A Study to Find Out if Fezolinetant Helps Reduce Moderate to Severe Hot Flashes in Women Going Through Menopause - 2

NCT ID: NCT04003142

Last Updated: 2024-11-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

501 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-07-10

Study Completion Date

2021-04-23

Brief Summary

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This study was for women in menopause with moderate to severe hot flashes. Menopause, a normal part of aging, is the time of a woman's last period. Hot flashes can interrupt a woman's daily life.

The study treatments are fezolinetant 30 mg (1 tablet of fezolinetant and 1 placebo tablet) once a day, fezolinetant 45 mg (2 tablets of fezolinetant) once a day or placebo (2 tablets) once a day. (Placebo is a dummy treatment that looks like medicine but does not have any medicine in it.) The study compared fezolinetant and placebo after 4 and 12 weeks of dosing. The study evaluated if fezolinetant reduces the number of hot flashes and the study evaluated if fezolinetant reduces the severity of the hot flashes.

Women in the study received an electronic handheld device at the first study visit. (It is similar to a smart phone.) Each day of the study, study participants used this to record their hot flashes. Their record for the 10 days before the start of study treatment was checked. They remained in the study if their record shows 7 or 8 moderate to severe hot flashes per day (50 or more per week). Next, they were picked for 1 of the 2 study treatments (fezolinetant or placebo) by chance alone. It is like flipping a coin.

The study participants took study treatment for 52 weeks. The first 12 weeks of study treatment was "double-blinded." That means that the study participants and the study doctors did not know who took which of the study treatments (fezolinetant 30 mg, fezolinetant 45 mg or placebo) during that time. The last 40 weeks of study treatment was "noncontrolled." That means that each study participant and the study doctors knew which study treatment that study participant took during that time. Women who took fezolinetant during the first 12 weeks continued to take the same dose. Women who took placebo during the first 12 weeks took fezolinetant. Their dose was either 30 mg or 45 mg fezolinetant.

At weeks 2, 4, 8, 12, 14, 16 and then once a month, the study participants went to the hospital or clinic for a check-up. They were asked about medications, side effects and how they felt. Other checks included physical exam and vital signs (heart rate, temperature and blood pressure). Blood and urine was collected for laboratory tests. Study participants completed questionnaires that were about how hot flashes affect their daily life. Study participants who had their uterus had the following 2 tests done at the first and last study visits. One of the 2 tests was endometrial biopsy. This test involved removing a small amount of tissue from the inside lining of the uterus. The tissue was then checked under a microscope. The other test was transvaginal ultrasound. This test used sound waves to create pictures of the organs in the pelvis. The sound waves are transmitted by a probe (transducer), which was placed inside the vagina. Study participants might have a screening mammogram done at the first and/or last study visit. A mammogram is an x-ray picture of the breasts used to screen for breast cancer. Study participants who did not have this test done in the last 12 months had it done at the first study visit. They had done at the last study visit if they were due for their screening mammogram and their own doctor agrees.

The last check-up at the hospital or clinic was 3 weeks after the last dose of study treatment.

Detailed Description

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This study consisted of a screening period and a 52 week treatment period. Safety follow up occurred 3 weeks after the last dose of study drug.

Conditions

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Hot Flashes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Double-blind Period: Fezolinetant 30 mg/Extension Period: Fezolinetant 30 mg

Participants received fezolinetant 30 mg (one 30 mg fezolinetant tablet and one placebo tablet) orally, QD up to week 12 during double-blind treatment period followed by fezolinetant 30 mg orally, QD from week 13 up to Week 52 during extension treatment period.

Group Type EXPERIMENTAL

Fezolinetant

Intervention Type DRUG

Oral tablet

Double-blind Period: Fezolinetant 45 mg/Extension Period: Fezolinetant 45 mg

Participants received fezolinetant 45 mg (one 30 mg tablet and one 15 mg tablet) orally, QD up to week 12 during double-blind treatment period followed by fezolinetant 45 mg orally, QD from week 13 up to Week 52 during extension treatment period.

