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

NCT06526923 | Recruiting Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: CFAAV-001
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Cystic Fibrosis

Keywords

gene therapy; SP-101; AAV; doxorubicin; inhaled; augmenter

Outcome Measures

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.

  52 weeks

Study Arms (3)

Cohort 1

EXPERIMENTAL

Single inhalational administration of SP-101 and doxorubicin Dose 1

Combination Product: SP-101 and doxorubicin Cohort 1

Cohort 2

EXPERIMENTAL

Single inhalational administration of SP-101 and doxorubicin Dose 2

Combination Product: SP-101 and doxorubicin Cohort 2

Dose Expansion

EXPERIMENTAL

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

SP-101 and doxorubicin Cohort 1 COMBINATION_PRODUCT

Single inhaled dose of SP-101 and doxorubicin Dose 1

Also known as: SP-101, doxorubicin

Cohort 1; Dose Expansion

SP-101 and doxorubicin Cohort 2 COMBINATION_PRODUCT

Single inhaled dose of SP-101 and doxorubicin Dose 2

Also known as: SP-101, doxorubicin

Cohort 2; Dose Expansion

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females, age 18 to 65 years at Screening Visit, inclusive
• Diagnosis of CF
• ppFEV1 value between 50-100% (inclusive)
• 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

You may not qualify if:

• 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.
• 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
• Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
• Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
• History of solid organ or hematological transplantation
• History of clinically significant cirrhosis with or without portal hypertension
• History of pulmonary hypertension
• History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
• 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
• History of allergic bronchopulmonary aspergillosis (ABPA)
• Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c \>9% at Screening
• Clinically significant laboratory abnormalities at Screening
• 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
• Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
• Subjects who have previously received any gene therapy agent

Sponsors & Collaborators

• Spirovant Sciences, Inc.: lead

Study Sites (4)

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

Related Publications (2)

• 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.

  PMID: 39155828 RESULT

• 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.

  PMID: 39155805 RESULT

MeSH Terms

Conditions

Cystic Fibrosis; Respiratory Aspiration

Interventions

EGFR inhibitor SP101; Doxorubicin

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Respiration Disorders; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Daunorubicin; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates

Study Officials

• Jessica Lee, MPH

  Spirovant Sciences

  STUDY DIRECTOR

Central Study Contacts

Spirovant.ClinicalTrials

CONTACT

(267) 805-6747; clinicaltrials@spirovant.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single ascending dose

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: July 30, 2024
• Study Start: September 16, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: November 25, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)