A Study to Evaluate the Safety and Effectiveness of ADX-914 in Subjects with Moderate to Severe Atopic Dermatitis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Q32 Bio Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

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

Trial duration

13 May 2024 → 11 Dec 2024

Decision date (initial)

2024-04-29

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Q32 Bio, Inc.

External identifiers

EU CT number

2023-510238-10-00

ClinicalTrials.gov

NCT05509023

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

Part A only: To identify the recommended ADX-914 dose for Part B
Part B only: To evaluate the efficacy of ADX-914 vs placebo

Secondary objectives 2

1. Part B only: To evaluate the efficacy of ADX-914 (Part B dose selected) vs placebo
2. Part A and B: To evaluate the safety and tolerability of ADX-914 compared with placebo in subjects with atopic dermatitis (AD) during the treatment period and follow-up

Conditions and MedDRA coding

Moderate to Severe Atopic Dermatitis

Study design 1 period

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Age ≥18 years, inclusive, at time of informed consent, with chronic AD (duration of disease ≥3 years) diagnosed by the Eichenfield revised criteria of Hanifin and Rajka.
2. 2. Moderate to severe disease activity at baseline and screening defined as: a. BSA affected ≥10% b. EASI Score ≥12 c. Investigators Global Score (IGA) ≥3
3. 3. Who, in the opinion of the Investigator, have a history of inadequate response to at least one of the following: a. at least 4 week course of medium-potency topical steroids or other approved topical immunomodulators (calcineurin, PDE-4 and JAK inhibitors) b. systemic steroids or phototherapy c. oral chemical synthetic immunomodulators (methotrexate [MTX], mycophenolate mofetil, azathioprine, cyclosporine, systemic approved biologics [dupilumab, ustekinumab or tralokinumab]), or approved systemic targeted synthetic JAK inhibitors (upadacitinib, abrocitinib)

Exclusion criteria 12

1. 1. Body weight ≤ 50.0 kg for men and ≤ 45.0 kg for women and > 120 kg at Screening
2. 2. Rescue therapy, topical or systemic, need anticipated within 4 weeks of randomization
3. 3. Recent (within 2 months of informed consent) or current clinically serious viral, bacterial, fungal, or parasitic infection or mycobacterial infection
4. 4. A positive QuantiFERON®TB Gold test at Screening or history of tuberculosis (TB)
5. 5. Have been exposed to a live vaccine within 12 weeks prior to planned randomization or are expected to receive a live vaccine during the study
6. 6. Systemic, topical or device-based therapy of AD or immunotherapy required for any other condition
7. 7. Serious concomitant illness that could require the use of systemic corticosteroids or otherwise interfere with study participation or require active frequent monitoring
8. 8. Other concomitant skin conditions that would interfere with evaluations of the effect of study medication on atopic dermatitis
9. 9. Other active autoimmune diseases other than those above that would make it difficult to appropriately assess AD disease activity or pose a risk to the subject's participation in the trial
10. 10. Pregnant or lactating women, or women planning to become pregnant or initiate breastfeeding.
11. 11. History of sensitivity to any of the study treatments, or components thereof, or a history of drug or other allergy that, in the opinion of the Investigator, contraindicates their participation.
12. 12. Has been in another investigational trial within 30 days or 5 half-lives of the investigational agent (whichever is greater) prior to the informed consent.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Part A only: Safety parameters including incidence of serious adverse events and adverse events of special interest
2. Part B only: Mean percentage change from baseline in Eczema Area and Severity Index (EASI) score at Week 14 for ADX-914 (Part B dose selected) vs placebo

Secondary endpoints 5

1. Mean percentage change from baseline in EASI score at Weeks 4, 8, 12, 16, and 24
2. Mean percentage change from baseline in Scoring Atopic Dermatitis (SCORAD) score at Weeks 4, 8, 12, 14, 16, and 24
3. Proportion of subjects achieving EASI 50, 75 and 90 at Weeks 4, 8, 12, 14, 16, and 24
4. Proportion of subjects achieving Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD) score of 0 or 1 with at least 2 grades of reduction from Baseline at Weeks 4, 8, 12, 14, 16, and 24
5. Part A and B: Safety of ADX-914 in the AD population as evaluated by adverse events (AEs), laboratory evaluations, physical examinations, vital signs, and 12-lead electrocardiogram (ECG)

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Q32 Bio Inc.

Sponsor organisation

Q32 Bio Inc.

Address

830 Winter Street Suite 2

City

Waltham

Postcode

02451-1477

Country

United States

Scientific contact point

Organisation

Q32 Bio Inc.

Contact name

Kristin Orr

Public contact point

Organisation

Q32 Bio Inc.

Contact name

Kristin Orr

Third parties 17

Locations

1 EU/EEA country · 6 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 26 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