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Roflumilast

Phase 3

Asthma | Small molecule | Respiratory |AstraZeneca PLC|Last Updated: Jun 10, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials5
Total Enrollment4,270

FDA Designations

No designations recorded

Clinical trial landscape

Roflumilast · 27 trials · 12 indications

Phase 3 18Phase 2 5Phase 1 4
NCT02165826Evaluation of Tolerability and Pharmacokinetics of Roflumilast, 250μg and 500μg, as add-on to Standard COPD Treatment to Treat Severe COPDChronic Obstructive Pulmonary Disease (COPD)
COMPLETED1,323 Analytics
NCT01509677Trial to Assess the Anti-inflammatory Effects of Roflumilast in Chronic Obstructive Pulmonary DiseaseCOPD
COMPLETED158 Analytics
NCT01313494A Chronic Obstructive Pulmonary Disease (COPD) Trial Investigating Roflumilast on Safety and Effectiveness in China, Hong Kong and Singapore:COPD
COMPLETED626 Analytics
NCT00424268Effect of Roflumilast in Chronic Obstructive Pulmonary Disease (COPD) Patients Treated With Tiotropium: The HELIOS Study (BY217/M2-128)Chronic Obstructive Pulmonary Disease
COMPLETED743 Analytics
NCT00313209Effect of Roflumilast on Lung Function in Chronic Obstructive Pulmonary Disease (COPD) Patients Treated With Salmeterol: The EOS Study (BY217/M2-127)Chronic Obstructive Pulmonary Disease (COPD)
COMPLETED933 Analytics
NCT00297115Effect of Roflumilast on Exacerbation Rate in Patients With Chronic Obstructive Pulmonary Disease (COPD): The HERMES Study (BY217/M2-125)Chronic Obstructive Pulmonary Disease (COPD)
COMPLETED1,568 Analytics
NCT00297102Effect of Roflumilast on Exacerbation Rate in Patients With Chronic Obstructive Pulmonary Disease (COPD): The AURA Study (BY217/M2-124)Chronic Obstructive Pulmonary Disease (COPD)
COMPLETED1,523 Analytics
NCT00242320Efficacy and Safety of Oral Roflumilast Taken Once Daily in Patients Older Than 40 Years With Chronic Obstructive Pulmonary Disease (BY217/M2-119)COPD
COMPLETED551 Analytics
NCT00246935Long-term Study of Safety and Efficacy of Roflumilast in Japanese Patients Older Than 40 Years With Chronic Obstructive Pulmonary Disease (APTA-2217-08)Chronic Obstructive Pulmonary Disease (COPD)
COMPLETED150 Analytics
NCT00246922Long-term Study of Safety and Efficacy of Roflumilast in Japanese Patients With Bronchial Asthma (20 to 71 y) (APTA-2217-07).Bronchial Asthma
COMPLETED150 Analytics
PHASE3COMPLETED
Evaluation of Tolerability and Pharmacokinetics of Roflumilast, 250μg and 500μg, as add-on to Standard COPD Treatment to Treat Severe COPD
Chronic Obstructive Pulmonary Disease (COPD)Unlock trial analytics
PHASE3COMPLETED
Trial to Assess the Anti-inflammatory Effects of Roflumilast in Chronic Obstructive Pulmonary Disease
COPDUnlock trial analytics
PHASE3COMPLETED
A Chronic Obstructive Pulmonary Disease (COPD) Trial Investigating Roflumilast on Safety and Effectiveness in China, Hong Kong and Singapore:
COPDUnlock trial analytics
PHASE3COMPLETED
Effect of Roflumilast in Chronic Obstructive Pulmonary Disease (COPD) Patients Treated With Tiotropium: The HELIOS Study (BY217/M2-128)
Chronic Obstructive Pulmonary DiseaseUnlock trial analytics
PHASE3COMPLETED
Effect of Roflumilast on Lung Function in Chronic Obstructive Pulmonary Disease (COPD) Patients Treated With Salmeterol: The EOS Study (BY217/M2-127)
Chronic Obstructive Pulmonary Disease (COPD)Unlock trial analytics
PHASE3COMPLETED
Effect of Roflumilast on Exacerbation Rate in Patients With Chronic Obstructive Pulmonary Disease (COPD): The HERMES Study (BY217/M2-125)
Chronic Obstructive Pulmonary Disease (COPD)Unlock trial analytics
PHASE3COMPLETED
Effect of Roflumilast on Exacerbation Rate in Patients With Chronic Obstructive Pulmonary Disease (COPD): The AURA Study (BY217/M2-124)
Chronic Obstructive Pulmonary Disease (COPD)Unlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Oral Roflumilast Taken Once Daily in Patients Older Than 40 Years With Chronic Obstructive Pulmonary Disease (BY217/M2-119)
COPDUnlock trial analytics
PHASE3COMPLETED
Long-term Study of Safety and Efficacy of Roflumilast in Japanese Patients Older Than 40 Years With Chronic Obstructive Pulmonary Disease (APTA-2217-08)
Chronic Obstructive Pulmonary Disease (COPD)Unlock trial analytics
PHASE3COMPLETED
Long-term Study of Safety and Efficacy of Roflumilast in Japanese Patients With Bronchial Asthma (20 to 71 y) (APTA-2217-07).
Bronchial AsthmaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants Prematurely Discontinuing Study Treatment Due to Any Reason
Baseline to Week 12 (Main Period)

