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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05097989




Registration number
NCT05097989
Ethics application status
Date submitted
18/10/2021
Date registered
28/10/2021

Titles & IDs
Public title
Study of ALXN2050 in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
Scientific title
A Phase 2, Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Evaluate the Efficacy and Safety of ALXN2050 in Adult Participants With Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
Secondary ID [1] 0 0
ALXN2050-NEPH-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lupus Nephritis 0 0
Immunoglobulin A Nephropathy 0 0
IgAN 0 0
LN 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ALXN2050
Treatment: Drugs - Placebo

Experimental: LN Cohort: ALXN2050 180 mg - Participants diagnosed with LN with an active flare will receive ALXN2050 in addition to standard-of-care background therapy.

Experimental: LN Cohort: ALXN2050 120 mg - Participants diagnosed with LN with an active flare will receive ALXN2050 in addition to standard-of-care background therapy.

Placebo comparator: LN Cohort: Placebo - Participants diagnosed with LN with an active flare will receive matched placebo in addition to standard-of-care background therapy.

Experimental: IgAN Cohort: ALXN2050 180 mg - Participants diagnosed with IgAN will receive ALXN2050 in addition to standard-of-care background therapy.

Experimental: IgAN Cohort: ALXN2050 120 mg - Participants diagnosed with IgAN will receive ALXN2050 in addition to standard-of-care background therapy.

Placebo comparator: IgAN Cohort: Placebo - Participants diagnosed with IgAN will receive matched placebo in addition to standard-of-care background therapy.


Treatment: Drugs: ALXN2050
Oral tablets

Treatment: Drugs: Placebo
Oral tablets
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Both Cohorts: Percentage Change In Proteinuria From Baseline To Week 26
Timepoint [1] 0 0
Baseline, Week 26
Secondary outcome [1] 0 0
Both Cohorts: Percentage Change In Proteinuria From Baseline To Week 50
Timepoint [1] 0 0
Baseline, Week 50
Secondary outcome [2] 0 0
Both Cohorts: Participants Achieving > 30% And > 50% Reduction In Proteinuria At Week 26 And Week 50 Compared To Baseline
Timepoint [2] 0 0
Baseline, Week 26 and Week 50
Secondary outcome [3] 0 0
Both Cohorts: Change From Baseline In Estimated Glomerular Filtration Rate (eGFR) At Week 26 And Week 50
Timepoint [3] 0 0
Baseline, Week 26 and Week 50
Secondary outcome [4] 0 0
LN Cohort: Participants Meeting The Criteria For Complete Renal Response At Week 26 And Week 50
Timepoint [4] 0 0
Week 26 And Week 50
Secondary outcome [5] 0 0
LN Cohort: Participants Meeting The Criteria For Partial Renal Response At Week 26 And Week 50
Timepoint [5] 0 0
Week 26 And Week 50
Secondary outcome [6] 0 0
LN Cohort: Time To The First Occurrence Of Urine Protein To Creatinine Ratio (UPCR) = 0.5 Gram/Gram (g/g) As Measured By Spot Urine Sample
Timepoint [6] 0 0
Up to Week 50
Secondary outcome [7] 0 0
LN Cohort: Participants Achieving Corticosteroid Taper To 7.5 mg/Day At Weeks 12, 26, And 50
Timepoint [7] 0 0
Week 12, Week 26, And Week 50
Secondary outcome [8] 0 0
LN Cohort: Participants Experiencing A Renal Flare Through Week 50
Timepoint [8] 0 0
Baseline through Week 50
Secondary outcome [9] 0 0
LN Cohort: Participants Experiencing An Extrarenal Systemic Lupus Erythematosus (SLE) Flare Through Week 50
Timepoint [9] 0 0
Baseline through Week 50
Secondary outcome [10] 0 0
LN Cohort: Participants Meeting The Criteria For Treatment Failure Through Week 50
Timepoint [10] 0 0
Baseline through Week 50
Secondary outcome [11] 0 0
LN Cohort: Absolute Values And Change From Baseline In Serum Albumin At Week 26 And Week 50
Timepoint [11] 0 0
Baseline, Week 26 and Week 50
Secondary outcome [12] 0 0
IgAN Cohort: Participants Meeting The Criteria For Partial Remission At Week 26 And Week 50
Timepoint [12] 0 0
Week 26 and Week 50
Secondary outcome [13] 0 0
Both Cohorts: Observed Plasma Concentrations Of ALXN2050 Over Time
Timepoint [13] 0 0
Baseline through Week 50
Secondary outcome [14] 0 0
Both Cohorts: Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 50
Timepoint [14] 0 0
Baseline, Week 50
Secondary outcome [15] 0 0
Both Cohorts: Absolute Values And Change From Baseline In Serum Alternative Pathway Activity At Week 50
Timepoint [15] 0 0
Baseline, Week 50