Group Type EXPERIMENTAL

Fezolinetant

Intervention Type DRUG

Oral tablet

Double-blind Period: Placebo

Participants received fezolinetant matching placebo (two fezolinetant matching placebo tablets) orally, once daily (QD) up to week 12 during double-blind treatment period.

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

Oral Tablet

Double-blind Period: Placebo/Extension Period: Fezolinetant 30 mg

Participants who received placebo during double-blind treatment period were re-randomized to receive fezolinetant 30 mg orally, QD from week 13 up to week 52 during extension treatment period.

Group Type EXPERIMENTAL

Fezolinetant

Intervention Type DRUG

Oral tablet

Double-blind Period: Placebo/Extension Period: Fezolinetant 45 mg

Participants who received placebo during double-blind treatment period were re-randomized to receive fezolinetant 45 mg orally, QD from week 13 up to week 52 during extension treatment period.

Group Type EXPERIMENTAL

Fezolinetant

Intervention Type DRUG

Oral tablet

Interventions

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Fezolinetant

Oral tablet

Intervention Type DRUG

placebo

Oral Tablet

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subject has a body mass index ≥ 18 kg/m\^2 and ≤ 38 kg/m\^2.
* Subject must be seeking treatment or relief for vasomotor symptoms (VMS) associated with menopause and confirmed as menopausal per 1 of the following criteria at the screening visit:

* Spontaneous amenorrhea for ≥ 12 consecutive months
* Spontaneous amenorrhea for ≥ 6 months with biochemical criteria of menopause (follicle-stimulating hormone \[FSH\] \> 40 IU/L); or
* Having had bilateral oophorectomy ≥ 6 weeks prior to the screening visit.
* Within the 10 days prior to randomization, subject must have a minimum average of 7 to 8 moderate to severe hot flashes (HFs) vasomotor symptoms (VMS) per day, or 50 to 60 per week.
* Subject is in good general health as determined on the basis of medical history and general physical examination, including a bimanual clinical pelvic examination and clinical breast examination devoid of relevant clinical findings, performed at the screening visit; hematology and biochemistry parameters, pulse rate and/or blood pressure and electrocardiogram (ECG) within the reference range for the population studied, or showing no clinically relevant deviations.
* Subject has documentation of a normal/negative or no clinically significant findings mammogram (obtained at screening or within the prior 12 months of study enrollment). Appropriate documentation includes a written report or an electronic report indicating normal/negative or no clinically significant mammographic findings.
* Subject is willing to undergo a transvaginal ultrasound (TVU) to evaluate the uterus and ovaries at screening and at week 52 end of treatment (EOT), and for subjects who are withdrawn from the study prior to completion, a TVU at the early discontinuation (ED) visit.
* Subject is willing to undergo an endometrial biopsy at screening and at week 52 (EOT), for subjects with uterine bleeding, and for subjects who are withdrawn from the study prior to completion. The endometrial biopsy obtained at screening must be considered evaluable.
* Subject has documentation of a normal or not clinically significant Papanicolaou (Pap) test (or equivalent cervical cytology) within the previous 12 months or at screening.
* Subject has a negative urine pregnancy test at screening.
* Subject has a negative serology panel (i.e. negative hepatitis B surface antigen, negative hepatitis C virus antibody and negative human immunodeficiency virus antibody screens) at screening.
* Subject agrees not to participate in another interventional study while participating in the present study.

Exclusion Criteria

* Subject uses a prohibited therapy (strong or moderate cytochrome P450 1A2 \[CYP1A2\] inhibitors, hormone replacement therapy \[HRT\], hormonal contraceptive or any treatment for VMS \[prescription, over the counter or herbal\]) or is not willing to wash out and discontinue use of such drugs for the full duration of study conduct.
* Subject has known substance abuse or alcohol addiction within 6 months of screening.
* Subject has previous or current history of a malignant tumor, except for basal cell carcinoma.
* Subject's systolic blood pressure is ≥ 130 mmHg or diastolic blood pressure is ≥ 80 mmHg based on the average of 2 to 3 readings, on at least 2 different occasions within the screening period.

