Cystic FibrosisPhase 1/2RESTORE-CFNCT03375047

Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis

A Phase 1/2 study testing MRT5005 and Normal saline in people with Cystic Fibrosis, run by Sanofi at 16 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This Phase 1/2, first-in-human study evaluated the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with cystic fibrosis (CF). Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: MRT5005 and Normal saline
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 42 participants
Where: 16 sitesUnited States
Study dates: 10 May 2018to 15 Mar 2022
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Confirmed diagnosis of CF as defined by both of the following:
Two CF disease-causing cystic fibrosis transmembrane conductance regulator (CFTR) mutations in Class I or II (genotype confirmed at the screening visit).
Chronic sinopulmonary disease and/or gastrointestinal/nutritional abnormalities consistent with CF disease.
Clinically stable CF disease, as judged by the investigator.
Forced expiratory volume in 1 second (FEV1) ≥50% and ≤90% of the predicted normal for age, gender, and height at screening.
Resting oxygen saturation ≥92% on room air (pulse oximetry).

Who cannot join

An acute upper or lower respiratory infection, pulmonary exacerbation, or clinically significant episode of hemoptysis or change in chronic respiratory medications (including antibiotics) for CF lung disease within 28 days prior to dosing with investigational product on Day 1.
Participants were receiving treatment with ivacaftor monotherapy (KALYDECO).
Parts A and B only: Were receiving treatment with triple combination therapy (TRIKAFTA).
Participants with a Class III, IV, or V CFTR gene mutation in at least 1 allele.
Infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus).
Treatment with ORKAMBI or SYMDEKO was not an exclusion for this study.

Trial locations and contacts

16 sites in 1 country.

City, postcode or hospital

16 sites in 1 country

United States16 sites

University of Michigan
Ann Arbor, Michigan, 48109
Johns Hopkins University
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Northwestern University
Chicago, Illinois, 60611
University of Cincinnati
Cincinnati, Ohio, 45267
University Hospitals
Cleveland, Ohio, 44106
Nationwide Children's Hospital
Columbus, Ohio, 43205
National Jewish Health
Denver, Colorado, 80206
University of Florida
Gainesville, Florida, 32610
University of Indiana
Indianapolis, Indiana, 46202
New Orleans Center for Clinical Research
Knoxville, Tennessee, 37920
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Maine Medical Center
Portland, Maine, 04102
Oregon Health and Sciences University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Utah
Salt Lake City, Utah, 84132

Other trials in United States

About this study

Official title: A Phase 1/2, Randomized, Double-Blinded, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 Administered by Nebulization to Adult Subjects With Cystic Fibrosis

Conditions: Cystic Fibrosis
Being tested: MRT5005, Normal saline
Keywords: Cystic Fibrosis, CF
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MRT5005Drug
Nebulization of MRT5005
Normal salineDrug
Normal Saline for Inhalation

