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Mucus Clearance Therapy for Primary Ciliary Dyskinesia

Phase < 1
Recruiting
Led By Lawrence Ostrowski, PhD
Research Sponsored by University of North Carolina, Chapel Hill
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 18 years old
Negative pregnancy test for females who are not s/p hysterectomy with oophorectomy.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 150 minutes
Awards & highlights

Study Summary

This trial is studying mucus clearance in people with a disease called Primary Ciliary Dyskinesia (PCD). It will compare different groups of people with PCD, some of which have mutations associated with a milder clinical phenotype. The hypothesis is that subjects with PCD caused by mutations in the milder group will have a lower, but significant, rate of mucus clearance, while patients with mutations in genes in the more severe group will have a complete absence of mucus clearance.

Who is the study for?
This trial is for adults over 18 with Primary Ciliary Dyskinesia (PCD) due to specific genetic mutations. Participants need a confirmed PCD diagnosis, normal lung function tests, and no other lung diseases like asthma or cystic fibrosis. Women must not be pregnant unless they've had certain surgeries.Check my eligibility
What is being tested?
The study measures how well mucus clears from the lungs in PCD patients with different gene mutations compared to healthy people. It aims to see if some genes cause milder symptoms by allowing some mucociliary clearance versus none at all, which could guide future treatments.See study design
What are the potential side effects?
Potential side effects of Albuterol include jitteriness, headache, rapid heart rate, and muscle cramps. Technetium99m - Sulfur Colloid may cause mild irritation where injected and has a rare risk of allergic reaction.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I am not pregnant or have had a hysterectomy with oophorectomy.
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I do not have any lung diseases like asthma or cystic fibrosis.
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I am a female who has not had a hysterectomy and my pregnancy test is negative.
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My condition is genetically confirmed Primary Ciliary Dyskinesia (PCD).

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~150 minutes
This trial's timeline: 3 weeks for screening, Varies for treatment, and 150 minutes for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Baseline MCC (Ave60Clr; average clearance over 60 minutes)
Secondary outcome measures
Change in MCC (Ave120Clr-Ave60Clr;average clearance between 60 and 120 minutes)
Other outcome measures
Change in cough clearance (Ave150Clr-Ave120Clr; average clearance between 120 and 150 minutes)

Trial Design

3Treatment groups
Active Control
Group I: Genotypes associated mild phenotypeActive Control2 Interventions
Subjects with 2 confirmed mutations in RSPH1, Radial Spoke Head Component 9 (RSPH9), Radial Spoke Head Component 4A (RSPH4a), or Dynein Axonemal Heavy Chain 11 (DNAH11). This group may also include subjects with mutations in newly identified genes that are associated with a milder clinical phenotype.
Group II: Genotypes associated with severe phenotypeActive Control2 Interventions
Subjects with 2 confirmed mutations in DNAH5, Dynein Axonemal Intermediate Chain 1 (DNAI1), Coiled-Coil Domain Containing 39 (CCDC39), or Coiled-Coil Domain Containing 40 (CCDC40). This group may also include subjects with mutations in newly identified genes that are associated with a more severe clinical phenotype.
Group III: Healthy ControlActive Control2 Interventions
Healthy subjects with no pre-existing lung disease.

Find a Location

Who is running the clinical trial?

University of North Carolina, Chapel HillLead Sponsor
1,508 Previous Clinical Trials
4,191,012 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,837 Previous Clinical Trials
47,851,564 Total Patients Enrolled
Lawrence Ostrowski, PhDPrincipal InvestigatorUniversity of North Carolina at Chapel Hil

Media Library

Primary Ciliary Dyskinesia Research Study Groups: Genotypes associated mild phenotype, Genotypes associated with severe phenotype, Healthy Control
Primary Ciliary Dyskinesia Clinical Trial 2023: Genotypes associated mild phenotype Highlights & Side Effects. Trial Name: NCT04901715 — Phase < 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the protocol of this research endeavor include adult participants?

"This study is open to those aged between 12 years and 90 years."

Answered by AI

What other experiments involving genotypes linked to extreme physical characteristics have been conducted?

"Currently, 17 trials concerning genotypes linked to severe phenotypes are underway with none of them at the Phase 3 level. These studies can be found across 75 clinical trial sites; a majority of which is based in San Francisco, California."

Answered by AI

What treatments are typically recommended for individuals with a particular genotype-to-phenotype correlation?

"Genotypes linked to severe phenotypes are useful for treating exercise-induced bronchospasm, as well as bronchodilator agents, respiratory infections, and other forms of related bronchial constriction."

Answered by AI

Who is eligible to take part in this clinical trial?

"This clinical trial is recruiting 30 people with asterixis over the age of 12 and under 90. Additionally, potential participants must adhere to certain eligibility criteria such as a confirmed PCD diagnosis with identified genetic mutations, being 18 years or older, not having any pre-existing lung disease (asthma, cystic fibrosis etc.), passing a negative pregnancy test for female patients who are neither post hysterectomy nor oophorectomy procedure and have an FEV1 score that is at least 30 percent of predicted value along with FVC/FEV1 ratio exceeding 80% of what was forecasted. Those whose results"

Answered by AI

Is this research program still recruiting participants?

"Verified. Clinicaltrials.gov has data showcasing that this medical experiment, which was firstly announced on June 10th 2021, is actively looking for patients to join its ranks. A total of 30 people are needed from a single location."

Answered by AI

How many individuals are formally participating in this clinical trial?

"Correct. According to the information available at clinicaltrials.gov, this investigation is actively searching for participants and was original posted on June 10th 2021. The trial has a goal of enrolling 30 people from one medical centre prior to its August 2022 update deadline."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Functional Studies of Novel Genes Mutated in Primary Ciliary Dyskinesia II: Genotype to Phenotype
2021. "Functional Studies of Novel Genes Mutated in Primary Ciliary Dyskinesia II: Genotype to Phenotype". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04901715.
~0 spots leftby Apr 2024
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