A study to evaluate IPN10200 safety and efficacy in the prevention of episodic or chronic migraine in adults

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ipsen Innovation

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

24 Feb 2026 → ongoing

Decision date (initial)

2025-07-15

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Ipsen Innovation: 70 rue Balard, 75015 Paris, France.

External identifiers

EU CT number

2024-511101-28-00

ClinicalTrials.gov

NCT06625060

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Prophylaxis, Dose response, Efficacy, Safety

To assess the safety and tolerability and potential immunogenicity of the IPN10200 single treatment cycle in adult participants with episodic migraine (EM) or chronic migraine (CM).To determine if IPN10200 is superior to placebo in prevention of migraine when administered as a single treatment cycle in participants with episodic (EM) or chronic migraine (CM) independently.

Secondary objectives 15

1. To compare the magnitude of change in the frequency of monthly migraine days (MMDs) following a single treatment cycle of IPN10200 or placebo in adult participants with EM or CM
2. To compare the magnitude of change in the frequency of monthly headache days (MHD) following a single treatment cycle of IPN10200 or placebo in adult participants with EM or CM
3. To compare the magnitude of change in the frequency of moderate/severe headache days following a single treatment cycle of IPN10200 or placebo in adult participants with EM or CM
4. To evaluate the improvement in migraine prevention response following a single treatment cycle of IPN10200 or placebo in adult participants with EM or CM
5. To evaluate the improvement in headache prevention response following a single treatment cycle of IPN10200 or placebo in adult participants with EM or CM
6. The change in the frequency of acute medication used by the participants to relieve migraine symptoms.
7. The change in the use of acute medication to relieve migraine symptoms in adult participants with EM or CM
8. To determine if IPN10200 is superior to placebo in the prevention of migraine as assessed by the magnitude of the change in the frequency of migraine days following IPN10200/placebo administration in the CM and EM groups independently.
9. To determine if IPN10200 is superior to placebo in the prevention of migraine as assessed by the magnitude of the change in the frequency of headache days following IPN10200/placebo administration in the CM and EM groups independently.
10. To determine if IPN10200 is superior to placebo in the prevention of migraine as assessed by the magnitude of the change in the frequency of moderate/severe headache days following IPN10200/placebo administration in the CM and EM groups independently.
11. To evaluate the improvement in migraine prevention response following a single treatment cycle of IPN10200 or placebo in the CM and EM groups independently.
12. To evaluate the improvement in headache prevention response following a single treatment cycle of IPN10200 or placebo in the CM and EM groups independently.
13. To determine if IPN10200 is superior to placebo in the prevention of migraine as assessed by change in the frequency of acute medication used by the participants to relieve migraine symptoms in the CM and EM groups independently.
14. To assess the safety and tolerability of the IPN10200 single treatment cycle in adult participants with EM or CM independently.
15. To assess the potential immunogenicity of IPN10200 in adult participants with EM or CM independently.

Conditions and MedDRA coding

Episodic Migraine Chronic Migraine

Regulatory references

Scientific advice from competent authorities

Pharmaceuticals And Medical Devices Agency, Food And Drug Administration, Center For Drug Evaluation

Plan to share IPD

Yes

IPD plan description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board. Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later. Access Criteria: Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/). URL: https://vivli.org/members/ourmembers/

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF. Participant has provided written informed consent and signed privacy/data protection documentation
2. Male or female ≥18 to 80 years of age at the time of signing the informed consent
3. Diagnosis of either EM or CM, per ICHD-3 criteria, for at least 12 months prior to the screening visit
4. Diagnosis of migraine at ≤50 years of age
5. Participants in the EM group: History of EM diagnosis and headache frequency (i.e. migraine and non-migraine headache): ≤14 headache days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary; migraine frequency: ≥6 migraine days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary;
6. Participants in the CM group: History of CM diagnosis and headache frequency (i.e. migraine and non-migraine headache): ≥15 headache days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary; migraine frequency: ≥8 migraine days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary;
7. Participant with a history of use of at least one preventive treatment for migraine for at least 8 weeks prior to the screening visit