Study groups in NCT03375047
Study group	Role	What they receive
Part A - SAD Group 1: MRT5005 8 mg	Experimental	Participants received single dose of MRT5005 8 milligrams (mg) by nebulization on Day 1. Receives: Drug: MRT5005
Part A - SAD Group 2: MRT5005 16 mg	Experimental	Participants received single dose of MRT5005 16 mg by nebulization on Day 1. Receives: Drug: MRT5005
Part A - SAD Group 3: MRT5005 20 mg	Experimental	Participants received single dose of MRT5005 20 mg by nebulization on Day 1. Receives: Drug: MRT5005
Part A - SAD Group 4: MRT5005 24 mg	Experimental	Participants received single dose of MRT5005 24 mg by nebulization on Day 1. Receives: Drug: MRT5005
Part A - SAD Groups: Pooled Placebo	Placebo comparator	Participants received single dose of placebo (normal saline) by nebulization on Day 1. Data were analyzed as a pooled population for all participants who received placebo in Part A SAD groups and reported in this arm. Receives: Drug: Normal saline
Part B - MAD Group 1: MRT5005 8 mg	Experimental	Participants received MRT5005 8 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Receives: Drug: MRT5005
Part B - MAD Group 2: MRT5005 12 mg	Experimental	Participants received MRT5005 12 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Receives: Drug: MRT5005
Part B - MAD Group 3: MRT5005 16 mg	Experimental	Participants received MRT5005 16 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Receives: Drug: MRT5005
Part B - MAD Group 4: MRT5005 20 mg	Experimental	Participants received MRT5005 20 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Receives: Drug: MRT5005
Part B - MAD Groups: Pooled Placebo	Placebo comparator	Participants received placebo (normal saline) once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Data were analyzed as a pooled population for all participants who received placebo in Part B MAD groups and reported in this arm. Receives: Drug: Normal saline
Part D: MRT5005 4 mg	Experimental	Participants received MRT5005 4 mg once daily by nebulization from Day 1 to Day 5. Receives: Drug: MRT5005
Part D: Placebo	Placebo comparator	Participants received placebo (normal saline) once daily by nebulization from Day 1 to Day 5. Receives: Drug: Normal saline

What is being measured

The primary outcome is the main question the study is designed to answer.

Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily had a causal relationship with the study treatment. A serious adverse event (SAE) was any untoward medical occurrence (whether considered to be related to study treatment or not) that at any dose: resulted in death; or was life-threatening; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital abnormality/birth defect; or was an important medical event. The TEAEs were defined as events that were newly reported or reported to worsen in severity after the start of study treatment up to 48 weeks (end of follow-up period) after the last dose of study treatment administration.
Measured: From the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341)

Secondary outcomes (1)

Parts A, B and D: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Spirometry was performed according to the guidelines published by the American Thoracic Society for standardization of spirometry. The ppFEV1 was assessed during the spirometry testing. For Parts A and B, the baseline value was defined as the average of the results from testing on Day -1 and at pre-dose on Day 1. For Part D, the baseline value was defined as the result from testing pre-dose on Day 1. Here, Baseline= BL, Day= D, pre-dose= PrD, hours post dose= hPD.
Measured: Part A:BL, D1(8hPD), D2(24hPD), D3, D8, D15 and D29; Part B:BL, D1(6hPD), D2, D8(PrD and 6hPD), D9, D15(PrD and 6hPD), D16, D22(PrD and 6hPD), D23, D29(PrD and 6hPD), D30, D36, D43 and D57; Part D:BL, D1(2hPD), PrD on D2, D3, D4 and D5, D11, D18 and D32

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

10 May 201815 Mar 2022

First posted15 Dec 2017
Study start10 May 2018
Primary completion15 Mar 2022
Study completion15 Mar 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
More trials run by SanofiThe sponsor's other registered studies.
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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 42 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03375047
Sponsor's protocol ID: MRT5005-101
Acronym: RESTORE-CF
Status last verified: Feb 2026
First submitted: 7 Sep 2017
Last updated: 9 Feb 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Publications

Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
PubMed 31215818

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2, Randomized, Double-Blinded, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 Administered by Nebulization to Adult Subjects With Cystic Fibrosis
ClinicalTrials.gov ID: NCT03375047
Acronym / sponsor ID: RESTORE-CF / MRT5005-101
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis, CF
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 42 participants (actual)
Lead sponsor: SanofiIndustry (pharma, biotech, devices)
Study start: 10 May 2018 (actual)
Primary completion: 15 Mar 2022 (actual)
Study completion: 15 Mar 2022 (actual)
First posted: 15 Dec 2017 (actual)
Results: Posted 9 Feb 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 12

