pre-menopausal female 20 to 49 years of age.
- Subject has a diagnosis of uterine fibroids documented by a pelvic ultrasound assessed by a central reader and verification that a fibroid present met the following criteria:
- At least 1 fibroid with diameter ≥ 2 cm (longest diameter), or multiple small fibroids with a total uterine volume of ≥ 200 cm³ to ≤ 2,500 cm³ (approximately 22 weeks' gestation) as documented by a centrally read ultrasound.
- Only intramural, submucosal non-pedunculated, and subserosal fibroids qualified subjects for enrollment (intracavitary pedunculated fibroids were exclusionary).
- Ultrasound procedures were performed during the Screening Period, and subjects were not randomized until the investigator reviewed the central reader results verifying the inclusion requirements.
- Subject has a history of regular menstrual cycles between 24 to 35 days.
- Subject has heavy uterine bleeding associated with uterine fibroids as evidenced by blood loss > 80 mL during 2 screening menstrual cycles, measured by the alkaline hematin method.
Exclusion Criteria
- Subject has had a myomectomy, uterine artery embolization, or high intensity focused ultrasound for fibroid destruction within 1 year prior to randomization or any history of endometrial ablation.
- Subject has a history of osteoporosis or other metabolic bone disease.
- Subject shows evidence of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric (including depression), or neurologic diseases or any uncontrolled medical illness such as uncontrolled type 2 diabetes.
- Subject has a history of clinically significant condition(s) including but not limited to:
- Endometriosis
- Epilepsy or seizures
- Type 1 diabetes
- Any cancer (except basal cell carcinoma of the skin), including breast or ovarian cancer or subject has taken any systemic cancer chemotherapy
Data sourced from ClinicalTrials.gov (NCT01441635). 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.