A clinical trial to evaluate efficacy and safety of Xeomin® injections for preventing episodic migraine

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Merz Therapeutics GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

6 Oct 2025 → ongoing

Decision date (initial)

2025-09-05

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Merz Therapeutics GmbH

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy

To establish evidence of efficacy of Xeomin Dose A in the treatment of EM by demonstrating superiority compared to Placebo in reducing monthly migraine days.

Secondary objectives 8

1. To establish evidence of efficacy of Xeomin Dose B in the treatment of EM by demonstrating superiority compared to Placebo in reducing monthly migraine days.
2. To establish evidence of efficacy of Xeomin Dose A in the treatment of EM by demonstrating superiority compared to Placebo in reducing monthly headache days.
3. To establish evidence of efficacy of Xeomin Dose B in the treatment of EM by demonstrating superiority compared to Placebo in reducing monthly headache days.
4. To establish evidence of efficacy of Xeomin Dose A in the treatment of EM by demonstrating superiority compared to Placebo in reducing monthly acute migraine medication days.
5. To establish evidence of efficacy of Xeomin Dose B in the treatment of EM by demonstrating superiority compared to Placebo in reducing monthly acute migraine medication days.
6. To establish evidence of a shorter “wear-off period” of Xeomin Dose A in the treatment of EM by demonstrating superiority to Xeomin Dose B in the reduction of two-week end-of-cycle migraine days (i.e., migraine days in weeks 11 and 12 of each injection cycle). The “wear-off period” is the period of a treatment cycle during which the treatment effect gradually decreases.
7. To support the primary efficacy objective by comparing the 50% responder rates.
8. To demonstrate safety and tolerability of Xeomin compared to placebo in participants with EM.

Conditions and MedDRA coding

Episodic migraine

Study design 3 periods

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 5

1. At least 18 years of age, at the time of signing the informed consent;
2. Participant has a diagnosis of EM with or without aura according to current International Classification of Headache Disorders (Edition 3 and Edition 4 alpha) criteria for ≥ 12 months and is able to distinguish migraine headaches from all other types of headaches;
3. Participant age < 50 years at the time of migraine onset;
4. Participant meeting the following headache and migraine day criteria in each of the 3 months prior to screening: history of ≤ 14 headache days per month and history of 6 to 14 migraine days per month; and
5. During the last 28 days of the screening period, participant experiencing: ≤ 14 headache days and 6 to 14 migraine days that qualify as such per the headache diary.

Exclusion criteria 6

1. Diagnosis of chronic migraine;
2. Diagnosis of other primary headache types, except tension-type headache, which is permitted;
3. Diagnosis of aura without headache, migraine with brainstem aura, hemicrania continua, hypnic headache, hemiplegic migraine, retinal migraine, persistent aura without infarction, migraine aura-triggered seizure, or previous migrainous infarction;
4. Diagnosis of secondary headache types, except medication overuse headache, which is permitted;
5. Currently taking > 1 prescribed drug for the preventive treatment of migraine;
6. Discontinuation of anti-calcitonin gene-related peptide (CGRP) / anti-CGRP receptor monoclonal antibody treatment less than 5 months prior to screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in monthly migraine days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection) – Dose A.

Secondary endpoints 8

1. Change in monthly migraine days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection) – Dose B.
2. Change in monthly headache days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection) – Dose A.
3. Change in monthly headache days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection) – Dose B.
4. Change in monthly acute migraine medication days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection) – Dose A.
5. Change in monthly acute migraine medication days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection) – Dose B.
6. Change in frequency of migraine days from baseline (baseline monthly migraine days divided by 2) to two-week end-of-cycle periods (weeks 11 and 12 of Cycle 1 to 4).
7. Percentage of participants who reported at least a 50% reduction in mean monthly migraine days from baseline (28 days before baseline visit) to Month 6 (weeks 21 to 24 after first injection).
8. Incidence of treatment‑emergent adverse events (TEAEs) related to treatment as assessed by the investigator in the placebo-controlled period (PCP).

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merz Therapeutics GmbH

Sponsor organisation

Merz Therapeutics GmbH

Address

Eckenheimer Landstrasse 100, Nordend West Nordend West

City

Frankfurt Am Main

Postcode

60318

Country

Germany

Scientific contact point

Organisation

Merz Therapeutics GmbH

Contact name

Contact Point Clinical Trials

Public contact point

Organisation

Merz Therapeutics GmbH

Contact name

Contact Point Clinical Trials

Third parties 10

Locations

9 EU/EEA countries · 61 investigational sites

By country

Investigational sites

Country notifications

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Corrective measures 1 · Art. 77 CTR

Results and documents

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

Documents 91 files

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

Application history

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