Randomized double blind phase 2 trial of baby exemestane versus baby tamoxifen in postmenopausal women at high risk for breast cancer

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ente Ospedaliero Ospedali Galliera Di Genova

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

1 Oct 2025 → ongoing

Decision date (initial)

2025-07-15

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Breast Cancer Research Foundation (BCRF)

External identifiers

EU CT number

2024-520004-26-00

ClinicalTrials.gov

NCT06364267

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Others

To compare low dose of exemestane (babyexe) versus low dose of tamoxifen (babytam) in terms of change of quality of life (overall score of Menopause-Specific Quality of Life Questionnaire - MENQOL)
from baseline to 12 months.

Secondary objectives 10

1. To compare differences between babyexe and babytam on Sex hormones (free estradiol, estradiol/SHBG), IGF system (IGF-I, IGFBP-3 and their ratio) at 12 months.
2. To determine differences between babyexe and babytam on Overall MENQOL domains at 6 months
3. To determine differences between babyexe and babytam on Sex hormones (free estradiol, estradiol/SHBG), IGF system (IGF-I, IGFBP-3 and their ratio) at 6 months
4. To determine differences between babyexe and babytam on Individual domains of MENQOL (physical, sexual, psychosocial, vasomotor) at 6 and 12 months
5. To determine differences between babyexe and babytam on Toxicity profile according to NCICTC AE version 5
6. To determine differences between babyexe and babytam on Pill adherence
7. To determine differences between babyexe and babytam on Joint pain and stiffness
8. To determine differences between babyexe and babytam on C-telopetide at 6 and 12 months
9. To determine differences between babyexe and babytam on Patient uptake through satisfaction and reasons for trial acceptance or refusal will be evaluated in all screened subjects.
10. To determine differences between babyexe and babytam on Toxicity of babyexe in comparison with full dose historical controls treated in the adjuvant setting will also be evaluated.

Conditions and MedDRA coding

Breast cancer

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post- menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. In addition, any of the following criteria must be met: a. Recent (within 12 months from date of consent form signature) histologic diagnosis of ER+ve (>5%) DCIS or diagnosis within 3 years of HRL (ADH, LCIS, ALH), or: b. At least 3% breast cancer risk at 5 years (or 5% risk at 10 yrs) per one of the following risk models: the Breast Cancer Surveillance Consortium risk calculator V3 and Tyrer-Cuzick model V8, or: c. Known carriers of a germline pathogenic/likely pathogenetic variant within 5 years in the following moderate penetrance genes (CHEK2 or ATM), or women with chest wall irradiation before age of 30 years.
2. Eastern Cooperative Oncology Group - Performance Status (ECOG-PS) 0-1.
3. Able to swallow oral medications.
4. Participants with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial. Specifically, all cancers diagnosed since 3 years or longer except for breast and endometrial are eligible.
5. Ability to understand and willingness to sign a written informed consent document.
6. Mammography performed up to 6 months before the trial consent form signature.
7. DEXA performed up to 12 months before the trial consent form signature.
8. Patients with life expectancy >= 10 years.
9. Patients with normal liver function tests and blood cell count.
10. Negative gynecological examination performed up to 6 months before the trial consent form signature.

Exclusion criteria 11

1. Pre/perimenopausal women
2. History of DVT or PE.
3. Endometrial cancer.
4. Macular disorders.
5. Inability to comply with study procedures.
6. Prior use of antiestrogens within 12 months from the date of the trial consent form signature.
7. Use of hormone replacement therapy (HRT) within 3 months from the date of the trial consent form signature.
8. Severe osteoporosis (T score <-2.5 at either spine or hip), or recent vertebral fracture (within 6 months) not treated with zolendronic acid or denosumab.
9. Use of terbinafine, quinidine, cinacalcet, rifampicin, phenytonin, carbamazepine, phenobarbital, and St. John’s wort, warfarin, erythromycin, cyclosporin, nifepidine and any concomitant type of coumarin-type of anticoagulant therapy.
10. Patients with moderate or severe renal impairment.
11. Patients with a known hypersensitivity to study drugs.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is the difference between the mean changes of individual scores (overall MENQOL) between the two arms, where the change is the difference between baseline and 12 months.

Secondary endpoints 9

1. The difference in sex hormones (free estradiol: estradiol/SHBG) and IGF system (IGF-I, IGFBP-3 and their ratio) after 12 months.
2. The difference between arms in the overall MENQOL score after 6 months of treatment.
3. Sex hormones (free estradiol, estradiol/SHBG), IGF system (IGF-I, IGFBP-3 and their ratio) at 6 months. The difference between arms in each of the four individual MENQOL domain scores (physical, vasomotor, sexual and psychosocial) at 6 and 12 months.
4. The difference between arms of toxicity and safety profile according to CTCAE v.5 at 6 and 12 months.
5. The difference between arms at 6 and 12 months in PMAS, Promise Medication Adherence Scale, a validated tool to measure pill adherence.
6. The difference between arms on BPI Brief Pain Inventory at 6 and 12 months.
7. The difference between arms in C-telopetide at 6 and 12 months.
8. Patient uptake at screening/baseline phase will be measured with a questionnaire including factors related to breast cancer worry and presence of life style risk factors, and participant satisfaction for study explanation.
9. Toxicity of babyexe in comparison with full dose historical controls treated in the adjuvant setting will also be evaluated.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ente Ospedaliero Ospedali Galliera Di Genova

Sponsor organisation

Ente Ospedaliero Ospedali Galliera Di Genova

Address

Mura Delle Cappuccine 14

City

Genoa

Postcode

16128

Country

Italy

Scientific contact point

Organisation

Ente Ospedaliero Ospedali Galliera Di Genova

Contact name

Andrea De Censi

Public contact point

Organisation

Ente Ospedaliero Ospedali Galliera Di Genova

Contact name

Sonia Mammoliti

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 29 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications