A Study to Test the Efficacy and Safety of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures

NCT ID: NCT05077904

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

350 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-07

Study Completion Date

2026-04-06

Brief Summary

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The purpose of the study is to assess the success of a single administration of Staccato alprazolam compared with placebo both in rapidly terminating a seizure episode within 90 seconds and with no recurrence of seizure(s) up to 2 hours after investigational medicinal product (IMP) administration.

Detailed Description

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Conditions

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Stereotypical Prolonged Seizures

Keywords

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Stereotypical prolonged seizures Phase 3 Staccato alprazolam

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Staccato alprazolam Arm

Participants randomized to this arm will receive a single dose of Staccato alprazolam by inhalation.

Group Type EXPERIMENTAL

Staccato alprazolam

Intervention Type DRUG

Route of administration: Inhalation

Participants will receive one dose of Staccato alprazolam during the Treatment Period.

Placebo Arm

Participants randomized to this arm will receive a single dose of placebo by inhalation.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Route of administration: Inhalation

Participants will receive one dose of placebo during the Treatment Period.

Interventions

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Staccato alprazolam

Route of administration: Inhalation

Participants will receive one dose of Staccato alprazolam during the Treatment Period.

Intervention Type DRUG

Placebo

Route of administration: Inhalation

Participants will receive one dose of placebo during the Treatment Period.

Intervention Type OTHER

Other Intervention Names

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UCB7538

Eligibility Criteria

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Inclusion Criteria

* Participant must be ≥12 years of age at the Baseline/Randomization Visit
* Participant must have a study caregiver ≥18 years of age at the Screening Visit; the study caregiver(s) must be a relative, partner, friend, or legally authorized representative (LAR) of the participant, or a person who provides daily care to the participant and has a significant personal relationship with the participant; the study caregiver(s) must be able to recognize and observe the participant's seizures
* Participants with an established diagnosis of focal or generalized epilepsy or combined focal and generalized epilepsy with a documented history of stereotypical episodes of prolonged seizures that includes at least 1 of the following:

1. Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
2. Episodes of a focal seizure with a minimum duration of 3 minutes
3. Episodes of a focal seizure or a flurry of myoclonic seizures for at least 90 seconds followed by a generalized/bilateral tonic-clonic seizure with a minimum total duration of 3 minutes
* Prior to the Screening Visit, participant has experienced ≥4 stereotypical episodes of prolonged seizures in the past 6 months, and the last 2 stereotypical episodes of prolonged seizures must have occurred within the 3 months prior to the Screening Visit
* Participant has had a documented brain computerized tomography or magnetic resonance imaging review, performed after diagnosis of epilepsy and within the 5 years prior to the Screening Visit, that confirms the absence of a progressive neurological disorder
* Participant is receiving a regimen of antiseizure medications (ASMs) that has been stable (ie, no addition or removal of ASM\[s\]; dose adjustments are permitted to ASM\[s\]; dose adjustments are not permitted for benzodiazepines) for 30 days prior to the Screening Visit
* Male and female participants:

1. A male participant must agree to use contraception during the Outpatient Treatment Period and for at least 7 days after IMP administration and refrain from donating sperm during this period
2. A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies:

i) Not a woman of childbearing potential (WOCBP) OR ii) A WOCBP who agrees to follow the contraceptive guidance during the Outpatient Treatment Period and for at least 30 days after IMP administration
* Participant is capable of giving signed informed consent (or giving assent, where required), which includes compliance with the requirements and restrictions listed in the informed consent form (ICF), the protocol, and the individualized participant management plan (iPMP). The ICF or a specific assent form, where required, will be signed and dated by minors
* The participant's study caregiver(s) must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF, the protocol, and the iPMP

