MedPath

A Study to Assess the Efficacy and Safety of OBE2109 in Subjects With Endometriosis

Phase 2
Completed
Conditions
Endometriosis
Interventions
Drug: Placebo
Registration Number
NCT02778399
Lead Sponsor
ObsEva SA
Brief Summary

The primary objective of this study is to assess the efficacy and safety of a range of oral doses of OBE2109 versus placebo, in reducing endometriosis associated pain.

Detailed Description

The study is a prospective, dose-finding, randomized, parallel group, double-blind, placebo-controlled phase 2b study investigating the efficacy and safety of OBE2109 in the treatment of 330 women with moderate-to-severe endometriosis associated pain.

Subject will be randomized to one of 6 treatment groups in a 1:1:1:1:1:1 ratio (1 placebo group, 5 dose groups with different dosage/regimen).

Eligible subjects will be offered the opportunity to continue treatment with OBE2109 in an extension phase. Subjects who do not continue in the extension will enter the treatment-free follow-up phase of the study.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
328
Inclusion Criteria
  • The subject must have had her most recent surgical and - if available - histological, diagnosis of pelvic endometriosis up to 10 years before screening.
  • The subject has moderate to severe endometriosis-associated pain during the screening period.
  • The subject has regular menstrual cycles.
  • The subject has a BMI ≥ 18 kg/m2 at the screening visit.

Key

Exclusion Criteria
  • The subject is pregnant or breast feeding or is planning a pregnancy within the duration of the treatment period of the study.
  • The subject had an interventional surgery for endometriosis performed within a period of 60 days before screening.
  • The subject did not respond to prior treatment with gonadotropin releasing hormone (GnRH) agonists or GnRH antagonists for endometriosis.
  • The subject has a history of, or known osteoporosis or other metabolic bone disease.
  • The subject has chronic pelvic pain that is not caused by endometriosis and requires chronic analgesic / therapy, or that would interfere with the assessment of endometriosis related pain.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
OBE2109 200 mgOBE2109-
Placebo / OBE2109 100mgPlaceboParticipants received placebo for the first 12 weeks and were then crossed-over to active treatment with OBE2109 100mg for a further 12 weeks.
Placebo / OBE2109 100mgOBE2109Participants received placebo for the first 12 weeks and were then crossed-over to active treatment with OBE2109 100mg for a further 12 weeks.
OBE2109 100mgOBE2109-
OBE2109 50 mgOBE2109-
OBE2109 75mg fixed dose (FD)OBE2109-
OBE2109 75mg titrated dose (TD)OBE2109-
Primary Outcome Measures
NameTimeMethod
Percentage of Subjects With 30% or Greater Reduction From Baseline to Week 12 in Mean Overall Pelvic Pain Score (0-3 VRS)From baseline to week 12

The primary efficacy endpoint of the study was a response at Week 12, with response defined as a reduction of 30% or greater from baseline in the mean overall pelvic pain score, defined as the mean of daily pain scores reported in electronic diary during the preceding 28 days (4-week period), assessed on a Verbal Rating Scale for pelvic pain of 0 (no pain) to 3 (severe pain). The baseline mean score was calculated as the mean of daily scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The relevant time points are Baseline and Week 12.

Secondary Outcome Measures
NameTimeMethod
Percentage of Subjects With 30% or Greater Reduction From Baseline to Week 12 in Mean Pelvic Pain Scores (0-3 VRS) for Days With Uterine BleedingFrom baseline to week 12

This endpoint corresponds to a response at Week 12, with response defined as a reduction of 30% or greater from baseline in the mean pelvic pain score for days with uterine bleeding/spotting, defined as the mean of daily pain scores on days with uterine bleeding/spotting recorded in electronic diary during the preceding 28 days (4-week period), assessed on a Verbal Rating Scale for pelvic pain of 0 (no pain) to 3 (severe pain). The baseline mean score was calculated as the mean of daily scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The relevant time points are Baseline and Week 12.

