Cystic Fibrosis (CF)NCT07841353
Impact of Trikafta on P2X7R and Clinical Outcomes in Cystic Fibrosis
An observational study of Cystic Fibrosis (CF), run by Ain Shams University at 1 site in Egypt.
Trial summary
The aim of this study is to assess the response of the ETI on P2X7R level as inflammatory marker and clinical outcomes in patients with cystic fibrosis. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Cystic Fibrosis (CF)
- How many people
- 35 participants
- Where
- 1 siteEgypt
- Study dates
- 25 Jul 2025to 31 Aug 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male and female pediatric and adult patients diagnosed with Cystic Fibrosis (CF) who are candidates for, or are currently initiating, treatment with Elexacaftor/Tezacaftor/Ivacaftor (ETI). Participants will be recruited from the outpatient Cystic Fibrosis clinic/pulmonology department at Children's Hospital, Ain Shams University Hospitals.
Who may be able to join
- Confirmed diagnosis of Cystic Fibrosis according to CF Foundation guidelines (based on positive sweat chloride test and/or presence of disease-causing mutations in homozygous state or compound heterozygous state in CFTR gene).
- Patients eligible for ETI therapy (having at least one F508del mutation or a mutation known to be responsive to ETI).
- Age above 5 years old.
Who cannot join
- Known allergy or hypersensitivity to ETI or any of its components.
- Moderate hepatic impairment (Child-Pugh Class B) or severe hepatic impairment (Child-Pugh Class C).
- Presence of chronic inflammatory diseases unrelated to CF (e.g., Crohn's disease, glomerulonephritis, or thrombotic disorders) that could confound P2X7R levels.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Impact of Elexacaftor/Tezacaftor/Ivacaftor on P2X7R as Inflammatory Marker and Clinical Outcomes in Patients With Cystic Fibrosis
The aim of this study is to assess the response of the ETI on P2X7R level as inflammatory marker and clinical outcomes in patients with cystic fibrosis.
Read the detailed description
The aim of this study is to assess the response of the ETI on P2X7R level as inflammatory marker and clinical outcomes including respiratory outcomes, gastrointestinal outcomes, anthropometric measurements in patients with cystic fibrosis. Written consent will be obtained from the legal guardians of the patients or the patients.The study will be 35 CF patients before starting ETI and same patients after 12 months of therapy .The data will be collected as Clinical parameters:Complete history taking including:sociodemographic data ( date of birth, gender, consanguinity and residency), age of diagnosis ,medication history,family history of sibling affection and history of sibling death,history of presenting symptoms as:respiratory symptoms(productive cough, expectoration, dyspnea , wheezes, chest pain),pancreatic function(failure to thrive, steatorrhea, thirst, and polyuria),gastrointestinal symptoms(abdominal pain, bloating and vomiting),symptoms of fat-soluble vitamins deficiency,detailed dietetic history, comorbidities(CF-related diabetes, GIT complications as constipation,and rectal prolapse, respiratory complications as hemoptysis, or pulmonary hypertension).Clinical examination including :complete general examination of the patients especially:respiratory rate, oxygen saturation by pulse oximetry on room air, clubbing,anthropometric measurements: including weight (kg), height (cm), body mass index (BMI) (kg/m²) will be measured and their Z-scores will be calculated according CDC growth charts at 6 and 12 months follow up and Full chest examination for signs of respiratory distress and the auscultatory findings.
- laboratory parameters:CBC,CRP,Liver function tests, sputum culture ,genotype (Homozygous or heterozygous Phe508del or any eligible genes for ETI( from medical records )), Human Purinergic Receptor P2X, Ligand Gated Ion Channel 7 (P2RX7), radiological assessment : low dose high-resolution CT (HRCT) of the chest,Pulmonary Function Test:Forced spirometry for cooperative patients with with comment mainly on FEV1 % Predicted and FEF25-75%Predicted and nutritional plan as macronutrients ,micronutrients and Pancreatic Enzyme Replacement Therapy.All of the following data will be collected before ETI and after 12months of treatment: presenting symptoms,all clinical assessment measures that were mentioned before,all laboratory investigations,pulmonary function tests and radiological assessment.