Arm	Type	Receives
Part A - SAD Group 1: MRT5005 8 mg	Experimental	Drug: MRT5005Participants received single dose of MRT5005 8 milligrams (mg) by nebulization on Day 1.
Part A - SAD Group 2: MRT5005 16 mg	Experimental	Drug: MRT5005Participants received single dose of MRT5005 16 mg by nebulization on Day 1.
Part A - SAD Group 3: MRT5005 20 mg	Experimental	Drug: MRT5005Participants received single dose of MRT5005 20 mg by nebulization on Day 1.
Part A - SAD Group 4: MRT5005 24 mg	Experimental	Drug: MRT5005Participants received single dose of MRT5005 24 mg by nebulization on Day 1.
Part A - SAD Groups: Pooled Placebo	Placebo comparator	Drug: Normal salineParticipants received single dose of placebo (normal saline) by nebulization on Day 1. Data were analyzed as a pooled population for all participants who received placebo in Part A SAD groups and reported in this arm.
Part B - MAD Group 1: MRT5005 8 mg	Experimental	Drug: MRT5005Participants received MRT5005 8 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29).
Part B - MAD Group 2: MRT5005 12 mg	Experimental	Drug: MRT5005Participants received MRT5005 12 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29).
Part B - MAD Group 3: MRT5005 16 mg	Experimental	Drug: MRT5005Participants received MRT5005 16 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29).
Part B - MAD Group 4: MRT5005 20 mg	Experimental	Drug: MRT5005Participants received MRT5005 20 mg once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29).
Part B - MAD Groups: Pooled Placebo	Placebo comparator	Drug: Normal salineParticipants received placebo (normal saline) once weekly by nebulization for 5 weeks (i.e., on Day 1, Day 8, Day 15, Day 22 and Day 29). Data were analyzed as a pooled population for all participants who received placebo in Part B MAD groups and reported in this arm.
Part D: MRT5005 4 mg	Experimental	Drug: MRT5005Participants received MRT5005 4 mg once daily by nebulization from Day 1 to Day 5.
Part D: Placebo	Placebo comparator	Drug: Normal salineParticipants received placebo (normal saline) once daily by nebulization from Day 1 to Day 5.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Confirmed diagnosis of CF as defined by both of the following:
Clinically stable CF disease, as judged by the investigator.
Forced expiratory volume in 1 second (FEV1) ≥50% and ≤90% of the predicted normal for age, gender, and height at screening.
Resting oxygen saturation ≥92% on room air (pulse oximetry).

Key exclusion criteria (6 listed)

An acute upper or lower respiratory infection, pulmonary exacerbation, or clinically significant episode of hemoptysis or change in chronic respiratory medications (including antibiotics) for CF lung disease within 28 days prior to dosing with investigational product on Day 1.
Participants were receiving treatment with ivacaftor monotherapy (KALYDECO).
Parts A and B only: Were receiving treatment with triple combination therapy (TRIKAFTA).
Participants with a Class III, IV, or V CFTR gene mutation in at least 1 allele.
Infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus).

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
MRT5005Nebulization of MRT5005	Drug	Part A - SAD Group 1: MRT5005 8 mg, Part A - SAD Group 2: MRT5005 16 mg, Part A - SAD Group 3: MRT5005 20 mg, Part A - SAD Group 4: MRT5005 24 mg, Part B - MAD Group 1: MRT5005 8 mg, Part B - MAD Group 2: MRT5005 12 mg, Part B - MAD Group 3: MRT5005 16 mg, Part B - MAD Group 4: MRT5005 20 mg, Part D: MRT5005 4 mg
Normal salineNormal Saline for Inhalation	Drug	Part A - SAD Groups: Pooled Placebo, Part B - MAD Groups: Pooled Placebo, Part D: Placebo

Primary outcome

Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events
Time frame: From the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341)
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily had a causal relationship with the study treatment. A serious adverse event (SAE) was any untoward medical occurrence (whether considered to be related to study treatment or not) that at any dose: resulted in death; or was life-threatening; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital abnormality/birth defect; or was an important medical event. The TEAEs were defined as events that were newly reported or reported to worsen in severity after the start of study treatment up to 48 weeks (end of follow-up period) after the last dose of study treatment administration.