Exclusion criteria 19

1. History or current diagnosis of migraine with brainstem aura, retinal migraine, complications of migraine, tension-type headache, trigeminal autonomic cephalalgias, hypnic headache, hemicrania continua or new daily persistent headache
2. Use of any of the following medications in the specified timeframe prior to the screening visit: - Botulinum toxin for migraine within 24 weeks (or for any other medical/aesthetic reason within 16 weeks); - Prior use of mAbs blocking CGRP pathway within 12 weeks for preventative treatment of migraine; - Prior use of oral CGRP receptor antagonist (gepants) for preventative treatment of migraine within 2 weeks; - Anaesthetic or steroid injection in any region targeted for treatment with study medication within 4 weeks; - Use of cannabidiol or other types of cannabinoids within 30 days; - Use of medical device to treat migraine within 4 weeks (e.g. non-invasive neuromodulation therapies such as nerve stimulation (gammaCore), transcranial magnetic stimulation (cephaly), external trigeminal nerve stimulation, transcutaneous electrical nerve stimulation and peripheral neuroelectrical stimulation); - Use of other intervention to treat migraine that is assessed to interfere with study evaluations within 4 weeks (e.g. acupuncture in the face, head and neck region, cranial traction, nociceptive trigeminal inhibition, occipital nerve block treatments and dental splints for headache); - Use of opioids or barbiturates for more than 2 days/month within the last 4 weeks.
3. Participation in another interventional clinical study or use of any treatment with an experimental drug within 30 days or within five times the documented terminal half-life of the respective drug or its metabolites (if the half-life is unknown, within 30 days) prior to the screening visit and during the conduct of the study
4. Diagnosis of fibromyalgia or other significant pain disorders that could confound the assessment of headaches/migraines or interfere with study participation, including but not limited to chronic pain disorders such as chronic low back pain and complex regional pain syndrome
5. Pregnant women, nursing women, premenopausal women, or WOCBP (i.e. not surgically sterile or 1 year postmenopausal) not willing to practice an acceptable contraceptive method at the beginning of the study, and for a minimum of 12 weeks following the administration of study treatment
6. Male subjects who are not vasectomised and who have female partners of childbearing potential and are not willing to use condoms with or without spermicide for a minimum of 12 weeks following the initial double-blind administration of the treatment
7. History of alcohol or drug abuse within 5 years of the screening visit (excluding medication overuse for headache)
8. Known clinically significant hypersensitivity to any of the study drugs, excipients or materials used to administer the study drug;
9. Patients who, in the clinician’s judgment, are actively suicidal, and therefore, deemed to be at significant risk for suicide
10. A diagnosis of a neuromuscular disorder or respiratory disorder, such as myasthenia gravis, Lambert-Eaton syndrome or amyotrophic lateral sclerosis that in the opinion of the investigator would compromise the safety of the study participant
11. Headache attributed to another disorder (e.g. secondary headaches), except medication overuse headache (MOH)
12. Current uncontrolled psychiatric or psychological condition, or one that could confound assessment of headaches/migraines or interfere with study participation
13. Risk of self-harm or harm to others as evidenced by past suicidal behaviour or endorsing items 3, 4, or 5 on the C-SSRS at Screening or Day 1 (except for France).
14. Participants presenting with a swallowing disorder of any origin which might be exacerbated by botulinum toxin treatment, such as: - Grade 3 or 4 on the Dysphagia Severity Scale (severe dysphagia) with swallowing difficulties and requiring a change in diet.
15. Clinically relevant skin condition or infection that could interfere with injection of study intervention
16. Participant has any medical condition or situation that would make them unsuitable for participation in the study
17. Participant receiving more than one allowable concomitant migraine preventive treatment
18. Known history of an inadequate response to >4 medications prescribed for the prevention of migraine (2 of which have different mechanisms of action to botulinum toxin)
19. Body mass index (BMI) ≥35 kg/m² at the screening visit

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 9

1. Percentage of participants experiencing any Adverse Event (AEs) including treatment emergent adverse events (TEAEs), serious adverse events (SAEs), adverse event of special interest (AESI) and AE leading to treatment discontinuation [ Time Frame: For step 1: From baseline until end of study at Week 36 ]
2. Percentage of Participants with clinically significant changes from baseline in Laboratory Parameters [ Time Frame: For step 1: At all timepoints post injection until Week 36 ] Clinically significant change in laboratory parameters will be reported. The clinical significance will graded by the investigator.
3. Percentage of Participants With Clinically Significant Changes from baseline in Vital Signs [ Time Frame: For step 1: At all timepoints post injection until Week 36 ] Clinically significant changes in vital signs will be reported. The clinical significance will be graded by the investigator.
4. Percentage of participants with clinically significant change from baseline in facial examination [ Time Frame: For step 1: At all timepoints post injection until Week 36 ] Clinically significant changes in facial examination and focused neurological/physical examinations will be reported. The clinical significance will be graded by the investigator.
5. Percentage of participants with clinically significant change from baseline in 12-lead Electrocardiogram (ECG) readings [ Time Frame: For step 1: At all timepoints post injection until Week 36 ]
6. Treatment-emergence of suicidal ideation/suicidal behaviour [ Time Frame: For step 1: At all timepoints post injection until Week 36 ]
7. Percentage of participants with Binding antibodies to IPN10200 [ Time Frame: For step 1: At baseline, Week 4, Week 12 and Week 36. ]
8. Percentage of participants with neutralising antibodies to IPN10200 [ Time Frame: For step 1: At baseline, Week 4, Week 12 and Week 36. ]
9. Change from baseline in the number of Monthly migraine days (MMD)s [ Time Frame: For step 2: At Week 12 (Weeks 9-12). ]

Secondary endpoints 9

1. Change from baseline in the number of MMD [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36. ]
2. Change from baseline in the number of Monthly Headache Days (MHD) [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36 ]
3. Change from baseline in the number of moderate/severe MHD [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36 ]
4. Migraine prevention response [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36 ] Migraine prevention response is assessed using two thresholds: by a reduction from baseline of either ≥50% or ≥75% in MMD
5. Headache prevention response [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36. ] Assessed using two thresholds: by a reduction from baseline of either ≥50% or ≥ 75% in MHD
6. Change from baseline in the number of days per 4 week period of acute medication use for migraine relief. [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36. ] An acute medication use day is defined as any day on which a participant reports, per eDiary, the intake of allowed medication(s) for the acute treatment of migraine.
7. The use of acute migraine medication (yes or no) [ Time Frame: For Step 1 and step 2: From Week 1 to Week 36. ] The use of acute migraine medication will be recorded in the daily eDiary.
8. Percentage of participants with Binding antibodies to IPN10200 [ Time Frame: For step 2 : At baseline, Week 4, Week 12 , Week 24 and Week 36. ]
9. Percentage of participants with neutralising antibodies to IPN10200 [ Time Frame: For step 2 : At baseline, Week 4, Week 12 , Week 24 and Week 36. ]

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ipsen Innovation

Sponsor organisation

Ipsen Innovation

Address

70 Rue Balard

City

Paris

Postcode

75015

Country

France

Scientific contact point

Organisation

Ipsen Innovation

Contact name

Ipsen Clinical Study Enquiries

Public contact point

Organisation

Ipsen Innovation

Contact name

Ipsen Clinical Study Enquiries

Third parties 5

Locations

5 EU/EEA countries · 42 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 69 files

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

Application history

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