Orchid 1

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Mc2 Therapeutics Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

12 Oct 2023 → 10 Nov 2024

Decision date (initial)

2023-10-09

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To explore the efficacy of MC2-25 cream compared to MC2-25 vehicle in vulvar lichen sclerosus (VLS).

Secondary objectives 2

1. To explore the safety of MC2-25 cream compared to MC2-25 vehicle in VLS.
2. To explore the burden of VLS on women’s lives.

Conditions and MedDRA coding

Vulvar lichen sclerosus (VLS)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Women, of any race or ethnicity, who are ≥18 years of age at the time of Screening 1.
2. Able to understand the trial and willing to comply with trial requirements.
3. Has provided written informed consent.
4. Clinical diagnosis of VLS made by either a dermatologist or a gynaecologist familiar with VLS.
5. Presence of at least one of the following signs of VLS: a. Hyperkeratosis (i.e., patches/plaques of bright white skin with a ‘powdery’ texture) and/or b. Sclerosis (i.e., areas of yellowish/ivory white skin with a smooth/waxy/firm texture. Sclerosis is often seen at the tips of the labia minora, or on periclitoral or perineal skin) in at least one of the following vulvar areas: Clitoris and/or periclitoral skin (C); Right interlabial sulcus and labium minus (R); Left interlabial sulcus and labium minus (L); Posterior Fourchette and/or perineum (P)
6. First symptoms of VLS (e.g., itching and/or pain) noticed by the patient at least 6 months before baseline.
7. At least four WI-NRS scores available in the diary for calculation of the average WI-NRS at the baseline visit.
8. At least moderate itch defined as average WI-NRS ≥4 at the Baseline visit (average WI-NRS is calculated as the average of all available and at least four WI-NRS scores which are to be reported once daily by the patient in the diary for 7 days prior to the Baseline visit
9. Women must be of either: a) Non-childbearing potential, i.e., post-menopausal* or confirmed sterile (e.g., hysterectomy, bilateral salpingectomy or bilateral oophorectomy) (*Note: a 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 postmenopausal 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) OR, b) Childbearing potential with a negative highly sensitive* urine pregnancy test at the Baseline visit (*Note: a highly sensitive urine pregnancy test must have a sensitivity down to at least 25 mIU/ml for human chorionic gonadotropin (hCG)).
10. Women of childbearing potential must agree to use a highly effective method of contraception (i.e., a method with a failure rate of less than 1 % per year when used consistently and correctly) while receiving double-blind treatment.

Exclusion criteria 17

1. Pregnant, breast feeding, or planning to become pregnant during the trial.
2. Any (other than VLS) ongoing localised or systemic disease involving the vulvar region – e.g., lichen planus, psoriasis, eczema, ulcerative colitis or known active infection (bacterial, viral or fungal).
3. Ongoing symptomatic Urinary Tract Infection (UTI).
4. Ongoing or prior diagnosis of any genitoanal malignancy or pre-malignancy
5. Any kind of ongoing cancer (or anti-cancer treatment within 3 months or 5 half-lives (whichever is longest) prior to the Baseline visit).
6. Any chronic or acute systemic medical condition that, in the opinion of the investigator, may pose a risk to the safety of the patient or may interfere with the assessment of efficacy in this trial.
7. Known history of allergic reaction to any ingredients in MC2-25 cream or MC2-25 vehicle
8. Start of a new or change of existing non-biologic systemic treatment, including but not limited to corticosteroids, cyclosporin, methotrexate and tacrolimus, within 21 days prior to the Baseline visit.
9. Start of a new or change of existing biologic systemic treatment, including but not limited to etanercept, adalimumab, alefacept, infliximab, and ustekinumab within 3 months or 5 half-lives (whichever is longest) prior to the Baseline visit.
10. Start of a new or change of existing systemic or intravaginal treatment with estrogen containing products, within 21 days prior to the Baseline visit
11. Start of new or change of menstrual care routines within 21 days prior to the Baseline visit.
12. Use of emollients (including but not limited to creams, ointments, oils, or soaps with emollient properties) on the vulvar region within 3 days prior to the Baseline visit.
13. Use of any topical treatment on the vulvar region, including but not limited to calcineurin inhibitors, corticosteroids or anti-infectives within 14 days prior to the Baseline visit.
14. Use of any light therapy on the vulvar region, including but not limited to UV-B, UV-A, and laser, within 28 days prior to the Baseline visit.
15. Received a non-marketed or blinded drug within 28 days or 5 half-lives (whichever is longer) prior to the Baseline visit.
16. If in the opinion of the investigator, the patient is unlikely to comply with the clinical trial protocol.
17. Previously randomised in this trial.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Mean change in weekly mean Worst Itch Numeric Rating Score (WI-NRS) recorded in the patient’s diary from Baseline to Week 12 for MC2-25 cream compared to MC2-25 vehicle.

Secondary endpoints 6

1. Mean change in weekly mean Worst Pain Numeric Rating Score (WP-NRS) recorded in the patient’s diary from Baseline to Week 12 for MC2-25 cream compared to MC2-25 vehicle.
2. Percentage of patients obtaining a ≥4-point improvement in weekly mean WI-NRS recorded in the patient’s diary from Baseline to Week 12 for MC2-25 cream compared to MC2-25 vehicle.
3. Percentage of patients obtaining a ≥4-point improvement in weekly mean WP-NRS recorded in the patient’s diary from Baseline to Week 12 for MC2-25 cream compared to MC2-25 vehicle.
4. Percentage of patients obtaining a ≥3-point improvement in weekly mean WI-NRS recorded in the patient’s diary from Baseline to Week 12 for MC2-25 cream compared to MC2-25 vehicle.
5. Percentage of patients obtaining a ≥3-point improvement in weekly mean WP-NRS recorded in the patient’s diary from Baseline to Week 12 for MC2-25 cream compared to MC2-25 vehicle.
6. Change in Skindex-29 domains from baseline to week 12 for MC2-25 cream compared to MC2-25 vehicle.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Mc2 Therapeutics Limited

Sponsor organisation

Mc2 Therapeutics Limited

Address

1a Guildford Business Park

City

Guildford

Postcode

GU2 8XG

Country

United Kingdom

Scientific contact point

Organisation

Mc2 Therapeutics Limited

Contact name

Rikke Pordel Vind

Public contact point

Organisation

Mc2 Therapeutics Limited

Contact name

Rikke Pordel Vind

Third parties 1

Locations

1 EU/EEA country · 7 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 4 files

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

Application history

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