PRECISION-E2: Teverelix in Women With Endometriosis - Pharmacodynamics, Safety and Precision Medicine Correlates
PRECISION-E2 Study is a Prospective Genomic Guided Precision Medicine Phase IIa, Randomized, Placebo Controlled Clinical Study in Women With Moderate to Severe Pain Associated With Symptomatic Endometriosis in UAE.
PRECISION-E2 is a Phase IIa randomized, placebo-controlled study evaluating the pharmacodynamic profile, safety and tolerability of teverelix in premenopausal women with moderate-to-severe pain associated with endometriosis in the United Arab Emirates. The study will characterize the magnitude and duration of estradiol suppression following a single administration of teverelix and evaluate dose and route of administration, including subcutaneous and intramuscular administration, to inform subsequent clinical development.
The study also incorporates a prospective precision medicine component designed to explore associations between genomic variation and pharmacodynamic and clinical response to teverelix. In participants enrolled in the Emirati Genome Program, genomic data will be integrated with hormonal, pharmacokinetic and clinical outcomes to investigate potential biological determinants of treatment response.
調査の概要
状態
詳細な説明
PRECISION-E2 is designed as an early clinical development and precision medicine study to characterize the pharmacodynamic response to long-acting injectable teverelix in women with symptomatic endometriosis and to inform dose and route selection for subsequent development. Participants will be randomized to receive a single administration of teverelix by the subcutaneous or intramuscular route or placebo. Serial pharmacodynamic and pharmacokinetic assessments will characterize the onset, magnitude and duration of gonadotropin and estradiol suppression.
The study will evaluate whether teverelix can achieve sustained estradiol concentrations within a prespecified therapeutic range while characterizing safety, tolerability and preliminary changes in endometriosis-associated symptoms and patient-reported outcomes.
A prospective precision medicine component will leverage the genomic infrastructure available in the United Arab Emirates to explore biological determinants of interindividual variability in pharmacodynamic and clinical response. Where available and appropriately consented, genomic information will be integrated with pharmacokinetic, hormonal and clinical data to generate hypotheses for subsequent biomarker-informed development of teverelix.
Teverelix TFA is a long-acting gonadotropin-releasing hormone (GnRH) antagonist developed as an injectable sustained-release formulation. Sustained exposure is driven primarily by depot release, supporting evaluation of infrequent dosing strategies and alternative routes of administration and provides the rationale for investigating whether controlled estradiol suppression can be achieved and maintained within a range intended to balance therapeutic activity with the consequences of excessive estrogen suppression.This pharmacologic profile differentiates Teverelix from both daily oral GnRH antagonists and injectable GnRH agonists and supports evaluation in estrogen-dependent conditions such as endometriosis.
研究の種類
入学 (推定)
段階
- フェーズ2
参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Inclusion Criteria:
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Female between the ages of 19 and 50 (premenopausal)
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Has given written consent
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Clinical diagnosis of endometriosis (laparoscopy or imaging) performed within 5 years of Screening
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The participant must have dysmenorrhea or pelvic pain associated with endometriosis, of which either one or both should be at least "moderate" as determined by the investigator or sub-investigator using the B&B scale.
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The participant must have maximum VAS score > 30 for pelvic pain associated with endometriosis
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The participant has experienced a regular menstrual cycle (25 to 38 days) that should include menstrual bleeding of at least 3 consecutive days (at least 3 regular menstrual cycles to be confirmed)
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Agrees to practice contraception, which does not include any form of hormonal contraception during the entire study period:
- Either by using double barrier contraception and in compliance with local guidelines,
- or, is truly sexually abstinent, when this is in line with the preferred and usual lifestyle of the patient
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Is not expected to undergo gynaecological surgery or other surgical procedures for treatment of endometriosis (including ablation, shaving, or excision) during the study, including during the Follow-Up Period, and the participant does not desire such treatment during this time frame.