Eligibility
Key inclusion criteria
Key

Both Cohorts

* Participants on sodium-glucose cotransporter-2 (SGLT 2) inhibitors (eg, empagliflozin) must be on a stable dose for = 3 months with no planned change in dose during the Blinded Treatment Periods (through Week 50).

LN Cohort

* Clinical diagnosis of SLE by 2019 American College of Rheumatology and European League Against Rheumatism criteria.
* Diagnosis of 2018 Revised International Society of Nephrology/Renal Pathology Society classification (active focal or diffuse proliferative LN Class III or IV) confirmed by biopsy obtained = 6 months prior to Screening or during Screening Period. Participants may co-exhibit Class V disease. Participants with de novo or relapsing disease may be eligible.
* Clinically active LN at Screening requiring/receiving immunosuppression induction treatment in the opinion of the Investigator.
* Proteinuria with UPCR = 1 g/g based on one 24 hour urine collection during the Screening Period.

IgAN Cohort

* Established diagnosis of primary IgAN based on kidney biopsy obtained any time prior to or during the Screening Period.
* Mean proteinuria = 1 g/day on 2 complete and valid 24 hour urine collections during the Screening Period.
* For participants with a kidney biopsy performed > 1 year prior to Screening that was used for eligibility: Presence of hematuria as defined by a positive result for blood on urine dipstick or = 10 red blood cells (RBCs)/high power field (hpf) microscopy on urine sediment (documented by the local laboratory) during Screening Period. Presence of hematuria documented by the central laboratory may also be acceptable.
* Compliance with stable and optimal dose of RAS inhibitor treatment including maximum allowed or tolerated ACE inhibitor and/or ARB dose for = 3 months prior to Screening with no expected change in dose during the Blinded Treatment Periods (through Week 50) (participants with established intolerance to RAS inhibitors may be included).
* Controlled and stable blood pressure (defined as < 140/90 millimeters of mercury [mmHg]) over the past 3 months prior to randomization.

Key
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Both Cohorts

* eGFR = 30 milliliters/minute/1.73 squared meters during Screening calculated by Chronic Kidney Disease Epidemiology Collaboration.
* For participants with eGFR < 45 mL/min/1.73 m2 at Screening, presence of any of the following in glomeruli on most recent kidney biopsy prior to or during the Screening

Period:

1. = 50% interstitial fibrosis and tubular atrophy
2. = 50% glomerular sclerosis
3. = 50% active crescent formation

* Concomitant significant renal disease other than LN or IgAN on the most recent biopsy prior to or during the Screening Period.
* History of solid organ or bone marrow transplant, or planned transplant during the Blinded Extended Treatment Period (50 weeks).
* Splenectomy or functional asplenia.
* Known or suspected complement deficiency, unless attributable to underlying disease (that is, LN and IgAN).
* Bone marrow insufficiency with absolute neutrophil count < 1.3 × 10^3/microliter; thrombocytopenia (platelet count < 50,000/cubic millimeter).