- Conditions
- Cystic Fibrosis (CF)
- Keywords
- Cystic Fibrosis, clinical outcomes, Trikafta, P2RX7, Cystic fibrosis, Clinical Outcomes, Trikafta, P2RX7
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in P2X7 Receptor (P2X7R) Expression Levels
Five milliliters of venous blood will be collected via venipuncture into serum-separating tubes. Serum levels of soluble P2X7 receptor (sP2X7R) will be quantified in picograms per milliliter (ng/L) using a commercial double-antibody sandwich enzyme-linked immunosorbent assay (ELISA) kit according to the manufacturer's instructions. A decrease in serum P2X7R levels indicates a reduction in systemic inflammatory activity following ETI treatment.
Measured: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)
Secondary outcomes (5)
Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%)
Evaluated via standard spirometry to assess pulmonary function response.
Measured: Baseline (pre-ETI initiation) and 6 and 12 months( post-ETI initiation)
Change in Body Mass Index (BMI)
Calculated as weight in kilograms divided by height in meters squared ({kg/m}^2).
Measured: Baseline (pre-ETI initiation) and 6 and 12 months( post-ETI initiation)
Change in BMI Z-Score
Standardized nutritional status score calculated relative to age- and sex-specific growth references.
Measured: Baseline (pre-ETI initiation) and 6 and 12 months( post-ETI initiation)
Change in Gastrointestinal Symptom Severity Score
Assessment of GI clinical status and symptom burden post-treatment.Assessment of gastrointestinal symptom burden using the Cystic Fibrosis Questionnaire-Abdomen (CFABD). Scores on the CFABD overall domains range from 0 to 100, where higher scores indicate worse symptom severity (greater GI burden).
Measured: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)
Change in Respiratory Symptom Severity Score
Assessment of Respiratory clinical status and symptom burden post-treatment.Assessment of respiratory symptom burden using the Cystic Fibrosis Respiratory Symptom Score (CRISS). Scores range from 0 to 100, where higher scores indicate worse symptom severity (greater respiratory burden).
Measured: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2026
- Study start25 Jul 2025
- Primary completion25 Jul 2026
- Study completion31 Aug 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis (CF)Studies of the same condition, open and under way.
- More trials run by Ain Shams UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mona R Hassan, M.D, Principal investigator, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07841353
- Sponsor's protocol ID
- MD150/2025
- Status last verified
- Sep 2026
- First submitted
- 8 Sep 2026
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Impact of Elexacaftor/Tezacaftor/Ivacaftor on P2X7R as Inflammatory Marker and Clinical Outcomes in Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT07841353
- Acronym / sponsor ID
- MD150/2025
- Conditions
- Cystic Fibrosis (CF)
- Keywords
- Cystic Fibrosis, clinical outcomes, Trikafta, P2RX7, Cystic fibrosis, Clinical Outcomes, Trikafta, P2RX7
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- 25 Jul 2025 (actual)
- Primary completion
- 25 Jul 2026 (actual)
- Study completion
- 31 Aug 2026 (actual)
- First posted
- 25 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to assess the response of the ETI on P2X7R level as inflammatory marker and clinical outcomes in patients with cystic fibrosis.
