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CFTR Modulator
Symdeko for Cystic Fibrosis
Phase < 1
Recruiting
Research Sponsored by University of Alabama at Birmingham
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Age ≥6 y.o.
Stable CF pulmonary regimen
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 32 weeks
Awards & highlights
Study Summary
This trial will test whether unapproved drugs can improve the function of CFTR mutations in people with CF.
Who is the study for?
This trial is for individuals with Cystic Fibrosis, aged 6 or older, who have specific CFTR mutations that might respond to certain drugs not yet approved for their condition. They must be on a stable CF treatment and able to give consent. People can't join if they have severe liver or kidney disease, had an organ transplant, are currently in another drug study, smoked recently, or take medications that don't mix well with the trial drugs.Check my eligibility
What is being tested?
The trial is testing Symdeko in patients with Cystic Fibrosis who have certain genetic mutations. It's designed to see if this medication can help even though it's not officially approved for these particular mutations.See study design
What are the potential side effects?
Symdeko may cause side effects such as headache, nausea, sinus congestion, dizziness and rash. More serious side effects could include elevated liver enzymes which would require monitoring by the healthcare team.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 6 years old or older.
Select...
My cystic fibrosis lung treatment has not changed recently.
Select...
I have been diagnosed with cystic fibrosis.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 32 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~32 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
spirometry
Side effects data
From 2022 Phase 3 trial • 458 Patients • NCT0404380625%
Cough
23%
Infective pulmonary exacerbation of cystic fibrosis
20%
Headache
16%
Upper respiratory tract infection
15%
Sputum increased
14%
Pyrexia
13%
Oropharyngeal pain
13%
Nasopharyngitis
11%
Nasal congestion
10%
Fatigue
10%
Viral upper respiratory tract infection
9%
Nausea
9%
Immunisation reaction
9%
Alanine aminotransferase increased
9%
Blood creatine phosphokinase increased
9%
Haemoptysis
8%
Rhinorrhoea
7%
Abdominal pain
7%
Diarrhoea
7%
COVID-19
7%
Sinusitis
7%
Aspartate aminotransferase increased
7%
Back pain
6%
Arthralgia
6%
Dyspnoea
6%
Productive cough
6%
Sinus congestion
6%
Rash
5%
Vomiting
5%
Pain
5%
Myalgia
5%
Respiration abnormal
2%
Pneumonia
1%
Distal intestinal obstruction syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part A: ELX/TEZ/IVA
Part B: ELX/TEZ/IVA
Trial Design
3Treatment groups
Experimental Treatment
Group I: SymdekoExperimental Treatment1 Intervention
Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern
Group II: OrkambiExperimental Treatment1 Intervention
Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern
Group III: IvacaftorExperimental Treatment1 Intervention
Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy.. Patients with a mutation equivalent to wild type will be given Ivacaftor.
Find a Location
Who is running the clinical trial?
University of Alabama at BirminghamLead Sponsor
1,589 Previous Clinical Trials
2,280,334 Total Patients Enrolled
22 Trials studying Cystic Fibrosis
41,069 Patients Enrolled for Cystic Fibrosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am 6 years old or older.My CFTR mutation could improve with specific approved treatments.I cannot use CFTR modulators due to conditions like advanced cirrhosis or ESRD.My cystic fibrosis lung treatment has not changed recently.I am not taking any medications that interact badly with others.I have been diagnosed with cystic fibrosis.You have smoked cigarettes within the last 6 months.I have had a solid organ transplant.I haven't needed antibiotics or steroids for a condition worsening for more than 28 days before joining this trial.You are currently taking part in a study for a drug that targets the CFTR gene.
Research Study Groups:
This trial has the following groups:- Group 1: Symdeko
- Group 2: Ivacaftor
- Group 3: Orkambi
Awards:
This trial has 3 awards, including:- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Approved for 5 Other Conditions - This treatment demonstrated efficacy for 5 other conditions.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there still positions available for participants in this investigation?
"According to the clinicaltrials.gov listing, this research is actively seeking participants. It was initially published on August 1st 2019 and underwent its most recent revision on August 30th 2022."
Answered by AI
Have there been any other experiments conducted with Symdeko?
"To date, 21 clinical trials for Symdeko are active. Of these studies, 14 have advanced to phase 3 and the participating medical centres span from Bochum to California with a total of 1015 sites."
Answered by AI
To what extent is the patient pool involved in this research experiment?
"Affirmative. Per the details provided on clinicaltrials.gov, this medical experiment is actively accepting participant applications with a start date of August 1st 2019 and most recent update as of August 30th 2022. At present, 20 individuals need to be enrolled from one research site."
Answered by AI
Does this clinical trial set a revolutionary precedent in the medical field?
"Vertex Pharmaceuticals Incorporated sponsored the initial Symdeko clinical trial which was completed in 2015 and involved 1044 participants. The drug's Phase 3 approval followed, leading to 21 active studies located across 23 countries with 109 cities participating in them."
Answered by AI
What conditions is Symdeko typically prescribed to treat?
"Symkeko is the recommended medication for homozygous f508del CTFR gene mutation, and can also be therapeutically effective with cystic fibrosis (CF), CFTR-related metabolic syndrome, those who are homozygous for f508del CFTM genes, and individuals whose mutations respond to ivacaftor."
Answered by AI
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