Open- label study in children and adolescents with migraine: Acute Treatment

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Pfizer Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

1 Feb 2022 → ongoing

Decision date (initial)

2024-07-24

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Pfizer Inc.

External identifiers

EU CT number

2024-512744-32-00

EudraCT number

2020-003761-18

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others

To evaluate the safety and tolerability of rimegepant in children and adolescents (age ≥ 6 to < 18 years of age).

Secondary objectives 1

1. To evaluate the frequency and severity of hepatic-related adverse events and the frequency of hepatic-related treatment discontinuations in children and adolescents (6 to less than 18 years of age) treated with rimegepant.

Conditions and MedDRA coding

Acute Migraine (with or without aura)

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002812-PIP02-20

Plan to share IPD

Yes

IPD plan description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. History of migraine (with or without aura) for ≥ 6 months before Screening.
2. History of 1 to 8 moderate or severe attacks per month during the 2 months prior to screening.
3. Participant had 1 or more migraine days requiring treatment during the Observation Phase.
4. Participants on prophylactic migraine medication are permitted to remain on therapy if the dose has been stable for at least 12 weeks prior to the Screening Visit.
5. Participants are required to verbally distinguish between migraine and other types of headaches.
6. Participants must have a body weight > 15 kg. For EU countries only: Participants 12 to < 18 years of age must have a body weight of >25 kg.
7. Participants must have adequate venous access for blood sampling. 8.
8. Participants 6 to < 18 years of age (participants must not reach their 18th birthday on or before enrollment into the study).Baseline visit).

Exclusion criteria 7

1. Participant has a history of cluster headache or hemiplegic migraine headache. 2.
2. The participant has a confounding and clinically significant pain syndrome that may interfere with their ability to participate in this study.
3. The participant has any current psychiatric condition that is uncontrolled and/or untreated for a minimum of 6 months prior to the Screening Visit. Participants with a lifetime history of psychosis and/or mania are excluded.
4. History of suicidal behavior or the participant is at risk of self-harm or harm to others.
5. History of major psychiatric disorder.
6. The participant has a current diagnosis or history of substance abuse.
7. The participant has reported current use of or has tested positive at the Screening visit for drugs of abuse.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The frequency and severity of on-treatment and treatment-emergent adverse events (TEAEs) that occur in at least 5% of treated participants, the frequency of serious adverse events (SAEs) , adverse events (AEs) leading to discontinuation and clinically significant laboratory abnormalities during the treatment period will be assessed. Clinically significant laboratory abnormalities will be identified as on-treatment Grade 3 to 4 laboratory test results.

Secondary endpoints 1

1. The frequency and severity of hepatic-related adverse events (AEs) and frequency of hepatic-related AEs leading to treatment discontinuation will be tabulated from case report forms and will be based on unique participants reporting such events. Severity will be assessed as the worst severity observed while the participant is on treatment.

Investigational products

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

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pfizer Inc.

Sponsor organisation

Pfizer Inc.

Address

66 Hudson Boulevard East

City

New York

Postcode

10001-2189

Country

United States

Scientific contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Public contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Third parties 4

Locations

2 EU/EEA countries · 8 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 23 files

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

Application history

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