RecruitingPhase 2interventionalRules rewrite

Phase 2: testing SPL84 for Cystic Fibrosis

The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation (3849 +10kb C--\>T).

Official registry: https://clinicaltrials.gov/study/NCT06429176 · SpliSense Ltd. · enrollment listed as 64

What this phase means

Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are SPL84, or Placebo. The registry lists allocation as randomized. The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF).

They plan to enroll about 64 people at 27 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

A Phase 2a, Randomized, Placebo-Controlled, Double Blind Multiple Ascending Dose Study in Patients With Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84

Interventions: SPL84, Placebo

Primary outcome (registry wording): Safety and Tolerability of SPL84 as evaluated by number of subjects with at least one treatment-related adverse event (AE) or serious adverse event (SAEs)

Who it is for (from inclusion criteria)

Ages 18 Years to any
Diagnosis of CF and two CF causing mutations; 3849+10 Kb C-\>T mutation on one allele in the CF transmembrane conductance regulator (CFTR) gene (homozygote or compound heterozygote). Source documentation from a certified genetic laboratory is required
Body mass index (body-mass index (BMI)) of ≥ 17 kg/m2
FEV1 40-90% predicted at screening
Non-smokers or vapers for at least 180 days (6 months) prior to screening, per participant report

Who the registry excludes

Use of Kalydeco, Orkambi, Symdeko/Symkevi or Trikafta/Kaftrio within 30 days of first dose with study intervention
Use of any investigational drug (other than SPL84) or device within 30 days of first dose with study intervention
Use of systemic steroids over 3 consecutive months in the last 6 months prior to screening, or use of systemic steroids in the last month prior to screening. Use of inhaled steroids above 1 mg
Hemoptysis of greater than 30 mL within 90 days prior to Day 1, or hospitalization for hemoptysis within 6 months of first dose with study intervention
Liver disease characterized by clinically significant cirrhosis and/or documented portal hypertension
History of any organ transplantation
Documented coronavirus disease (COVID-19) infection within 4 weeks prior to dosing

Contact

No central phone on the registry card. Use the official listing to reach a site.

Sites listed on ClinicalTrials.gov

27 listed

Memorial Care Long Beach Medical Center — Long Beach, California (recruiting)
University of Southern California — Los Angeles, California (recruiting)
University of California, San Francisco — San Francisco, California (recruiting)
University of California Los Angeles — Santa Monica, California (not_yet_recruiting)
National Jewish Health — Denver, Colorado (recruiting)
Joe DiMaggio Children's Hospital — Hollywood, Florida (not_yet_recruiting)
Northwestern University — Chicago, Illinois (not_yet_recruiting)
Boston Children'S Hospital — Boston, Massachusetts (recruiting)
University of Minnesota — Minneapolis, Minnesota (not_yet_recruiting)
Lenox Hill Cystic Fibrosis Center — New York, New York (recruiting)
Columbia University Cystic Fibrosis Program — New York, New York (not_yet_recruiting)
University of North Carolina at Chapel Hill — Chapel Hill, North Carolina (not_yet_recruiting)

+ 11 sites outside the US on the same record

Related recruiting trials in this snapshot

RecruitingNot a traditional drug phaseBethesda, Maryland

Von Hippel-Lindau (VHL): Clinical Manifestations, Diagnosis, Management and Molecular Bases of Inherited Re…

We will investigate the clinical manifestations and molecular genetic defects of heritable urologic malignant disorders. Families with urologic malignancy with known or suspected genetic basis will be enrolled.

NCT00001238 · Official ClinicalTrials.gov record · National Cancer Institute (NCI)

RecruitingNot a traditional drug phaseBethesda, Maryland

Study of Systemic Lupus Erythematosus

This protocol will evaluate patients with systemic lupus erythematosus (SLE) and their relatives to learn more about how the disease develops and changes over time. It will also study genetic factors that make a person susceptible to SLE.

NCT00001372 · Official ClinicalTrials.gov record · National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

RecruitingNot a traditional drug phaseWashington D.C., District of Columbia

Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics

This study is designed to explore the genetics and pathophysiology of diseases presenting with intermittent fever, including familial Mediterranean fever, TRAPS, hyper-IgD syndrome, and related diseases. The following individuals may be eligible for this natural history study: 1) patients with known or suspected familial Mediterranean fever, TRAPS, hyper-IgD syndrome or related disorders; 2) relatives of these patients; 3) healthy, normal volunteers 7 years of age or older.

NCT00001373 · Official ClinicalTrials.gov record · National Human Genome Research Institute (NHGRI)

FAQ about this NCT record

Is NCT06429176 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT06429176?

Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT06429176?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary