Actively Recruiting

Phase 1
Phase 2
Age: 18Years - 65Years
All Genders
ID06526923

A Single Ascending Dose, Phase 1/2 Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SP-101 Via Nebulizer for the Treatment of Cystic Fibrosis (CF)

Led by Spirovant Sciences, Inc. · Updated on 2024-11-25

15

Participants Needed

4

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating SP-101, an investigational gene therapy, in adults with cystic fibrosis (CF) who cannot use or tolerate CFTR modulator therapy. This Phase 1/2, first-in-human study aims to assess the safety, how the body processes the drug, and its biological effects with varying doses. The trial is open-label and conducted at multiple centers. Participants receive a single inhaled dose of SP-101 combined with doxorubicin at different dose levels. The study includes three groups: Dose 1, Dose 2, and a selected dose expansion group. Each participant will receive only one inhalation treatment during the trial. During the study, researchers will monitor participants for adverse events over 52 weeks to evaluate safety. They will also measure how the drug behaves in the body and its impact. Participants will have regular assessments including lung function tests and oxygen levels. The trial lasts about a year, during which participants will be closely observed and evaluated.

CONDITIONS

Brief Title

A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

Who Can Participate

Age: 18Years - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Males or females aged 18 to 65 years at Screening Visit, inclusive
  • Diagnosis of cystic fibrosis
  • ppFEV1 value between 50-100% (inclusive)
  • Resting oxygen saturation of 94% or higher on room air by pulse oximetry
  • Clinically stable cystic fibrosis disease not requiring new interventional treatment within 3 months prior to Screening
Not Eligible

You will not qualify if you...

  • Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening, except inhaled beta-agonists within 2 weeks prior
  • Clinically significant hemoptysis (>50 mL or 1/4 cup per day) within 12 weeks prior to dosing
  • Lung infection with Mycobacterium abscessus causing rapid decline
  • Active treatment for lung infection with Burkholderia cenocepacia or Burkholderia dolosa
  • History of solid organ or blood transplantation
  • History of clinically significant cirrhosis with or without portal hypertension
  • History of pulmonary hypertension
  • History of cardiotoxicity, coronary artery disease, or cardiomyopathy
  • Current active fungal infection, acute blood, lung, or bladder infection, significant liver or kidney dysfunction, or viral infection requiring new therapy within 30 days prior
  • History of allergic bronchopulmonary aspergillosis (ABPA)
  • Uncontrolled diabetes with hemoglobin A1c >9% at Screening
  • Clinically significant lab abnormalities at Screening
  • Any medical condition or lab result interfering with safe study completion
  • Receipt of any investigational products within 30 days or 5 half-lives prior to Screening
  • Prior gene therapy
  • Known sensitivity to SP-101, doxorubicin, or excipients

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Single dose with follow-up up to 52 weeks

Participants receive a single inhaled dose of SP-101 and doxorubicin to evaluate safety and pharmacokinetics.

1 treatment visit and multiple follow-up visits over 52 weeks

Trial Site Locations

Total: 4 locations

1

University of Kansas Medical Center

Kansas City, Kansas, United States, 66160

Actively Recruiting

2

Boston Children's Hospital, Brigham & Women's Hospital

Boston, Massachusetts, United States, 02115

Actively Recruiting

3

Columbia University

New York, New York, United States, 10032

Actively Recruiting

4

Hospital at University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

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Research Team

S

Spirovant.ClinicalTrials

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

3

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Published Research Related To This Trial

Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRΔR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis Ferrets.

Katherine J D A Excoffon, Mark D Smith, Lillian Falese...

https://pubmed.ncbi.nlm.nih.gov/39155828

SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis.

Katherine J D A Excoffon, Shen Lin, Poornima Kotha Lakshmi Narayan...

https://pubmed.ncbi.nlm.nih.gov/39155805