Exclusion Criteria

* Participant has a current history of alcohol or drug use disorder, as defined in the Diagnostic and Statistical Manual of Mental Disorders 5, within the previous 1 year
* Participant has a known hypersensitivity to any components of the IMP or comparable drugs (and/or an investigational device) as stated in this protocol or to albuterol (or similar bronchospasm rescue medication if needed to meet country-specific requirements)
* Participant has a diagnosis of atrial fibrillation or mitral stenosis
* Participant has a history of convulsive (generalized tonic-clonic) status epilepticus in the 8 weeks prior to the Screening Visit
* Participant has a history or presence of known nonepileptic seizures which cannot be distinguished from qualifying epileptic seizures
* Participant has a clinically significant known airway hypersensitivity (eg, bronchospasm to known allergens, such as pollen, animals, or food) and/or acute respiratory signs/symptoms (eg, shortness of breath, wheezing on lung auscultation)
* Participant has a clinically significant chronic pulmonary disorder other than mild asthma (eg, chronic obstructive pulmonary disease, restrictive lung diseases \[including idiopathic pulmonary fibrosis\]) and/or recent history or presence of hemoptysis or pneumothorax
* Participant has had a positive antigen test for acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and experienced moderate to severe signs/symptoms of respiratory distress necessitating hospitalization or outpatient treatment such as ambulatory oxygen, extensive treatment with inhaler medications, and/or oral medications for a duration of 4 weeks or more, unless full resolution occurred at least 6 months prior to Screening
* Participant has experienced a upper respiratory tract infection within 4 weeks or bronchitis/pneumonia within 3 months before the Screening Visit
* Participant has a history or presence of acute narrow-angle glaucoma
* Participant has a condition for which oral alprazolam is contraindicated (eg, myasthenia gravis, severe respiratory insufficiency, and sleep apnea syndrome)
* Participant has a history or presence of long QT syndrome, a family history of sudden death due to long QT syndrome, or unexplained syncope
* Chronic use of benzodiazepines for more than 3 days within a period of 7 days will be allowed for approximately 30 % of study participants
* Participant is taking any drug that is a strong CYP3A4 inhibitor, including azole antifungal agents (ketoconazole and itraconazole) and nefazodone
* Participant is taking any opioids (eg, fentanyl, oxycodone, morphine) or sedative hypnotics on a chronic basis
* Participant is taking nonselective beta blockers on a chronic basis
* Participant is taking pharmacotherapy for an active major psychiatric disorder where major changes in regimen are needed or anticipated during the study
* Participant has been treated with vagal nerve stimulation (VNS) for less than 6 months or VNS settings have changed within 30 days before the Screening Visit
* Participant has a clinically significant laboratory abnormality that may increase the risk associated with study participation or may interfere with the interpretation of study results, according to the judgment of the Investigator
* Participant has an oxygen saturation \<95 % (or less than normal in regions of altitude \>2500 meters) for greater than 30 seconds during the Screening Visit. In case of an out-of-range result, 1 repeat will be allowed. If the readings are out of range again, the study participant will be excluded
* Participant has \>2.0x upper limit of normal (ULN) of any of the following: alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), or \>1.0xULN total bilirubin (≥1.5xULN total bilirubin if known Gilbert's syndrome or \>2.0xULN total bilirubin for liver impairment)
* Participant has current unstable liver or biliary disease per Investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis
* Participant has a QT interval corrected for heart rate (QTc) \>450 msec (males), QTc interval \>470 msec (females), or QTc interval \>480 msec (participants with bundle branch block), PR interval ≥220 msec, or any other clinically significant electrocardiogram (ECG) abnormality according to the Investigator i) The QTc is the QT interval corrected for heart rate according to Fridericia's formula (QTcF). It is either machine-read or manually over-read
* Participant has a positive urine screen for drugs of abuse at the Screening Visit
* Participant has a blood pressure (BP) or heart rate (HR) outside the following range after 5 minutes rest: systolic BP: 90 mmHg to 150 mmHg; diastolic BP: 4 0mmHg to 95 mmHg; HR: 50 bpm to 100 bpm. In case of an out-of-range result, 1 repeat will be allowed. If the readings are out of range again, the study participant will be excluded
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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UCB Biopharma SRL

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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UCB Cares

Role: STUDY_DIRECTOR

001 844 599 2273 (UCB)

Locations

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Ep0162 50506

Phoenix, Arizona, United States

Site Status RECRUITING

Ep0162 50720

Scottsdale, Arizona, United States

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Little Rock, Arkansas, United States

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Downey, California, United States

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La Jolla, California, United States

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Long Beach, California, United States

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Los Angeles, California, United States

Site Status WITHDRAWN

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Orange, California, United States

Site Status COMPLETED

Ep0162 50367

New Haven, Connecticut, United States

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Washington D.C., District of Columbia, United States