* Subjects who do not meet these criteria may be re-assessed after initiation or review of antihypertensive measures.
* Subjects with a medical history of hypertension can be enrolled once they are medically clear (stable and compliant).
* Subject has history of severe allergy, hypersensitivity or intolerance to drugs in general, including the study drug and any of its excipients.
* Subject has an unacceptable result from the TVU assessment at screening (i.e., full length of endometrial cavity cannot be visualized or presence of a clinically significant finding).
* Subject has an endometrial biopsy confirming presence of disordered proliferative endometrium, endometrial hyperplasia, endometrial cancer or other clinically significant findings at screening.
* Subject has a history within the last 6 months of undiagnosed uterine bleeding.
* Subject has a history of seizures or other convulsive disorders.
* Subject has a medical condition or chronic disease (including history of neurological \[including cognitive\], hepatic, renal, cardiovascular, gastrointestinal, pulmonary \[e.g., moderate asthma\], endocrine or gynecological disease) or malignancy that could confound interpretation of the study outcome.
* Subject has active liver disease, jaundice or elevated liver aminotransferases (alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\]), elevated total or direct bilirubin, elevated international normalized ratio (INR), or elevated alkaline phosphatase (ALP). Patients with mildly elevated ALT or AST up to 1.5 times the upper limit of normal (ULN) can be enrolled if total and direct bilirubin are normal. Patients with mildly elevated ALP (up to 1.5 x ULN) can be enrolled if cholestatic liver disease is excluded and no cause other than fatty liver is diagnosed. Patients with Gilbert's syndrome with elevated total bilirubin may be enrolled as long as direct bilirubin, hemoglobin and reticulocytes are normal.
* Subject has creatinine \> 1.5 × ULN; or estimated glomerular filtration rate (eGFR) using the Modification of Diet in Renal Disease formula ≤ 59 mL/min per 1.73 m\^2 at screening.
* Subject has a history of suicide attempt or suicidal behavior within the last 12 months or has suicidal ideation within the last 12 months (a response of "yes" to question 4 or 5 on the suicidal ideation portion of the Columbia Suicide Severity Rating Scale \[C-SSRS\]), or who is at significant risk to commit suicide at screening and at randomization.
* Subject has previously been enrolled in a clinical trial with fezolinetant.
* Subject is participating concurrently in another interventional study or participated in an interventional study within 28 days prior to screening, or received any investigational drug within 28 days or within 5 half-lives prior to screening, whichever is longer.
* Subject is unable or unwilling to complete the study procedures.
* Subject has any condition which makes the subject unsuitable for study participation.
* Subject has had partial or full hysterectomy.
Minimum Eligible Age

40 Years

Maximum Eligible Age

65 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Astellas Pharma Global Development, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Executive Medical Director

Role: STUDY_DIRECTOR

Astellas Pharma Global Development, Inc.

Locations

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Mesa Obstetricians and Gynecologists

Mesa, Arizona, United States

Site Status

Precision Trials

Phoenix, Arizona, United States

Site Status

Visions Clinical Research - Tuscon

Tucson, Arizona, United States

Site Status

Excell Research

Oceanside, California, United States

Site Status

Dream Team Clinical Research, LLC

Pomona, California, United States

Site Status

Clinical Trials Research

Sacramento, California, United States

Site Status

Wake Research Associates, LLC

San Diego, California, United States

Site Status

Women's Healthcare Affiliates

San Diego, California, United States

Site Status

Bayview Research Group

Valley Village, California, United States

Site Status

Downtown Women's Health Care

Denver, Colorado, United States

Site Status

Horizons Clincial Research Center LLC

Denver, Colorado, United States

Site Status

Physicians' Research Options/Red Rocks OB/GYN

Lakewood, Colorado, United States

Site Status

Helix Biomedics

Boynton Beach, Florida, United States

Site Status

Renaissance Research and Medical Group, Inc.

Cape Coral, Florida, United States

Site Status

Nature Coast Clinical Research

Crystal River, Florida, United States

Site Status

Bioclinica Research, Melbourne

Melbourne, Florida, United States

Site Status

LCC Medical Research Institute, LLC

Miami, Florida, United States

Site Status

Suncoast Clinical Research, Inc.

