A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

Официальное название

An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)

Обзор

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.

Первичные конечные точки

• Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene [Срок оценки: From Baseline up to 30 days after the last palovarotene dose.]
• Percentage of Participants With Serious and Non-serious treatment-related TEAEs [Срок оценки: 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 [Срок оценки: 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. [Срок оценки: From Baseline up to 30 days after the last palovarotene dose.]

Вторичные конечные точки (12)

• Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score [Срок оценки: From Baseline and every six months up to eleven years.]
• Change from Baseline in use of assistive devices [Срок оценки: 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 [Срок оценки: From Baseline and every six months up to eleven years.]
• Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC) [Срок оценки: 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) [Срок оценки: From Baseline and every six months up to eleven years.]
• Change from Baseline in absolute and percent predicted FEV1/FVC ratio [Срок оценки: 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) [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: From Baseline and every six months up to eleven years.]
• Number of flare-up outcomes [Срок оценки: From Baseline and every six months up to eleven years.]
• Flare-up Duration [Срок оценки: From Baseline and every six months up to eleven years.]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

Канада · 3 центра

• Edmonton Clinic Health Academy (ECHA)- University of Alberta — Edmonton
• Bone Research and Education Centre — Oakville
• University Health Network (UHN) - Toronto General — Toronto

США · 2 центра

• The Regents of the University of California — San Francisco
• The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services — Philadelphia

Идентификаторы

NCT: NCT06089616 · CLIN-60120-453

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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