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Agree to continue to use only the analgesic medication permitted by the protocol during the Treatment and Follow-up Periods
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For patients who already participate or are willing to participate in the Emirati Genome Program (EGP, willing to participate in the Pharmacogenomic Study
Exclusion Criteria:
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Subject is pregnant or breast feeding or is planning a pregnancy within the next 24 months or is less than 6 months postpartum, post-abortion, or post-pregnancy at the time of entry into the Screening Period.
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Subject has breast fed in the previous six months
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Subject has a history of previous non-response to Gonadotropin-releasing hormone (GnRH) agonists, GnRH antagonists, Depot Medroxyprogesterone Acetate, or aromatase inhibitors as assessed by subject report of no improvement in dysmenorrhea or non-menstrual pelvic pain (subject report of partial response to or side effects from these agents is not exclusionary).
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Subject has chronic pelvic pain that is not caused by endometriosis that requires chronic analgesic or other chronic therapy, or that would interfere with the assessment of endometriosis related pain.
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Known clinically significant gynaecologic condition, PID, abnormal genital bleeding or uterine anomaly
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Bone mineral density by dual energy x-ray absorptiometry Z score of less than -2 at the lumbar spine, total hip or femoral neck
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Subject has a history of osteoporosis or other metabolic bone disease
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BMI <17 or > 35
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Participated in another investigational study within 3 months before recruitment
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Has abnormal screening and/or baseline laboratory values that suggest a clinically significant underlying disease, or the following laboratory values:
- Liver function test (aspartate aminotransferase [ASAT/SGOT], alanine aminotransferase [ALAT/SGPT]), exceeding >2X the upper limit of the normal (ULN) range
- Total bilirubin exceeding >1.5X the upper limit of the normal (ULN) range
- An estimated glomerular filtration rate (eGFR) < 30 mL/min, based on creatinine clearance calculation by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation normalised to an average surface area of 1.73m2, at the screening visit.
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Has congenital long QT syndrome or ECG abnormalities at screening
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Use of the following medications prior to and during study participation:
- Any other IMP (within 3 months of enrolment)
- Herbal medications known to have anti-androgenic effects
- Bethanechol chloride
- Any other medication or herbal product that may affect hormone levels and might, therefore, confound interpretation of the study results (e.g. St. John's wort) (within 3 months of enrolment)
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:トリプル
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
アクティブコンパレータ:Teverelix DP 90 mg IM
|
Single dose at Day 1 of 60 mg injection SC Single dose at Day 1 of 90 mg injection SC. Single dose at Day 1 of 90 mg injection IM |
|
アクティブコンパレータ:Teverelix DP 60 mg SC
|
Single dose at Day 1 of 60 mg injection SC Single dose at Day 1 of 90 mg injection SC. Single dose at Day 1 of 90 mg injection IM |
|
アクティブコンパレータ:Teverelix DP 90 mg SC
|
Single dose at Day 1 of 60 mg injection SC Single dose at Day 1 of 90 mg injection SC. Single dose at Day 1 of 90 mg injection IM |
|
プラセボコンパレーター:Teverelix DP placebo SC
|
Single dose at Day 1 of placebo injection SC. |
この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Percentage of Participants With Serum Estradiol Maintained Within 20-50 pg/mL for at Least 14 Consecutive Days
時間枠:From Day 1 to Day 197
|
Serum estradiol (E2) will be measured using a validated assay at the central laboratory at baseline, Days 1, 2, 8, 15, 22 and 29, and monthly through Day 197. A participant will be classed as a responder if E2 remains within 20-50 pg/mL inclusive (the estradiol therapeutic window described by Barbieri) for at least 14 consecutive days, as derived according to the Statistical Analysis Plan. The number and percentage of responders will be reported by treatment arm. |
From Day 1 to Day 197
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of Teverelix
時間枠:Day 1 (pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Cmax (ng/mL) derived by non-compartmental analysis of plasma teverelix concentrations, measured using a validated analytical method at a specialised laboratory, following a single SC or IM dose of Teverelix DP. Reported by treatment arm. |
