Healthy Control for Primary Ciliary Dyskinesia

University of North Carolina Chapel Hill, Chapel Hill, NC
Primary Ciliary DyskinesiaAlbuterol - Drug
Eligibility
12 - 90
All Sexes

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.

Eligible Conditions
  • Primary Ciliary Dyskinesia

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase < 1

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Study Objectives

1 Primary · 1 Secondary · Reporting Duration: 150 minutes

120 minutes
Change in MCC (Ave120Clr-Ave60Clr;average clearance between 60 and 120 minutes)
150 minutes
Change in cough clearance (Ave150Clr-Ave120Clr; average clearance between 120 and 150 minutes)
60 minutes
Baseline MCC (Ave60Clr; average clearance over 60 minutes)

Trial Safety

Phase-Based Safety

1 of 3

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1
active auricular tVNS
1
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Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
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 10 Other Conditions
This treatment demonstrated efficacy for 10 other conditions.

Trial Design

3 Treatment Groups

Healthy Control
1 of 3
Genotypes associated with severe phenotype
1 of 3
Genotypes associated mild phenotype
1 of 3

Active Control

30 Total Participants · 3 Treatment Groups

Primary Treatment: Healthy Control · No Placebo Group · Phase < 1

Healthy ControlActiveComparator Group · 2 Interventions: Albuterol, Technetium99m - Sulfur Colloid (Tc99m-SC) · Intervention Types: Drug, DiagnosticTest
Genotypes associated with severe phenotypeActiveComparator Group · 2 Interventions: Albuterol, Technetium99m - Sulfur Colloid (Tc99m-SC) · Intervention Types: Drug, DiagnosticTest
Genotypes associated mild phenotypeActiveComparator Group · 2 Interventions: Albuterol, Technetium99m - Sulfur Colloid (Tc99m-SC) · Intervention Types: Drug, DiagnosticTest

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 150 minutes

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)NIH
3,674 Previous Clinical Trials
47,133,880 Total Patients Enrolled
University of North Carolina, Chapel HillLead Sponsor
1,400 Previous Clinical Trials
3,749,041 Total Patients Enrolled
Lawrence Ostrowski, PhDPrincipal InvestigatorUniversity of North Carolina at Chapel Hil

Eligibility Criteria

Age 12 - 90 · All Participants · 8 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Requirements for patients with Primary Ciliary Dyskinesia (PCD) to be included in the study.
You have been diagnosed with PCD and have confirmed genetic mutations.
If you are a female who has not had a hysterectomy with oophorectomy (removal of the uterus and ovaries), you need to have a negative pregnancy test.
You are able to exhale a minimum of 30% of the amount of air that is expected for someone of your age and size.
You are 18 years old or older.
You do not have any pre-existing lung conditions like asthma or cystic fibrosis.
Your lung function tests should show that your lungs are healthy and working at least as well as expected. If your test results are not normal, you will be given a copy of the test to share with your doctor.
Female participants who have not had their uterus and ovaries removed need to have a negative pregnancy test.
References

Frequently Asked Questions

Does the protocol of this research endeavor include adult participants?

"This study is open to those aged between 12 years and 90 years." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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" - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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