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.
Tutkimuksen yleiskatsaus
Tila
Tila
Ehdot
Ehdot
Interventio / Hoito
Interventio / Hoito
Yksityiskohtainen kuvaus
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.
Opintotyyppi
Opintotyyppi
Ilmoittautuminen (Arvioitu)
Ilmoittautuminen
Vaihe
Vaihe
- Vaihe 2
Osallistumiskriteerit
Kelpoisuusvaatimukset
Kelpoisuusvaatimukset
Opintokelpoiset iät
Hyväksyy terveitä vapaaehtoisia
Sukupuolet, jotka voivat opiskella
Kuvaus
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
-
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)
Opintosuunnitelma
Miten tutkimus on suunniteltu?
Suunnittelun yksityiskohdat
- Ensisijainen käyttötarkoitus: Hoito
- Jako: Satunnaistettu
- Inventiomalli: Rinnakkaistehtävä
- Naamiointi: Kolminkertaistaa
Aseiden lukumäärä
Aseet ja interventiot
Osallistujaryhmä / ArmOsallistujaryhmä / Arm |
Interventio / HoitoInterventio / Hoito |
|---|---|
|
Active Comparator: 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 |
|
Active Comparator: 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 |
|
Active Comparator: 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 |
|
Placebo Comparator: Teverelix DP placebo SC
|
Single dose at Day 1 of placebo injection SC. |
Mitä tutkimuksessa mitataan?
Ensisijaiset tulostoimenpiteet
Ensisijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
|---|---|---|
|
Percentage of Participants With Serum Estradiol Maintained Within 20-50 pg/mL for at Least 14 Consecutive Days
Aikaikkuna: 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
|
Toissijaiset tulostoimenpiteet
Toissijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of Teverelix
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
|
Yhteistyökumppanit ja tutkijat
Sponsori
Sponsori
Opintojen ennätyspäivät
Opi tärkeimmät päivämäärät
Opiskelun aloitus (Arvioitu)
Opiskelun aloitus
Ensisijainen valmistuminen (Arvioitu)
Ensisijainen valmistuminen
Opintojen valmistuminen (Arvioitu)
Opintojen valmistuminen
Opintoihin ilmoittautumispäivät
Ensimmäinen lähetetty
Ensimmäinen lähetetty
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen Lähetetty (Todellinen)
Ensimmäinen Lähetetty
Tutkimustietojen päivitykset
Viimeisin päivitys julkaistu (Todellinen)
Viimeisin päivitys julkaistu
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeksi vahvistettu
Viimeksi vahvistettu
Lisää tietoa
Tähän tutkimukseen liittyvät termit
Avainsanat
Muita asiaankuuluvia MeSH-ehtoja
- Urogenitaaliset sairaudet
- Sukuelinten sairaudet
- Kipu
- Neurologiset ilmenemismuodot
- Mielenterveyshäiriöt
- Patologiset prosessit
- Sukuelinten sairaudet, mies
- Miesten urogenitaaliset sairaudet
- Naisten virtsa- ja sukupuolielinten sairaudet
- Naisten virtsa- ja sukupuolielinten sairaudet ja raskauden komplikaatiot
- Sukuelinten sairaudet, naiset
- Seksuaalinen toimintahäiriö, fysiologinen
- Seksuaaliset häiriöt, psykologiset
- Lantiokipu
- Kuukautiskiertohäiriöt
- Patologiset tilat, merkit ja oireet
- Merkit ja oireet
- Sairaus
- Hedelmättömyys
- Endometrioosi
- Dyspareunia
- Dysmenorrea
Muut tutkimustunnusnumerot
Muut tutkimustunnusnumerot
- ANT-3111-01
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