Day 1 (pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Time to Maximum Observed Plasma Concentration (Tmax) of Teverelix
時間枠:Day 1 (pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Tmax (hours) derived by non-compartmental analysis of plasma teverelix concentrations, measured using a validated analytical method at a specialised laboratory, following a single SC or IM dose of Teverelix DP. Reported by treatment arm. |
Day 1 (pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Area Under the Plasma Concentration-Time Curve From Time Zero to the Last Quantifiable Concentration (AUC0-t) of Teverelix
時間枠:Day 1 (pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
AUC0-t derived by non-compartmental analysis of plasma teverelix concentrations, measured using a validated analytical method at a specialised laboratory, following a single SC or IM dose of Teverelix DP. Reported by treatment arm. |
Day 1 (pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Change From Baseline in Serum Estradiol Concentration
時間枠:Baseline (Day 1 pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Serum estradiol (pg/mL) measured using a validated assay at the central laboratory. Baseline is the Day 1 pre-dose value. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Change From Baseline in Serum Luteinising Hormone (LH) Concentration
時間枠:Baseline (Day 1 pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Serum LH (IU/L) measured using a validated assay at the central laboratory. Baseline is the Day 1 pre-dose value. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Change From Baseline in Serum Follicle-Stimulating Hormone (FSH) Concentration
時間枠:Baseline (Day 1 pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Serum FSH (IU/L) measured using a validated assay at the central laboratory. Baseline is the Day 1 pre-dose value. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 2, 8, 15, 22, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Duration of Serum Estradiol Within 20-50 pg/mL
時間枠:Day 1 to Day 197
|
Number of days from the first to the last consecutive serum estradiol value within 20-50 pg/mL during the treatment period, measured using a validated assay at the central laboratory and derived according to the Statistical Analysis Plan. Reported by treatment arm |
Day 1 to Day 197
|
|
Percent Change From Baseline in Serum Bone-Specific Alkaline Phosphatase (BAP)
時間枠:Baseline (Day 1 pre-dose) and Days 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Serum BAP concentrations (ng/mL) measured using a validated assay at the central laboratory. Percent change from baseline is calculated for each post-baseline visit, where baseline is the Day 1 pre-dose value. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Percent Change From Baseline in Urinary N-Telopeptide of Type I Collagen (NTx)
時間枠:Baseline (Day 1 pre-dose) and Days 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Urinary NTx (nmol BCE/mmol creatinine) measured using a validated assay at the central laboratory. Percent change from baseline is calculated for each post-baseline visit, where baseline is the Day 1 pre-dose value. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
時間枠:Day 1 through Day 365
|
Adverse events will be coded using MedDRA and severity graded according to CTCAE v6.0. Reported by treatment arm. |
Day 1 through Day 365
|
|
Number of Participants with Serious Adverse Events (SAEs)
時間枠:Day 1 through Day 365
|
Serious adverse events coded using MedDRA. Reported by treatment arm |
Day 1 through Day 365
|
|
Number of Participants with Clinically Significant Abnormal Laboratory Values
時間枠:Baseline (Day 1 pre-dose) and Days 15, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Fasted haematology, biochemistry and coagulation tests and urinalysis, with abnormal values judged clinically significant by the investigator. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 15, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Number of Participants with Clinically Significant 12-Lead ECG Abnormalities
時間枠:Baseline (Day 1 pre-dose) and Days 15, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Triplicate 12-lead ECGs, with findings judged clinically significant by the investigator. On Day 1, ECGs are recorded pre-dose and 2 hours post-dose. Reported by treatment arm. |
Baseline (Day 1 pre-dose) and Days 15, 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
|
Change From Baseline in Dysmenorrhoea Score on the Biberoglu and Behrman (B&B) Scale
時間枠:Baseline (Day 1) and Days 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
Dysmenorrhoea severity rated using the patient symptom component of the B&B scale (0 = absent to 3 = severe); higher scores indicate more severe symptoms. Reported by treatment arm. |
Baseline (Day 1) and Days 29, 57, 85, 113, 141, 169, 197, 281 and 365
|
協力者と研究者
スポンサー
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- ANT-3111-01
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
米国で製造され、米国から輸出された製品。
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