Change From Baseline to Week 12 in the Mean Overall Pelvic Pain Score (0-10 NRS)From baseline to week 12

This endpoint corresponds to the change from baseline to Week 12 in the mean overall pelvic pain score, defined as the mean of daily pain scores reported in electronic diary during the preceding 28 days (4-week period), assessed on a Numerical Rating Scale (NRS) for pelvic pain of 0 (no pelvic pain) to 10 (worst pelvic pain imaginable). The baseline mean score was calculated as the mean of daily scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The relevant time points are Baseline and Week 12.

Percentage Change From Baseline to Week 24 in Bone Mineral Density (BMD)From baseline up to week 24

Change from baseline to Week 24 in BMD assessed by dual-energy X-ray absorptiometry (DXA) scan of LUMBAR SPINE.

Percentage of Subjects With 30% or Greater Reduction From Baseline to Week 12 in Mean Pelvic Pain Scores (0-3 VRS) for Days With no Uterine BleedingFrom baseline to week 12

This endpoint corresponds to a response at Week 12, with response defined as a reduction of 30% or greater from baseline in the mean pelvic pain score for days with no uterine bleeding, defined as the mean of daily pain scores on days with no uterine bleeding recorded in electronic diary during the preceding 28 days (4-week period) on a Verbal Rating Scale for pelvic pain of 0 (no pain) to 3 (severe pain). The baseline mean score was calculated as the mean of daily scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The relevant time points are Baseline and Week 12.

Percentage of Subjects With Any Analgesics Use at Week 12Up to week 12

This endpoint corresponds to the percentage of subjects at week 12 who recorded at least one pain medication intake in electronic diary during the preceding 28 days (4-week period).

Change From Baseline to Week 12 in the Mean Dyschezia Score (0-10 NRS)From baseline to week 12

This endpoint corresponds to the change from baseline to week 12 in the mean dyschezia score, defined as the mean of weekly dyschezia scores reported in electronic diary during the preceding 28 days (4-week period), assessed on a 0-10 Numerical Rating Scale for dyschezia, with 0 representing no pain and 10 representing the worst pain imaginable. The baseline mean score was calculated as the mean of weekly scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The relevant time points are Baseline and Week 12.

Number of Non Benign Endometrial Biopsies at Week 24Week 24

Any pathological changes in the endometrium at week 24 were assessed from endometrial biopsies. The number of non benign biopsies at Week 24 is presented per treatment arm.

Note: an isolated case of hyperplasia (without atypia) was observed at week 12 in the 200 mg group in a subject whose screening biopsy results were normal. A follow-up biopsy at week 24 revealed no abnormalities.

Change From Baseline to Week 12 in the Mean Dyspareunia Score (0-3 VRS)From baseline to week 12

This endpoint corresponds to the change from baseline to Week 12 in the mean dyspareunia score, defined as the mean of daily dyspareunia scores recorded in electronic diary during the preceding 28 days (4-week period), assessed on a 0-3 Verbal Rating Scale (VRS) for dyspareunia, with 0 representing "No discomfort during sexual intercourse" and 3 representing "I avoided sexual intercourse because of pain". The baseline mean score was calculated as the mean of daily scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The dyspareunia questionnaire also included an option "not applicable: I was not sexually active for reasons other than my endometriosis or did not have sexual intercourse"; for scoring, answering "not applicable" was considered like a missing value.

The relevant time points are Baseline and Week 12.

Percentage of Subjects With Improvement in the Patient Global Impression of Change (PGIC) Score at Week 12Up to week 12

The PGIC questionnaire consists of one question rated on a seven point scale (1="Very Much Improved" to 7="Very Much Worse"), with which the subject had to qualify her overall status since the start of the study. The PGIC was answered on electronic diary after activation by site staff during Week 12 visit at site.

This endpoint corresponds to the percentage of subjects with an "improvement" in the PGIC score, which includes all subjects who answered "Very much improved" or "Much improved" or "Minimally improved" at Week 12.