Miami, Florida, United States

Site Status

Suncoast Research

Miami, Florida, United States

Site Status

Medical Health Center & Research

Miami, Florida, United States

Site Status

Healthcare Clinical Data Inc

North Miami, Florida, United States

Site Status

Sensible Healthcare LLC

Ocoee, Florida, United States

Site Status

Bioclinica Research

Orlando, Florida, United States

Site Status

Ormond Medical Arts Pharmaceutical Research Center

Ormond Beach, Florida, United States

Site Status

Progressive Medical Research

Port Orange, Florida, United States

Site Status

Physician Care Clinical Research, LLC

Sarasota, Florida, United States

Site Status

Meridien Research

St. Petersburg, Florida, United States

Site Status

GCP Clinical Research, LLC

Tampa, Florida, United States

Site Status

Comprehensive Clinical Development

West Palm Beach, Florida, United States

Site Status

Clinical Research of Central Florida

Winter Haven, Florida, United States

Site Status

Georgia Research for Women

Atlanta, Georgia, United States

Site Status

Agile Clinical Research Trials, LLC

Atlanta, Georgia, United States

Site Status

iResearch Atlanta LLC

Decatur, Georgia, United States

Site Status

WR-Mount Vernon Clinical Research

Sandy Springs, Georgia, United States

Site Status

Georgia Clinical Research

Snellville, Georgia, United States

Site Status

Elite Clinical Trials

Blackfoot, Idaho, United States

Site Status

ASR, LLC-Advanced Specialty Research

Nampa, Idaho, United States

Site Status

Affinity Clinical Research Institute

Oak Brook, Illinois, United States

Site Status

Cypress Medical Research Center

Wichita, Kansas, United States

Site Status

Praetorian Pharmaceutical Research

Marrero, Louisiana, United States

Site Status

Southern Clinical Research Associates

Metairie, Louisiana, United States

Site Status

Women Under Study, LLC

New Orleans, Louisiana, United States

Site Status

Pharmasite Research Inc

Baltimore, Maryland, United States

Site Status

Clinical Research Center of Nevada (CRCN)

Las Vegas, Nevada, United States

Site Status

Dr.R. Garn Mabey, MD,Office Of

Las Vegas, Nevada, United States

Site Status

Hassman Research Institute, LLC

Berlin, New Jersey, United States

Site Status

Albuquerque Clinical Trials, Inc.

Albuquerque, New Mexico, United States

Site Status

Bosque Women's Care

Albuquerque, New Mexico, United States

Site Status

Circuit Clinical

West Seneca, New York, United States

Site Status

Premier Gynecology & Wellness

Charlotte, North Carolina, United States

Site Status

Medication Management, LLC

Greensboro, North Carolina, United States

Site Status

Hwc Women's Research Center

Englewood, Ohio, United States

Site Status

OB/GYN Associates of Erie

Erie, Pennsylvania, United States

Site Status

Dr. Marvin Kalafer MD, Office Of

Jenkintown, Pennsylvania, United States

Site Status

Research Protocol Management Specialists

Pittsburgh, Pennsylvania, United States

Site Status

Clinical Neuroscience Solutions, Inc

Memphis, Tennessee, United States

Site Status

The Clinical Research Center, LLC

Carrollton, Texas, United States

Site Status

Advances in Health

Houston, Texas, United States

Site Status

Centex Studies, Inc.

