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

November 21, 2024 updated by: Spirovant Sciences, Inc.

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)

This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This multi-center study is a first-in-human, single ascending dose, Phase 1/2 trial to evaluate the safety, pharmacokinetics, and pharmacodynamics of various dose levels in people with CF who are ineligible or intolerant to CFTR modulator therapy.

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
        • Principal Investigator:
          • Joel Mermis
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
    • New York
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University
        • Contact:
        • Principal Investigator:
          • Claire Keating
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Males or females, age 18 to 65 years at Screening Visit, inclusive
  2. Diagnosis of CF
  3. ppFEV1 value between 50-100% (inclusive)
  4. Resting oxygen saturation ≥94% on room air by pulse oximetry 5 . Clinically stable CF disease as assessed by the Investigator and not requiring any new class of interventional treatment within the last 3 months prior to Screening

Exclusion Criteria:

  1. Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening Visit. However, inhaled beta-agonists can be included within 2 weeks prior to Screening Visit.
  2. Clinically significant episode of hemoptysis (>50 mL or ¼ cup or 10 teaspoons per day) within 12 weeks prior to dosing with study drug on Day 1
  3. Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
  4. Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
  5. History of solid organ or hematological transplantation
  6. History of clinically significant cirrhosis with or without portal hypertension
  7. History of pulmonary hypertension
  8. History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
  9. Current active fungal infection (not just a positive culture), acute blood, lung, or bladder infection, clinically significant hepatic or renal dysfunction, and/or viral infection (including human immunodeficiency virus or hepatitis virus B or C) requiring the initiation of new therapy within 30 days prior to Screening
  10. History of allergic bronchopulmonary aspergillosis (ABPA)
  11. Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c >9% at Screening
  12. Clinically significant laboratory abnormalities at Screening
  13. Subjects with any medical condition or abnormal laboratory result that, in the opinion of the Investigator, will interfere with the safe completion of the study
  14. Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
  15. Subjects who have previously received any gene therapy agent
  16. Subjects with known sensitivity to SP-101, doxorubicin or its excipients

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Single inhalational administration of SP-101 and doxorubicin Dose 1
Combination product: SP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
Other Names:
  • doxorubicin
  • SP-101
Experimental: Cohort 2
Single inhalational administration of SP-101 and doxorubicin Dose 2
Combination product: SP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Other Names:
  • doxorubicin
  • SP-101
Experimental: Dose Expansion
Single inhalational administration of SP-101 and doxorubicin Selected Dose
Combination product: SP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
Other Names:
  • doxorubicin
  • SP-101
Combination product: SP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Other Names:
  • doxorubicin
  • SP-101

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence and severity of adverse events
Time Frame: 52 weeks
Safety and tolerability of SP-101 following a single inhalation dose, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Spirovant Sciences, Inc.

Investigators

  • Study Director: Jessica Lee, MPH, Spirovant Sciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 16, 2024

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2026

Study Registration Dates

First Submitted

July 18, 2024

First Submitted That Met QC Criteria

July 24, 2024

First Posted (Actual)

July 30, 2024

Study Record Updates

Last Update Posted (Estimated)

November 25, 2024

Last Update Submitted That Met QC Criteria

November 21, 2024

Last Verified

November 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CFAAV-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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