Percentage Change From Baseline to Week 24 in the Clinical Laboratory Assessments: LDLFrom baseline up to week 24

This endpoint reports the change from baseline up to Week 24 in the clinical laboratory assessments: LDL cholesterol.

Change From Baseline to Week 12 in the Mean Score of Endometriosis Health Profile-30 (EHP-30) Pain DomainFrom baseline to week 12

This endpoint corresponds to the change from baseline to Week 12 in the mean score of pain dimension of the EHP-30. The EHP-30 questionnaire was answered on electronic diary after activation by site staff during subject's monthly visits at site.

The EHP-30 pain dimension consists of 11 items each addressing the effect of pain on various activities in the past 4 weeks and each assessed on a 5-point scale (0=Never through to 4=Always). Scaled score was equalled to total of raw score of each item in scale divided by the maximum possible raw score of all the items in the dimension, multiplied by 100, resulting in a score on a scale from 0 (best possible health status) to 100 (worst possible health status).

The relevant time points are Baseline and Week 12.

Percentage of Subjects With an Endometriosis Severity Score of "Severe" at Week 12Up to week 12

Subject was asked monthly on electronic diary to assess their impression of endometriosis severity, considering the preceding 4-weeks, with following possible answers: no symptoms, very mild, mild, moderate, severe. This question was programmed to raise automatically every 4 weeks on the subject electronic diary. Result reported here is the percentage of subjects who answered "severe" at week 12.

Change From Baseline to Week 12 in the Difficulty in Doing Daily Activities Mean ScoreFrom baseline to week 12

This endpoint corresponds to the change from baseline to Week 12 in the mean of daily scores for "difficulty in doing daily activities", assessed via electronic diary during the preceding 28 days (4-week period), on a Numerical Rating Scale (NRS) of 0 (no difficulty doing daily activities) to 10 (unable to do daily activities). The baseline mean score was calculated as the mean of daily scores recorded in electronic diary over the two complete menstrual cycles performed during the screening period.

The relevant time points are Baseline and Week 12.

Change From Baseline to Week 24 in Endometrial Thickness Measured by Transvaginal Ultrasound (TVUS)From baseline up to week 24

The endometrium thickness was measured by TVUS at screening and at Week 24 visit by the gynaecologist and result was recorded in mm. This endpoint reports the changes from baseline to Week 24 in the endometrial thickness.

Percentage Change From Baseline to Week 24 in Clinical Laboratory Assessments: HDLFrom Baseline up to week 24

This endpoint reports the change from baseline to week 24 in clinical laboratory assessments: HDL cholesterol.

Percentage Change From Baseline to Week 24 in Clinical Laboratory Assessments: TriglyceridesFrom baseline up to week 24

This endpoint reports the change from baseline to week 24 in clinical laboratory assessments: triglycerides.

Trial Locations

Locations (86)

Site reference ID 418

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Clearwater, Florida, United States

Site reference ID 471

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Fall River, Massachusetts, United States

Site reference ID 462

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Arcadia, California, United States

Site reference ID 405

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Chino, California, United States

Site reference ID 440

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Tustin, California, United States

Site reference ID 437

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Gainesville, Florida, United States

Site reference ID 420

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Miami Lakes, Florida, United States

Site reference ID 458

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Jensen Beach, Florida, United States

Site reference ID 441

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Miami Springs, Florida, United States

Site reference ID 435

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Miami, Florida, United States

Site reference ID 442

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Wellington, Florida, United States

Site reference ID 475

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Nampa, Idaho, United States

Site reference ID 465

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Oak Brook, Illinois, United States

Site reference ID 404

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Shawnee Mission, Kansas, United States

Site reference ID 430

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Ann Arbor, Michigan, United States

Site reference ID 409

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Bay City, Michigan, United States

Site reference ID 466

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New York, New York, United States

Site reference ID 408

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Bristol, Tennessee, United States

