Cystic FibrosisPhase 1/2SAAVeNCT06526923
A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
A Phase 1/2 study testing SP-101 and doxorubicin Cohort 1 and SP-101 and doxorubicin Cohort 2 in people with Cystic Fibrosis, run by Spirovant Sciences, Inc. at 4 sites in United States.
Trial 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 more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- SP-101 and doxorubicin Cohort 1 and SP-101 and doxorubicin Cohort 2
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 4 sitesUnited States
- Study dates
- 16 Sep 2024to 31 Dec 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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
- Subjects with known sensitivity to SP-101, doxorubicin or its excipients
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, 02115
Monica Ulles, Monica.Ulles@childrens.harvard.edu
Ahmet Uluer
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Taryn McNickle, t425m975@kumc.edu
Joel Mermis
- Recruiting
Columbia University
New York, New York, 10032
Barbara Djan, bd2693@cumc.columbia.edu
Claire Keating
- Recruiting
Hospital at University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Melissa Molter, melissa.molter@pennmedicine.upenn.edu
Daniel Dorgan
About this study
Official title: 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.
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.
- Conditions
- Cystic Fibrosis
- Being tested
- SP-101 and doxorubicin Cohort 1, SP-101 and doxorubicin Cohort 2
- Keywords
- gene therapy, SP-101, AAV, doxorubicin, inhaled, augmenter
- Medical subject headings
- Cystic Fibrosis, Respiratory Aspiration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SP-101 and doxorubicin Cohort 1Combination product
Also called SP-101, doxorubicin
Single inhaled dose of SP-101 and doxorubicin Dose 1
SP-101 and doxorubicin Cohort 2Combination product
Also called SP-101, doxorubicin
Single inhaled dose of SP-101 and doxorubicin Dose 2
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Single inhalational administration of SP-101 and doxorubicin Dose 1 Receives: Combination Product: SP-101 and doxorubicin Cohort 1 |
| Cohort 2 | Experimental | Single inhalational administration of SP-101 and doxorubicin Dose 2 Receives: Combination Product: SP-101 and doxorubicin Cohort 2 |
| Dose Expansion | Experimental | Single inhalational administration of SP-101 and doxorubicin Selected Dose Receives: Combination Product: SP-101 and doxorubicin Cohort 1; Combination Product: SP-101 and doxorubicin Cohort 2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2024
- Study start16 Sep 2024
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Single ascending dose
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Spirovant Sciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jessica Lee, MPH, Study director, Spirovant Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06526923
- Sponsor's protocol ID
- CFAAV-001
- Acronym
- SAAVe
- Status last verified
- Nov 2024
- First submitted
- 18 Jul 2024
- Last updated
- 25 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
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 39155828Excoffon 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
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06526923
- Acronym / sponsor ID
- SAAVe / CFAAV-001
- Conditions
- Cystic Fibrosis
- Keywords
- gene therapy, SP-101, AAV, doxorubicin, inhaled, augmenter
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Spirovant Sciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Sep 2024 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 30 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Single ascending dose
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Combination Product: SP-101 and doxorubicin Cohort 1Single inhalational administration of SP-101 and doxorubicin Dose 1 |
| Cohort 2 | Experimental | Combination Product: SP-101 and doxorubicin Cohort 2Single inhalational administration of SP-101 and doxorubicin Dose 2 |
| Dose Expansion | Experimental | Combination Product: SP-101 and doxorubicin Cohort 1, Combination Product: SP-101 and doxorubicin Cohort 2Single inhalational administration of SP-101 and doxorubicin Selected Dose |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SP-101 and doxorubicin Cohort 1Also: SP-101, doxorubicinSingle inhaled dose of SP-101 and doxorubicin Dose 1 | Combination product | Cohort 1, Dose Expansion |
| SP-101 and doxorubicin Cohort 2Also: SP-101, doxorubicinSingle inhaled dose of SP-101 and doxorubicin Dose 2 | Combination product | Cohort 2, Dose Expansion |
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2024.
- Results publication: PubMed 39155828Excoffon 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.
- Results publication: PubMed 39155805Excoffon 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jessica Lee, MPH study director
Spirovant Sciences
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SP-101 and doxorubicin Cohort 1 and SP-101 and doxorubicin Cohort 2 in people with Cystic Fibrosis, run by Spirovant Sciences, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Spirovant.ClinicalTrials ((267) 805-6747, clinicaltrials@spirovant.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06526923, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Nov 2024.