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Boca Raton, Florida, United States

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Gulf Breeze, Florida, United States

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Jacksonville, Florida, United States

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Jacksonville, Florida, United States

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Miami, Florida, United States

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Miami, Florida, United States

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Orlando, Florida, United States

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Rockledge, Florida, United States

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Tampa, Florida, United States

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Honolulu, Hawaii, United States

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Boise, Idaho, United States

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Chicago, Illinois, United States

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Springfield, Illinois, United States

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Fort Wayne, Indiana, United States

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Lexington, Kansas, United States

Site Status WITHDRAWN

Ep0162 50395

New Orleans, Louisiana, United States

Site Status WITHDRAWN

Ep0162 50517

New Orleans, Louisiana, United States

Site Status RECRUITING

Ep0162 50093

Baltimore, Maryland, United States

Site Status WITHDRAWN

Ep0162 50488

Bethesda, Maryland, United States

Site Status WITHDRAWN

Ep0162 50615

Boston, Massachusetts, United States

Site Status RECRUITING

Ep0162 50047

Boston, Massachusetts, United States

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Ep0162 50110

Ann Arbor, Michigan, United States

Site Status COMPLETED

Ep0162 50507

St Louis, Missouri, United States

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Las Vegas, Nevada, United States

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Neptune City, New Jersey, United States

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New Brunswick, New Jersey, United States

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Buffalo, New York, United States

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New Hyde Park, New York, United States

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New York, New York, United States

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New York, New York, United States

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New York, New York, United States

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Rochester, New York, United States

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Charlotte, North Carolina, United States

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Ep0162 50487

Charlotte, North Carolina, United States

Site Status WITHDRAWN

Ep0162 50371

Akron, Ohio, United States

Site Status WITHDRAWN

Ep0162 50528

Cleveland, Ohio, United States

Site Status WITHDRAWN

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Portland, Oregon, United States

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Portland, Oregon, United States

Site Status WITHDRAWN

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Chattanooga, Tennessee, United States

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Memphis, Tennessee, United States

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Nashville, Tennessee, United States

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Nashville, Tennessee, United States

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Austin, Texas, United States

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Houston, Texas, United States

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Houston, Texas, United States

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Ep0162 50496

Round Rock, Texas, United States

Site Status WITHDRAWN

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Salt Lake City, Utah, United States