Site reference ID 414

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Westerville, Ohio, United States

Site reference ID 449

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Jenkintown, Pennsylvania, United States

Site reference ID 476

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Columbia, South Carolina, United States

Site reference ID 452

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Austin, Texas, United States

Site reference ID 461

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Beaumont, Texas, United States

Site reference ID 460

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Dallas, Texas, United States

Site reference ID 464

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Fort Worth, Texas, United States

Site reference ID 422

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Draper, Utah, United States

Site reference ID 467

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Centreville, Virginia, United States

Site reference ID 432

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Webster, Texas, United States

Site reference ID 407

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Norfolk, Virginia, United States

Site reference ID 412

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Richmond, Virginia, United States

Site reference ID 417

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Richmond, Virginia, United States

Site reference ID 101

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Katowice, Poland

Site reference ID 102

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Katowice, Poland

Site reference ID 103

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Szczecin, Poland

Site reference ID 202

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Moscow, Russian Federation

Site reference ID 201

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Moscow, Russian Federation

Site reference ID 204

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Moscow, Russian Federation

Site reference ID 203

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Moscow, Russian Federation

Site reefrence ID 303

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Kyiv, Ukraine

SIte reference ID 304

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Kyiv, Ukraine

Site reference ID 411

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Miami, Florida, United States

Site reference ID 428

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Atlanta, Georgia, United States

Site reference ID 413

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Houston, Texas, United States

Site reference ID 479

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Houston, Texas, United States

Site reference ID 451

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San Antonio, Texas, United States

Site reference ID 426

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Tampa, Florida, United States

Site reference ID 439

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Scottsdale, Arizona, United States

Site reference ID 463

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Huntington Park, California, United States

Site reference ID 474

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Denver, Colorado, United States

Site reference ID 450

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Lakewood, Colorado, United States

Site reference ID 425

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Longmont, Colorado, United States

Site reference ID 457

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Boca Raton, Florida, United States

Site reference ID 424

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Miami, Florida, United States

Site reference ID 423

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New Port Richey, Florida, United States

Site reference ID 459

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Atlanta, Georgia, United States

Site reference ID 456

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Wichita, Kansas, United States

Site reference ID 453

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Metairie, Louisiana, United States

Site reference ID 478

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Glen Burnie, Maryland, United States

Site reference ID 445

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Fall River, Massachusetts, United States

Site reference ID 436

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Greensboro, North Carolina, United States

Site reference ID 433

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Morehead City, North Carolina, United States

Site reference ID 443

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Dayton, Ohio, United States

Site reference ID 472

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Franklin, Ohio, United States

Site reference ID 419

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Bryn Mawr, Pennsylvania, United States

Site reference ID 403

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Chattanooga, Tennessee, United States

Site reference ID 429

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Nashville, Tennessee, United States

Site reference ID 447

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Dallas, Texas, United States

Site reference ID 434

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Houston, Texas, United States

Site reference ID 402

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Schertz, Texas, United States

Site reference ID 406

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Seattle, Washington, United States

Site reference ID 105

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Lublin, Poland

Site reference ID 205

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Saint Petersburg, Russian Federation

Site refenrec ID 469

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Northridge, California, United States

Site reference ID 431

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San Diego, California, United States

Site reference ID 427

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Southfield, Michigan, United States

Site reference ID 473

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Saginaw, Michigan, United States

Site reference ID 421

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Albuquerque, New Mexico, United States

Site reference ID 415

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Tiffin, Ohio, United States

Site reference ID 410

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Washington, District of Columbia, United States

Site reference ID 104

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Lublin, Poland

Site reference ID 455

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Chandler, Arizona, United States

Site reference ID 454

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Marrero, Louisiana, United States

Site reference ID 302

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Kyiv, Ukraine

Site reference ID 305

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Ivano-Frankivs'k, Ukraine

Site reference ID 301

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Kyiv, Ukraine

Site reference ID 468

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Saginaw, Michigan, United States

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