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Gene Therapy
Gene Therapy (4D-710) for Cystic Fibrosis (CF Trial)
Phase 1 & 2
Recruiting
Research Sponsored by 4D Molecular Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Mutation Status: Bi-allelic mutations in the CFTR gene, or Single mutation in the CFTR gene and clinical manifestations of CF lung disease
18 years and older
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights
CF Trial Summary
This trial is testing a new gene therapy for adults with CF who can't take CFTR modulator therapy.
Who is the study for?
Adults with cystic fibrosis who can't take CFTR modulator therapy or had bad reactions to it. They must be 18+, have certain genetic mutations, and their lung function should be within a specific range. People with recent serious infections, liver disease, diabetes not well controlled, or those on chronic steroids/immunosuppressants can't join.Check my eligibility
What is being tested?
The trial is testing a single dose of an investigational gene therapy called 4D-710 in adults with cystic fibrosis. It's an early-stage study (Phase 1/2) to see how safe the treatment is and how the body responds to it.See study design
What are the potential side effects?
Since this is an early-phase trial for gene therapy, exact side effects are unknown but may include immune reactions, changes in blood tests like liver enzymes, potential for new allergies or worsening of existing ones due to genetic modification.
CF Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have either two mutations in the CFTR gene or one mutation with CF lung symptoms.
Select...
I am 18 years old or older.
Select...
I can't take CFTR modulator therapy or stopped it due to side effects.
Select...
I have been diagnosed with cystic fibrosis and lung disease related to CF.
CF Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence and severity of adverse events
CF Trial Design
5Treatment groups
Experimental Treatment
Group I: 4D-710 Dose Exploration Cohort 4Experimental Treatment1 Intervention
Single inhalational administration of 4D-710 Dose 4
Group II: 4D-710 Dose Exploration Cohort 3Experimental Treatment1 Intervention
Single inhalational administration of 4D-710 Dose 3
Group III: 4D-710 Dose Exploration Cohort 2Experimental Treatment1 Intervention
Single inhalational administration of 4D-710 Dose 2
Group IV: 4D-710 Dose Exploration Cohort 1Experimental Treatment1 Intervention
Single inhalational administration of 4D-710 Dose 1
Group V: 4D-710 Dose Expansion CohortExperimental Treatment1 Intervention
Single inhalational administration of 4D-710 at the selected dose
Find a Location
Who is running the clinical trial?
4D Molecular TherapeuticsLead Sponsor
7 Previous Clinical Trials
349 Total Patients Enrolled
Alan H. Cohen, MDStudy Director4D Molecular Therapeutics
Susan Limb, MDStudy Director4D Molecular Therapeutics
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have either two mutations in the CFTR gene or one mutation with CF lung symptoms.I have not had gene therapy before, except for mRNA-based treatments.I am 18 years old or older.I am being treated for a severe lung allergy with steroids or antifungal medication.I can't take CFTR modulator therapy or stopped it due to side effects.I am currently being treated for an active Mycobacterium abscessus infection.My last A1C level was above 7.5%.I have been diagnosed with cystic fibrosis and lung disease related to CF.My A1C level is 6.5% or higher and I don't have a known diabetes diagnosis.I've needed IV antibiotics for lung issues at least twice in the last 6 months.I cannot take systemic corticosteroids due to health reasons.I regularly take steroids or immunosuppressants for another condition.
Research Study Groups:
This trial has the following groups:- Group 1: 4D-710 Dose Exploration Cohort 3
- Group 2: 4D-710 Dose Exploration Cohort 4
- Group 3: 4D-710 Dose Exploration Cohort 1
- Group 4: 4D-710 Dose Exploration Cohort 2
- Group 5: 4D-710 Dose Expansion Cohort
Awards:
This trial has 1 awards, including:- 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.
Is there capacity for participants in this investigation?
"Indeed. According to clinicaltrials.gov, this scientific investigation is currently recruiting participants since its initial posting on March 29th 2022 and most recent update occurring September 21st 2022. Patients are being sought at 9 different trial sites with a total of 21 people required for enrollment."
Answered by AI
How many clinical venues are conducting this experiment?
"Presently, participants can enroll at 9 separate medical sites. These include Hershey, Denver and Richmond in addition to 6 other locations. To reduce travel strain, it is advisable to seek out the nearest clinic when selecting an enrollment site."
Answered by AI
How many subjects are being evaluated in this research endeavor?
"This clinical trial requires 21 suitable candidates for participation. Participating patients can be recruited at Penn State Health in Hershey, Pennsylvania and National Jewish Health in Denver, Colorado."
Answered by AI
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