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RecruitingNCT06526923SAAVeUpdated Nov 25, 2024

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

A Phase 1/2 interventional study of SP-101 and doxorubicin Cohort 1 and SP-101 and doxorubicin Cohort 2 in Cystic Fibrosis, sponsored by Spirovant Sciences, Inc.. Recruiting at 4 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2024-11-25.

Sponsored by Spirovant Sciences, Inc. · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Started Sep 2024; still recruiting 2 years later.
Phase
Phase 1/2
Study type
Interventional
Enrollment
15
Allocation
Non-randomized
Ages
18 Years to 65 Years
Sex
All
01

Study summary

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.

Read the 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.

02

Conditions studied

  • Cystic Fibrosis

Keywords

  • gene therapy
  • SP-101
  • AAV
  • doxorubicin
  • inhaled
  • augmenter
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's planned enrollment of 15 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

This is the only study on the registry with Spirovant Sciences, Inc. as lead sponsor.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
15 participants (estimated)

Study arms

  • Experimental
    Cohort 1

    Single inhalational administration of SP-101 and doxorubicin Dose 1

    Combination Product: SP-101 and doxorubicin Cohort 1

  • Experimental
    Cohort 2

    Single inhalational administration of SP-101 and doxorubicin Dose 2

    Combination Product: SP-101 and doxorubicin Cohort 2

  • Experimental
    Dose Expansion

    Single inhalational administration of SP-101 and doxorubicin Selected Dose

    Combination Product: SP-101 and doxorubicin Cohort 1 · Combination Product: SP-101 and doxorubicin Cohort 2

Interventions

  • Combination productSP-101 and doxorubicin Cohort 1

    Single inhaled dose of SP-101 and doxorubicin Dose 1

    Also known as: SP-101, doxorubicin

  • Combination productSP-101 and doxorubicin Cohort 2

    Single inhaled dose of SP-101 and doxorubicin Dose 2

    Also known as: SP-101, doxorubicin

06

What researchers measure

Primary outcomes

  1. Incidence and severity of adverse events

    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.

    Time frame: 52 weeks

07

Study locations

4 of 4 sites recruiting
  • University of Kansas Medical Center
    Kansas City, Kansas 66160, United States
    Recruiting
  • Boston Children's Hospital, Brigham & Women's Hospital
    Boston, Massachusetts 02115, United States
    Recruiting
  • Columbia University
    New York, New York 10032, United States
    Recruiting
  • Hospital at University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
    Recruiting
08

References and documents

Publications

  • Excoffon KJDA, Smith MD, Falese L, Schulingkamp R, Lin S, Mahankali M, Narayan PKL, Glatfelter MR, Limberis MP, Yuen E, Kolbeck R. Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRDeltaR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis Ferrets. Hum Gene Ther. 2024 Sep;35(17-18):710-725. doi: 10.1089/hum.2024.064. Epub 2024 Sep 4. PubMed 39155828 ↗
  • Excoffon KJDA, Lin S, Narayan PKL, Sitaraman S, Jimah AM, Fallon TT, James ML, Glatfelter MR, Limberis MP, Smith MD, Guffanti G, Kolbeck R. 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. Hum Gene Ther. 2024 Sep;35(17-18):695-709. doi: 10.1089/hum.2024.063. Epub 2024 Aug 29. PubMed 39155805 ↗

Individual participant data

Plan to share: No

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 25, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06526923
Lead sponsor
Spirovant Sciences, Inc.
Responsible party
Sponsor
First posted
Jul 30, 2024
Start date
Sep 16, 2024
Primary completion
Dec 31, 2026 (estimated)
Completion
Dec 31, 2026 (estimated)
Last update
Nov 25, 2024

Study contacts

Spirovant.ClinicalTrials
Contact
clinicaltrials@spirovant.com
(267) 805-6747
Jessica Lee, MPH
study director · Spirovant Sciences

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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