1 secondary outcome

Parts A, B and D: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: Part A:BL, D1(8hPD), D2(24hPD), D3, D8, D15 and D29; Part B:BL, D1(6hPD), D2, D8(PrD and 6hPD), D9, D15(PrD and 6hPD), D16, D22(PrD and 6hPD), D23, D29(PrD and 6hPD), D30, D36, D43 and D57; Part D:BL, D1(2hPD), PrD on D2, D3, D4 and D5, D11, D18 and D32

Results and safety

Results posted to the registry on 9 Feb 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part A - SAD Group 1: MRT5005 8 mg	3	3
Part A - SAD Group 2: MRT5005 16 mg	3	3
Part A - SAD Group 3: MRT5005 20 mg	3	2
Part A - SAD Group 4: MRT5005 24 mg	3	3
Part A - SAD Groups: Pooled Placebo	4	4
Part B - MAD Group 1: MRT5005 8 mg	3	3
Part B - MAD Group 2: MRT5005 12 mg	3	3
Part B - MAD Group 3: MRT5005 16 mg	4	3
Part B - MAD Group 4: MRT5005 20 mg	3	3
Part B - MAD Groups: Pooled Placebo	5	4
Part D: MRT5005 4 mg	6	6
Part D: Placebo	2	2

Primary outcome: Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events

Time frame: From the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341). Count of participants. Unit: Participants

Measure	Part A - SAD Group 1: MRT5005 8 mg n = 3	Part A - SAD Group 2: MRT5005 16 mg n = 3	Part A - SAD Group 3: MRT5005 20 mg n = 3	Part A - SAD Group 4: MRT5005 24 mg n = 3	Part A - SAD Groups: Pooled Placebo n = 4	Part B - MAD Group 1: MRT5005 8 mg n = 3	Part B - MAD Group 2: MRT5005 12 mg n = 3	Part B - MAD Group 3: MRT5005 16 mg n = 4	Part B - MAD Group 4: MRT5005 20 mg n = 3	Part B - MAD Groups: Pooled Placebo n = 5	Part D: MRT5005 4 mg n = 6	Part D: Placebo n = 2
Any TEAE	3	3	3	3	4	3	3	4	3	5	6	2
Any Serious TEAE	0	0	1	1	3	1	1	0	2	2	1	1

Population analysed: The Safety analysis set included all randomized participants who received study treatment.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Serious adverse events (AEs), other AEs and all-cause mortality (deaths) were collected from the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part A - SAD Group1: MRT5005 8 mg	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part A - SAD Group 2: MRT5005 16 mg	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part A - SAD Group 3: MRT5005 20 mg	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Part A - SAD Group 4: MRT5005 24 mg	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Part A - SAD Groups: Pooled Placebo	3 of 4 (75%)	4 of 4 (100%)	0 of 4
Part B - MAD Group 1: MRT5005 8 mg	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Part B - MAD Group 2: MRT5005 12 mg	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Part B - MAD Group 3: MRT5005 16 mg	0 of 4 (0%)	4 of 4 (100%)	0 of 4
Part B - MAD Group 3: MRT5005 20 mg	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3
Part B - MAD Groups: Pooled Placebo	2 of 5 (40%)	5 of 5 (100%)	0 of 5
Part D: MRT5005 4 mg	1 of 6 (16.7%)	6 of 6 (100%)	0 of 6
Part D: Placebo	1 of 2 (50%)	2 of 2 (100%)	0 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 31215818Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

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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. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

16 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 MRT5005 and Normal saline in people with Cystic Fibrosis, run by Sanofi at 16 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 6 inclusion and 6 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: finished, as of the sponsor's last update on 9 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 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. Some participants in this study may receive a placebo. 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?

Yes. Results are posted in the registry (first posted 9 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (time frame: From the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341)). 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 NCT03375047, which also lists 1 linked publication.

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 9 Feb 2026.