Эта страница была переведена автоматически, точность перевода не гарантируется. Пожалуйста, обратитесь к английской версии для исходного текста.

Исследование для документирования и дальнейшего описания долгосрочной безопасности и эффективности паловаротена у участников с прогрессивной оссифицирующей фибродисплазией (ФОП) (FOPal)

20 октября 2026 г. обновлено: Ipsen

Исследование международного наблюдательного регистра для дальнейшего описания долгосрочной безопасности и эффективности паловаротена у пациентов с прогрессирующей оссифицирующей фибродисплазией (ФОП)

The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP).

FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)).

HO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups).

This registry study will take place in countries where the treatment, known as palovarotene, has been approved for use. Participants will either be treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) or untreated with palovarotene.

The main aim of this registry study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene.

This registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance.

In addition, this registry study aims to descriptively compare key safety outcomes (i.e. flare-up episodes, growth outcomes, and bone fractures) between participants exposed and unexposed to palovarotene.

Обзор исследования

Статус

Рекрутинг

Условия

Тип исследования

Наблюдательный

Регистрация (Оцененный)

100

Контакты и местонахождение

В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.

Контакты исследования

  • Имя: Ipsen Clinical Study Enquiries
  • Номер телефона: see email
  • Электронная почта: clinical.trials@ipsen.com

Места учебы

  • Канада
      • Edmonton, Канада
        • Рекрутинг
        • Edmonton Clinic Health Academy (ECHA)- University of Alberta
      • Oakville, Канада
        • Активный, не рекрутирующий
        • Bone Research and Education Centre
      • Toronto, Канада
        • Рекрутинг
        • University Health Network (UHN) - Toronto General
  • Соединенные Штаты
    • California
      • San Francisco, California, Соединенные Штаты, 94103
        • Рекрутинг
        • The Regents of the University of California
    • Pennsylvania
      • Philadelphia, Pennsylvania, Соединенные Штаты, 19104
        • Рекрутинг
        • The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine

Критерии участия

Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.

Критерии приемлемости

Возраст, подходящий для обучения

8 лет и старше (Ребенок, Взрослый, Пожилой взрослый)

Принимает здоровых добровольцев

Нет

Полы, имеющие право на обучение

Все

Метод выборки

Невероятностная выборка

Исследуемая популяция

Adults and children with FOP who have been prescribed palovarotene, as per local label, by their treating physician for which informed consent has been obtained and maintained. Participants cannot be included if they are currently participating in a palovarotene clinical trial or an interventional clinical trial for FOP or if they have any contraindication for palovarotene (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).

Описание

Inclusion Criteria :

  • Adult or child with FOP who have been prescribed palovarotene (prior to and independently of the decision to enroll the patient in this registry study and as per local label) by their treating physician according to the locally approved product information;
  • Signed informed consent as per local regulations must be obtained and maintained. Consent/assent from the participant should be obtained as appropriate before any registry study data collection are conducted. If applicable, parents or legally authorized representatives must give signed informed consent.

Exclusion Criteria :

  • Currently participating in a palovarotene clinical trial;
  • Currently participating in any interventional clinical trial for FOP;
  • Have any contraindication to palovarotene as per the locally approved label (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).

Учебный план

В этом разделе представлена ​​подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.

Как устроено исследование?

Детали дизайна

  • Наблюдательные модели: OTHER
  • Временные перспективы: PROSPECTIVE

Когорты и вмешательства

Группа / когорта
Exposed Cohort

Participants treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information)
Unexposed Cohort

Participants untreated with palovarotene

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene
Временное ограничение: From Baseline up to 30 days after the last palovarotene dose.

Adverse events will be coded according to MedDRA and will be classified by Preferred Term (PT) and system organ class (SOC).
From Baseline up to 30 days after the last palovarotene dose.
Percentage of Participants With Serious and Non-serious treatment-related TEAEs
Временное ограничение: From Baseline up to 30 days after the last palovarotene dose.

Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
From Baseline up to 30 days after the last palovarotene dose.
Percentage of Participants With all serious TEAEs, whether or not they are considered as related to the palovarotene
Временное ограничение: From Baseline up to 30 days after the last palovarotene dose.

Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
From Baseline up to 30 days after the last palovarotene dose.
Percentage of Participants With nonserious TEAEs whether or not they are considered as related to the palovarotene.
Временное ограничение: From Baseline up to 30 days after the last palovarotene dose.

Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
From Baseline up to 30 days after the last palovarotene dose.

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Количество вспышек
Временное ограничение: От 12 месяцев до включения в базовый уровень до одиннадцати лет.
От 12 месяцев до включения в базовый уровень до одиннадцати лет.
Количество мест, затронутых на участника
Временное ограничение: От базового уровня и ежегодно до одиннадцати лет.
От базового уровня и ежегодно до одиннадцати лет.
Эволюция повлиявшего местоположения (сколько раз на местоположении затрагивается) на местоположение
Временное ограничение: От базового уровня и ежегодно до одиннадцати лет.
От базового уровня и ежегодно до одиннадцати лет.
Средняя доза/год паловаротина для участников с FOP, подвергнутым воздействию и непредубежденной на паловаротен для хронического лечения
Временное ограничение: От базового уровня и каждые шесть месяцев до одиннадцати лет.
От базового уровня и каждые шесть месяцев до одиннадцати лет.
Средняя доза/цикл паловаротина для участников с FOP, подвергнутым воздействию и непрерывной на паловаротин для вспышки лечения
Временное ограничение: От базового уровня и каждые шесть месяцев до одиннадцати лет.
От базового уровня и каждые шесть месяцев до одиннадцати лет.
Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score
Временное ограничение: From Baseline and every six months up to eleven years.

The CAJIS is an objective measure of joint movement completed by the Investigator to document total joint involvement. The CAJIS total score is calculated as the sum of the scores of all joints/regions and ranges from 0 (no involvement) to 30 (maximally involved).
From Baseline and every six months up to eleven years.
Change from Baseline in use of assistive devices
Временное ограничение: From Baseline and every six months up to eleven years.

Assistive devices is a questionnaire to assess the assistive devices used by particpants and adaptations for their daily living.
From Baseline and every six months up to eleven years.
Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score
Временное ограничение: From Baseline and every six months up to eleven years.

Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) consists of questions which are scored on a scale of 1 to 5, lower scores indicating that the participant has more difficulties.
From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)
Временное ограничение: From Baseline and every six months up to eleven years.

The lung function parameters of observed FVC (liters) and percent predicted FVC are obtained by spirometry.
From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1)
Временное ограничение: From Baseline and every six months up to eleven years.

The lung function parameters of observed FEV1 (liters) and percent predicted FEV1 are obtained by spirometry.
From Baseline and every six months up to eleven years.
Change from Baseline in absolute and percent predicted FEV1/FVC ratio
Временное ограничение: From Baseline and every six months up to eleven years.

The lung function parameters of the absolute and percent predicted FEV1/FVC ratio are obtained by spirometry.
From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
Временное ограничение: From Baseline and every six months up to eleven years.

The lung function parameters of observed (traditional unit of mL/min/mmHg or SI unit of mmol/min/kPa) and percent predicted DLCO is obtained by the DLCO test.
From Baseline and every six months up to eleven years.
Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) physical and mental function (mean global physical and mental health score converted into T-scores) for participants ≥15 years old
Временное ограничение: From Baseline and every six months up to eleven years.

PROMIS Global Health Scale for the adult version, the global physical health and global mental health scores will be calculated as follows:

  • Global physical health scores will be calculated as the sum of scores from Questions 3, 6, 7 and 8 and will range from 4 (worse health) to 20 (better health);
  • Global mental health scores will be calculated as the sum of scores from Questions 2, 4, 5 and 10 and will range from 4 (worse health) to 20 (better health).
From Baseline and every six months up to eleven years.
Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old
Временное ограничение: From Baseline and every six months up to eleven years.

PROMIS Global Health Scale for the paediatric version, the total score will be calculated as the sum of scores from the first 7 questions and will range from 7 (worse health) to 35 (better health).

If more than 3 questions contributing to the total score are missing, the total score will not be calculated. It will be considered as missing.
From Baseline and every six months up to eleven years.
Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall
Временное ограничение: From Baseline and every six months up to eleven years.