Detailed description
The aim of this study is to assess the response of the ETI on P2X7R level as inflammatory marker and clinical outcomes including respiratory outcomes, gastrointestinal outcomes, anthropometric measurements in patients with cystic fibrosis. Written consent will be obtained from the legal guardians of the patients or the patients.The study will be 35 CF patients before starting ETI and same patients after 12 months of therapy .The data will be collected as Clinical parameters:Complete history taking including:sociodemographic data ( date of birth, gender, consanguinity and residency), age of diagnosis ,medication history,family history of sibling affection and history of sibling death,history of presenting symptoms as:respiratory symptoms(productive cough, expectoration, dyspnea , wheezes, chest pain),pancreatic function(failure to thrive, steatorrhea, thirst, and polyuria),gastrointestinal symptoms(abdominal pain, bloating and vomiting),symptoms of fat-soluble vitamins deficiency,detailed dietetic history, comorbidities(CF-related diabetes, GIT complications as constipation,and rectal prolapse, respiratory complications as hemoptysis, or pulmonary hypertension).Clinical examination including :complete general examination of the patients especially:respiratory rate, oxygen saturation by pulse oximetry on room air, clubbing,anthropometric measurements: including weight (kg), height (cm), body mass index (BMI) (kg/m²) will be measured and their Z-scores will be calculated according CDC growth charts at 6 and 12 months follow up and Full chest examination for signs of respiratory distress and the auscultatory findings.
- laboratory parameters:CBC,CRP,Liver function tests, sputum culture ,genotype (Homozygous or heterozygous Phe508del or any eligible genes for ETI( from medical records )), Human Purinergic Receptor P2X, Ligand Gated Ion Channel 7 (P2RX7), radiological assessment : low dose high-resolution CT (HRCT) of the chest,Pulmonary Function Test:Forced spirometry for cooperative patients with with comment mainly on FEV1 % Predicted and FEF25-75%Predicted and nutritional plan as macronutrients ,micronutrients and Pancreatic Enzyme Replacement Therapy.All of the following data will be collected before ETI and after 12months of treatment: presenting symptoms,all clinical assessment measures that were mentioned before,all laboratory investigations,pulmonary function tests and radiological assessment.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male and female pediatric and adult patients diagnosed with Cystic Fibrosis (CF) who are candidates for, or are currently initiating, treatment with Elexacaftor/Tezacaftor/Ivacaftor (ETI). Participants will be recruited from the outpatient Cystic Fibrosis clinic/pulmonology department at Children's Hospital, Ain Shams University Hospitals.
Key inclusion criteria (3 listed)
- Confirmed diagnosis of Cystic Fibrosis according to CF Foundation guidelines (based on positive sweat chloride test and/or presence of disease-causing mutations in homozygous state or compound heterozygous state in CFTR gene).
- Patients eligible for ETI therapy (having at least one F508del mutation or a mutation known to be responsive to ETI).
- Age above 5 years old.
Key exclusion criteria (3 listed)
- Known allergy or hypersensitivity to ETI or any of its components.
- Moderate hepatic impairment (Child-Pugh Class B) or severe hepatic impairment (Child-Pugh Class C).
- Presence of chronic inflammatory diseases unrelated to CF (e.g., Crohn's disease, glomerulonephritis, or thrombotic disorders) that could confound P2X7R levels.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in P2X7 Receptor (P2X7R) Expression Levels
Time frame: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)
Five milliliters of venous blood will be collected via venipuncture into serum-separating tubes. Serum levels of soluble P2X7 receptor (sP2X7R) will be quantified in picograms per milliliter (ng/L) using a commercial double-antibody sandwich enzyme-linked immunosorbent assay (ELISA) kit according to the manufacturer's instructions. A decrease in serum P2X7R levels indicates a reduction in systemic inflammatory activity following ETI treatment.
5 secondary outcomes
Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%)
Time frame: Baseline (pre-ETI initiation) and 6 and 12 months( post-ETI initiation)
Change in Body Mass Index (BMI)
Time frame: Baseline (pre-ETI initiation) and 6 and 12 months( post-ETI initiation)
Change in BMI Z-Score
Time frame: Baseline (pre-ETI initiation) and 6 and 12 months( post-ETI initiation)
Change in Gastrointestinal Symptom Severity Score
Time frame: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)
Change in Respiratory Symptom Severity Score
Time frame: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mona R Hassan, M.D principal investigator
Ain Shams University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis (CF), run by Ain Shams University at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in P2X7 Receptor (P2X7R) Expression Levels (time frame: Baseline (pre-ETI initiation) and post-ETI initiation (12 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026.