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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

No Placebo-Only Group

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.

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.

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 150 minutes

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Baseline MCC (Ave60Clr; average clearance over 60 minutes)

Secondary study objectives

Change in MCC (Ave120Clr-Ave60Clr;average clearance between 60 and 120 minutes)

Other study objectives

Change in cough clearance (Ave150Clr-Ave120Clr; average clearance between 120 and 150 minutes)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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.

0 / 5Eligibility criteria met