LN Cohort

* Participants who have initiated any of the following treatments for the current active LN flare:

1. Cyclophosphamide = 6 months prior to Screening
2. CNIs = 1 months prior to Screening
3. A cumulative dose of intravenous (IV) methylprednisolone > 3 g
4. Mycophenolate mofetil > 2 g/day (or equivalent) for = 8 consecutive weeks prior to Screening
5. Prednisone or prednisone equivalent = 0.5 mg/kg/day for = 8 consecutive weeks prior to Screening
* Uncontrolled hypertension (systolic blood pressure > 160 mmHg or diastolic blood pressure > 110 mmHg) on 2 or more measurements during the Screening Period.
* Prior history or clinically active SLE-related cerebritis, seizures, stroke, or stroke syndrome requiring treatment or clinically active pericarditis
* Inability to take or tolerate the standard of care background therapies

IgAN Cohort

* Diagnosis of rapid progressive glomerulonephritis as measured by eGFR loss = 30% over a period of 3 months prior to or during the Screening Period.
* Secondary etiologies of IgAN.
* Prednisone or prednisone equivalent > 20 mg/day for > 14 consecutive days or any other systemic immunosuppression for the treatment of IgAN = 6 months prior to Screening
* Blood pressure of = 140/90 mmHg during the Screening Period confirmed on 2 measures > 30 minutes apart.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/01/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/06/2026
Actual
Sample size
Target
70
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Clayton
Recruitment hospital [2] 0 0
Research Site - Heidelberg
Recruitment hospital [3] 0 0
Research Site - Liverpool
Recruitment hospital [4] 0 0
Research Site - Nedlands
Recruitment hospital [5] 0 0
Research Site - St Leonards
Recruitment hospital [6] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3084 - Heidelberg
Recruitment postcode(s) [3] 0 0
2170 - Liverpool
Recruitment postcode(s) [4] 0 0
6009 - Nedlands
Recruitment postcode(s) [5] 0 0
2065 - St Leonards
Recruitment postcode(s) [6] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
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California
Country [4] 0 0
United States of America
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Florida
Country [5] 0 0
United States of America
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Idaho
Country [6] 0 0
United States of America
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Iowa
Country [7] 0 0
United States of America
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Missouri
Country [8] 0 0
United States of America
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New Mexico
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United States of America
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New York
Country [10] 0 0
United States of America
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Texas
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Argentina
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Ciudad Autonoma Bs As
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Argentina
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Córdoba
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Argentina
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La Plata
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Argentina
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Mendoza
Country [15] 0 0
Argentina
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Rosario
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Argentina
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San Miguel de Tucuman
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Brazil
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Belo Horizonte
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Brazil
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Curitiba
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Brazil
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Fortaleza
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Brazil
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Juiz de Fora
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Brazil
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Porto Alegre
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Brazil
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Rio de Janeiro
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Brazil
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Salvador
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Brazil
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Santo Andre
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Brazil
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Sao Paulo
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Brazil
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Vitória
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Chuangchun
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China
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Guangzhou
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China
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McKinney
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China
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Nanchang
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China
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Shanghai
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Germany
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Berlin
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Germany
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Essen
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Germany
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Fulda
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Köln
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Luebeck
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Ashkelon
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Bari
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Nis
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Serbia
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Novi Sad
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Avilés
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Barcelona
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Cordoba
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Girona
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L'Hospitalet de Llobregat
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Spain
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Madrid
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Malaga
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Palma de Mallorca
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Spain
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Sevilla
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Spain
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Valencia
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Taiwan
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Kaohsiung
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Taiwan
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Keelung
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Taiwan
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New Taipei City
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New Taipei
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Taiwan
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Taichung
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Thailand
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Bangkok
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Turkey
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Ankara
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Turkey
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Antalya
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Turkey
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Istanbul
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United Kingdom
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Bristol
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United Kingdom
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Cambridge
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United Kingdom
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Doncaster
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Leicester
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London
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Manchester
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United Kingdom
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Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexion Pharmaceuticals, Inc. (Sponsor)
Address 0 0
Country 0 0
Phone 0 0
1-855-752-2356
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05097989