A Study of Females With CF Throughout Pregnancy and Post-partum, and Follow up of Their Offspring

Официальное название

An Observational Study to Evaluate the Impact of Pregnancy and Parenthood in Females With Cystic Fibrosis and Their Offspring in the CFTR Modulator Era.

Обзор

In this study, investigators aim to study in detail the physical (including nutritional and pulmonary) and mental health of females with cystic fibrosis (CF) planning a pregnancy, during pregnancy, and in the early parenthood period. Additionally the health of offspring in infancy and early life will be studied to understand if further screening investigations or clinical care models should be part of clinical guidelines

Подробное описание

Improvements in treatments for people with CF have meant that more are becoming pregnant. CFTR modulators (CFTRm) are one of these treatments. They work by tackling the underlying cause of CF. These changes have created a need and an opportunity for research into the health and experiences of people with CF and their children in the CFTRm era.

The study is called 'MATRIARCH\_CF' and includes 3 related sub-studies:

'Mama' is enrolling participants aged 16 years or older with CF under the care of the Royal Brompton Hospital (RBH) adult CF Unit who are planning a pregnancy or pregnant. The aim is to describe the impact of pregnancy and the first 12-24 months of parenthood in females with CF on their physical and psychological health. Investigations in eight visits include blood tests, lung function, imaging, and interviews.

'Mini' is enrolling biological offspring of people with CF (mothers and fathers) cared for by the RBH Adult CF Service, from birth to age two. The aim is to collect information that will allow for assessment of health outcomes in offspring of parents with CF in the short term. There will be up to four visits over two years with investigations including blood tests, sweat tests, and brain ultrasound.

'Midi' explores the same question as 'Mini' but in the longer term for those aged three-to-six. There will be up to two visits, and they include lung function testing and a lung MRI.

This study is described as 'observational' as investigators will not provide or change any treatment. Participant's health will be monitored with a range of investigations, many of which are optional. Knowledge gained from this study will be used to create guidelines to help families with CF and their medical teams make decisions around pregnancy and their offspring.

Первичные конечные точки

• Change in FEV1 [Срок оценки: 9 months]
• Incidence of CF-related pulmonary complications during pregnancy. [Срок оценки: 9 months]
• Incidence of premature delivery [Срок оценки: 3 years]
• Frequency of liver dysfunction in infants [Срок оценки: 2 years]
• Incidence of congential abnormalities [Срок оценки: 3 years]
• Frequency of CF diagnoses in infants despite negative newborn screen testing [Срок оценки: 3 years]
• Frequency of abnormal lung clearance index results in young children [Срок оценки: 3 years]

Критерии участия

Критерии включения

'Mama' sub-study:

• Written informed consent obtained from participant.
• Under the care of Royal Brompton Hospital CF Reproductive and Maternal Health Service
• Age 16 years or above at time of recruitment
• Confirmed diagnosis of CF
• Planning a pregnancy or pregnant at time of recruitment.
• Ability to adhere to the required visits and investigations.

'Mini' sub-study:

• Written informed consent obtained from participant's legal guardian.
• Infants who have one biological parent with a confirmed diagnosis of CF under care of Royal Brompton Hospital Adult CF Service
• Less than 12 months of age at first visit.
• Ability to adhere to the required visits and investigations.

'Midi' sub-study:

• Written informed consent obtained from participant's legal guardian.
• Children who have one biological parent with a confirmed diagnosis of CF who is under the care of Royal Brompton Hospital Adult CF Service.
• Age three to six years at time of visit
• Ability to adhere to the required visits and investigations.

Критерии исключения

'Mama' sub-study:

• Any significant health condition which would cause inability to comply with protocol based on investigator discretion.
• History of lung transplantation

'Mini' sub-study:

• Legal guardians' inability to provide consent to participate in the study.
• Mother of infant has significant comorbidities unrelated to CF which could affect infant outcomes based on investigator discretion.

'Midi' sub-study:

• Legal guardians' inability to provide consent to participate in the study.
• A significant health condition which is known to affect lung function or imaging based on investigator discretion

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Великобритания · 1 центр

• Royal Brompton Hospital — London

Публикации

• Downes A, Bokobza I, Weitnauer L, Scott R, Dobra R, Short C, Semple T, Beaumont O, Williamson C, Davies JC, Felton I. Maternal, Infant, Reproductive and Child Health in Cystic Fibrosis (MATRIARCH_CF): a prospective, observational study to evaluate pregnancy and parenthood in females with cystic fibrosis and health of the offspring in the CFTR-modulator era. BMJ Open Respir Res. 2026 Jun 30;13(1):e PMID 42379640

Идентификаторы

NCT: NCT06797206 · 352398

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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