The annualized number of flare-ups will be derived from the number of flare-ups the participant experienced since last visit.
From Baseline and every six months up to eleven years.
Number of flare-up outcomes
Временное ограничение: From Baseline and every six months up to eleven years.

Number of flare-up outcomes as measured by new bone growth, restricted movement at each visit will be reported.
From Baseline and every six months up to eleven years.
Flare-up Duration
Временное ограничение: From Baseline and every six months up to eleven years.

Flare-up duration will be assessed by body location and overall, at each visit.
From Baseline and every six months up to eleven years.
Movement mobility change
Временное ограничение: From Baseline and annually up to eleven years.

Assessed by key body location: better movement/the same movement/slightly worse movement/moderately worse movement/severely worse movement)
From Baseline and annually up to eleven years.
Movement mobility outcomes: Percentage of participants with new bone growth overall and by flare-up status
Временное ограничение: From Baseline and every six months up to eleven years.

The number of extra bone growths, location of the extra bone growth, whether it was preceded by injury, illness, vaccination and/or other events, whether it was preceded by pain, soft tissue swelling, decreased range of motion, stiffness, redness, or warmth, extra bone growth start and stop date and whether it is ongoing, will be collected.
From Baseline and every six months up to eleven years.
Percentage of participants with any fractures overall
Временное ограничение: From baseline up to 30 days after the last palovarotene dose.

Assessed by radiological imaging and clinical examination.
From baseline up to 30 days after the last palovarotene dose.
Change from Baseline in annualized number of fractures by body location and overall
Временное ограничение: From baseline up to 30 days after the last palovarotene dose.

Assessed by radiological imaging and clinical examination.
From baseline up to 30 days after the last palovarotene dose.
Incidence of pregnancy
Временное ограничение: From Baseline and every month up to 30 days after the last palovarotene dose.

A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy and the health status of the baby will be verified
From Baseline and every month up to 30 days after the last palovarotene dose.
Description of pregnancy characteristics and outcomes
Временное ограничение: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.

A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy.
From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of pregnancy related Adverse Events (AEs)
Временное ограничение: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.

A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy.
From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of birth and developmental outcomes through the first year after birth
Временное ограничение: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.

A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy.
From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of birth and developmental outcomes through the first year after birth
Временное ограничение: From Baseline and every six months up to eleven years.

Linear height and knee height velocity will be derived for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
From Baseline and every six months up to eleven years.
Change from Baseline in height z-score
Временное ограничение: From Baseline and every six months up to eleven years.

Linear height z-score will be derived for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
From Baseline and every six months up to eleven years.
Mean difference between chronological age and bone age
Временное ограничение: From Baseline and every six months up to eleven years.

Bone age (assessment as per the SmPC/PI) and chronological age will be collected for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
From Baseline and every six months up to eleven years.
Frequency of premature physeal closure (PPC) overall
Временное ограничение: From baseline up to 30 days after the last palovarotene dose

Epiphyseal status ("open" or "closed") (assessment as per the SmPC/PI) will be collected for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
From baseline up to 30 days after the last palovarotene dose

Соавторы и исследователи

Здесь вы найдете людей и организации, участвующие в этом исследовании.

Спонсор

Ipsen

Следователи

  • Директор по исследованиям: Ipsen Medical, Director, Ipsen

Даты записи исследования

Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.

Изучение основных дат

Начало исследования (Действительный)

5 декабря 2024 г.

Первичное завершение (Оцененный)

5 декабря 2035 г.

Завершение исследования (Оцененный)

5 декабря 2035 г.

Даты регистрации исследования

Первый отправленный

6 октября 2023 г.

Впервые представлено, что соответствует критериям контроля качества

13 октября 2023 г.

Первый опубликованный (Действительный)

18 октября 2023 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

23 октября 2026 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

20 октября 2026 г.

Последняя проверка

1 октября 2026 г.

Дополнительная информация

Термины, связанные с этим исследованием

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • CLIN-60120-453

Планирование данных отдельных участников (IPD)

Планируете делиться данными об отдельных участниках (IPD)?

ДА

Описание плана IPD

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.

Сроки обмена IPD

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.

Критерии совместного доступа к IPD

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Информация о лекарствах и устройствах, исследовательские документы

Изучает лекарственный продукт, регулируемый FDA США.

Нет

Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.

Нет

Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .

Подписаться