Houston, Texas, United States

Site Status

FMC Science

Lampasas, Texas, United States

Site Status

ClinRx Research

Plano, Texas, United States

Site Status

Granger Medical Clinic

Riverton, Utah, United States

Site Status

Wasatch Clinical Research, LLC

Salt Lake City, Utah, United States

Site Status

Seattle Women's: Health, Research, Gynecology

Seattle, Washington, United States

Site Status

North Spokane Women's Health

Spokane, Washington, United States

Site Status

Site CA15006

Sarnia, Ontario, Canada

Site Status

Site CA15009

Toronto, Ontario, Canada

Site Status

Site CA15007

Lévis, Quebec, Canada

Site Status

Site CA15002

Sherbrooke, Quebec, Canada

Site Status

Site CA15001

Victoriaville, Quebec, Canada

Site Status

Site CA15008

Québec, , Canada

Site Status

Site CZ42007

Písek, , Czechia

Site Status

Site CZ42006

Prague, , Czechia

Site Status

Site CZ42004

Prague, , Czechia

Site Status

Site CZ42005

Tábor, , Czechia

Site Status

Site CZ42003

Vsetín, , Czechia

Site Status

Site LV37101

Riga, , Latvia

Site Status

Site LV37103

Riga, , Latvia

Site Status

Site PL48001

Bydgoszcz, , Poland

Site Status

Site PL48013

Elblag, , Poland

Site Status

Site PL48009

Katowice, , Poland

Site Status

Site PL48011

Krakow, , Poland

Site Status

Site PL48002

Lublin, , Poland

Site Status

Site PL48012

Lublin, , Poland

Site Status

Site PL48004

Piaseczno, , Poland

Site Status

Site PL48006

Poznan, , Poland

Site Status

Site PL48005

Szczecin, , Poland

Site Status

Site PL48010

Warsaw, , Poland

Site Status

Site PL48003

Warsaw, , Poland

Site Status

Site ES34003

Aravaca, , Spain

Site Status

Site ES34002

Centelles, , Spain

Site Status

Site ES34001

Madrid, , Spain

Site Status

Site GB44001

Shipley, , United Kingdom

Site Status

Countries

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United States Canada Czechia Latvia Poland Spain United Kingdom

References

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Santoro N, Neal-Perry G, Stute P, Blogg M, Mancuso S, Morga A, Ottery FD, Siddiqui E. Fezolinetant effect on vasomotor symptoms due to menopause in women unsuitable for hormone therapy. Curr Med Res Opin. 2025 Feb;41(2):375-384. doi: 10.1080/03007995.2025.2470752. Epub 2025 Mar 5.

Reference Type DERIVED
PMID: 40044127 (View on PubMed)

Kagan R, Cano A, Nappi RE, English ML, Mancuso S, Wu X, Ottery FD. Safety of Fezolinetant for Treatment of Moderate to Severe Vasomotor Symptoms Due to Menopause: Pooled Analysis of Three Randomized Phase 3 Studies. Adv Ther. 2025 Feb;42(2):1147-1164. doi: 10.1007/s12325-024-03073-8. Epub 2024 Dec 30.

Reference Type DERIVED
PMID: 39739195 (View on PubMed)

Morga A, Zimmermann L, Valluri U, Siddiqui E, McLeod L, Bender RH. Validation and Application of Thresholds to Define Meaningful Change in Vasomotor Symptoms Frequency: Analysis of Pooled SKYLIGHT 1 and 2 Data. Adv Ther. 2024 Jul;41(7):2845-2858. doi: 10.1007/s12325-024-02849-2. Epub 2024 May 22.

Reference Type DERIVED
PMID: 38775925 (View on PubMed)

Schultz NM, Morga A, Siddiqui E, Rhoten SE. Psychometric Evaluation of the MENQOL Instrument in Women Experiencing Vasomotor Symptoms Associated with Menopause. Adv Ther. 2024 Jun;41(6):2233-2252. doi: 10.1007/s12325-024-02787-z. Epub 2024 Feb 24.

Reference Type DERIVED
PMID: 38396203 (View on PubMed)

Schultz NM, Morga A, Siddiqui E, Rhoten SE. Psychometric evaluation of the PROMIS SD-SF-8b instrument in individuals experiencing vasomotor symptoms due to menopause. Health Qual Life Outcomes. 2023 Nov 21;21(1):126. doi: 10.1186/s12955-023-02206-x.

Reference Type DERIVED
PMID: 37990323 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://www.trialsummaries.com/Study/StudyDetails?id=14833&tenant=MT_AST_9011

Link to plain language summary of the study on the Trial Results Summaries website.

Other Identifiers

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2018-003529-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2693-CL-0302

Identifier Type: -

Identifier Source: org_study_id

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