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Gene Therapy Trial Report

Summary

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


NCTID NCT06526923 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Cystic Fibrosis
Disease Ontology Term DOID:1485
Compound Name SP-101
Compound Description AAV2.5T-SP183-hCFTRΔR
Sponsor Spirovant Sciences, Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 15 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant CFTRΔR
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Inhalational
Drug Product Type Viral vector
Target Tissue/Cell Human airway epithelia
Delivery System Viral transduction
Vector Type AAV2.5T
Editor Type none
Dose 1 Undisclosed dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-07-18
Completion Date 2026-12-31
Last Update 2024-11-25

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates First patient dosed November 2024

Resources/Links