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Fitzroy, , Australia

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Herston, , Australia

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Melbourne, , Australia

Site Status WITHDRAWN

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South Brisbane, , Australia

Site Status ACTIVE_NOT_RECRUITING

Ep0162 40650

Blagoevgrad, , Bulgaria

Site Status RECRUITING

Ep0162 40708

Pazardzhik, , Bulgaria

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Ep0162 40665

Pleven, , Bulgaria

Site Status COMPLETED

Ep0162 40709

Pleven, , Bulgaria

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Sofia, , Bulgaria

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Ep0162 20128

Beijing, , China

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Beijing, , China

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Ep0162 20268

Beijing, , China

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Beijing, , China

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Changchun, , China

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Chengdu, , China

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Chengdu, , China

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Chongqing, , China

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Chongqing, , China

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Fuzhou, , China

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Guangzhou, , China

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Guangzhou, , China

Site Status COMPLETED

Ep0162 20264

Guangzhou, , China

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Guangzhou, , China

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Guangzhou, , China

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Hangzhou, , China

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Kunming, , China

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Lanzhou, , China

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Nanchang, , China

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Nanjing, , China

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Nanning, , China

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Qingdao, , China

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Shanghai, , China

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Shanghai, , China

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Shenzhen, , China

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Shijiazhuang, , China

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Suzhou, , China

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Tianjin, , China

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Wenzhou, , China

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Wuhan, , China

Site Status WITHDRAWN

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Yinchuan, , China

Site Status WITHDRAWN

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Zhanjiang, , China

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Zhengzhou, , China

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Ep0162 40670

Brno, , Czechia

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Ep0162 40672

Ostrava - Poruba, , Czechia

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Ep0162 40063

Prague, , Czechia

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Prague, , Czechia

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Prague, , Czechia

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Ep0162 40681

Bron, , France

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Ep0162 40688

Bron, , France

Site Status WITHDRAWN

Ep0162 40680

Dijon, , France

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Ep0162 40130

Marseille, , France

Site Status RECRUITING

Ep0162 40019

Paris, , France

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Ep0162 40682

Paris, , France

Site Status RECRUITING

Ep0162 40201

Rennes, , France

Site Status ACTIVE_NOT_RECRUITING

Ep0162 40199

Strasbourg, , France

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Ep0162 40577

Aachen, , Germany

Site Status WITHDRAWN

Ep0162 40683

Berlin, , Germany

Site Status RECRUITING

Ep0162 40685

Bielefeld, , Germany

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Ep0162 40023

Erlangen, , Germany

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Ep0162 40645

Frankfurt am Main, , Germany

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Ep0162 40689

Kehl-kork, , Germany

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Ep0162 40529

Marburg, , Germany

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Ep0162 40724

München, , Germany

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Balassagyarmat, , Hungary

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Ep0162 40673

Budapest, , Hungary

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Ep0162 40704

Budapest, , Hungary

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Ep0162 40653

Debrecen, , Hungary

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Ep0162 40690

Catanzaro, , Italy

Site Status WITHDRAWN

Ep0162 40674

Genova, , Italy

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Milan, , Italy

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Pavia, , Italy

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Roma, , Italy

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Ep0162 40675

Roma, , Italy

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Fukuoka, , Japan

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Hachioji-shi, , Japan

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Ep0162 20237

Hamamatsu, , Japan

Site Status WITHDRAWN

Ep0162 20249

Hiroshima, , Japan

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Ep0162 20236

Hōfu, , Japan

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Itami, , Japan

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Kodaira, , Japan

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Koshi-shi, , Japan

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Kyoto, , Japan

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Nagakute, , Japan

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Ep0162 20235

Nagoya, , Japan

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Niigata, , Japan

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Osaka, , Japan

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Ep0162 20241

Ōmura, , Japan

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Ep0162 20316

Sapporo, , Japan

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Ep0162 20297

Shinjuku-ku, , Japan

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Ep0162 20240

Shizuoka, , Japan

Site Status RECRUITING

Ep0162 20242

Suita, , Japan

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Ep0162 20266

Tōon, , Japan

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Ep0162 20244

Yamagata, , Japan

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Ep0162 40707

Bydgoszcz, , Poland

Site Status RECRUITING

Ep0162 40677

Gdansk, , Poland

Site Status RECRUITING

Ep0162 40219

Krakow, , Poland

Site Status WITHDRAWN

Ep0162 40502

Krakow, , Poland

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Ep0162 40842

Krakow, , Poland

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Ep0162 40676

Lublin, , Poland

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Ep0162 40091

Nowa Sól, , Poland

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Ep0162 40153

Poznan, , Poland

Site Status WITHDRAWN

Ep0162 40678

Świdnik, , Poland

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Ep0162 40160

Barcelona, , Spain

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L'Hospitalet de Llobregat, , Spain

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Ep0162 40540

Madrid, , Spain

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Ep0162 40352

Pamplona, , Spain

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Ep0162 40668

Seville, , Spain

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Ep0162 40453

Terrassa, , Spain

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Ep0162 40230

Valencia, , Spain

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Ep0162 40667

Valladolid, , Spain

Site Status COMPLETED

Ep0162 40686

Birmingham, , United Kingdom

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Ep0162 40300

Cardiff, , United Kingdom

Site Status WITHDRAWN

Ep0162 40735

Glasgow, , United Kingdom

Site Status RECRUITING

Ep0162 40163

Oxford, , United Kingdom

Site Status RECRUITING

Ep0162 40108

Salford, , United Kingdom

Site Status RECRUITING

Countries

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United States Australia Bulgaria China Czechia France Germany Hungary Italy Japan Poland Spain United Kingdom

Central Contacts

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UCB Cares

Role: CONTACT

Phone: 1-844-599-2273 (USA)

Email: [email protected]

UCB Cares

Role: CONTACT

Phone: 0018445992273

Email: [email protected]

Other Identifiers

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2021-002686-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1303-2588

Identifier Type: OTHER

Identifier Source: secondary_id

2024-510950-29

Identifier Type: REGISTRY

Identifier Source: secondary_id

EP0162

Identifier Type: -

Identifier Source: org_study_id