The primary endpoint is the percentage of participants prematurely discontinuing study treatment for any reason during the Main Period from Visit 1 (V1) to Last Visit (Vend). Discontinuation is defined as permanently stopping randomized treatment; participants who resume randomized treatment after an interval will not be counted as having discontinued. The analysis used discontinuations occurring during the Main Period, irrespective of whether a participant subsequently entered into the Down-Titration Period.

Number of CD8+ Inflammatory Cells in Bronchial Biopsy Tissue.
16 weeks
Change in Number of CD8+ Inflammatory Cells in Bronchial Biopsy Tissue
Baseline to 16 weeks
Change From Baseline in Pre-bronchodilator Forced Expiratory Volume in First Second (FEV1)
Baseline to Week 24

FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Pulmonary function testing was performed using centralized spirometry prior to taking study medication. Change from baseline over 24 weeks of treatment was calculated from a repeated measures analysis of covariance (ANCOVA) model with treatment, baseline value of pre-bronchodilator FEV1, time and a treatment-by-time interaction as independent variables.

Pre-bronchodilator Forced Expiratory Volume in First Second (FEV1)
Change from baseline over 24 weeks of treatment

Mean change from baseline during the treatment period in pre-bronchodilator FEV1 \[L\]

COPD Exacerbation Rate (Moderate or Severe)
52 weeks treatment period

Mean rate of COPD exacerbations requiring oral or parenteral glucocorticosteroids (=moderate COPD exacerbations), or requiring hospitalization, or leading to death (=severe COPD exacerbations), per patient per year. A COPD exacerbation is an event in the natural course of the disease characterized by a change in the patient's baseline dyspnea, cough and/or sputum beyond day-to-day variability sufficient to warrant a change in management \[American Thoracic Society (ATS) / European Respiratory Society (ERS) 2005\].

mean change from randomization to endpoint in lung function (post bronchodilator)
12 weeks
Long-term safety after 28 weeks treatment of Roflumilast (total 52 weeks, 24 weeks of study APTA-2217-06 followed by 28 weeks) .
Long-term safety after 28 weeks treatment of Roflumilast at (total 52 weeks, 24 weeks of study APTA-2217-05 followed by 28 weeks) .
Lung function parameters indicative of hyperinflation in patients with COPD
mean change from randomization to endpoint in forced expiratory volume in one second.
change in FEV1 from baseline during the treatment period; number of COPD exacerbations.
change in lung function.
change in lung function from baseline to final visit.
pulmonary function.
change in forced expiratory volume in 1 second from baseline to final visit.
The frequency of patients experiencing at least one moderate or severe exacerbation during the treatment period.
Change in FEV1 from baseline during the treatment period.
Change From Baseline in Pre-Dose (Trough) Pre-Bronchodilator Forced Expiratory Volume in 1 Second (FEV1)
Baseline (Days 1 and 56) and after 4 weeks of treatment (Days 28 and 84)

FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEV1 will be measured using spirometry in accordance with the American Thoracic Society / European Respiratory Society (ATS/ERS) consensus guidelines. An ANCOVA model with treatment sequence, treatment period, and study treatment as fixed factors with Baseline FEV1 measurement as the covariate was used for analysis.

Mean change in HbA1c [percent] from baseline to the last study visit (Vlast)
Baseline to last visit
Change of percent eosinophils in sputum 24 hours after allergen challenge
From baseline until end of treatment up to 9 weeks

The primary outcome was the effect of roflumilast on allergen-induced airway eosinophilia. Two 15 days treatment periods (14 days with treatment and day 15 without treatment) separated by a washout period (approximately 3 to 5 weeks).

change in lung function parameters (post-bronchodilator).
change in lung function parameters.
Change From Baseline in the Verbal Recall Memory (VRM) Total Number of Correct Responses for Delayed Recall at 1 Hour After Scopolamine Administration
Baseline and 1 hour after scopolamine administration on Day 1 of each treatment period. Baseline is defined as the assessment 1 hour before roflumilast/donepezil administration (3 hours before scopolamine administration).

VRM measures the ability to encode and subsequently retrieve verbal information. This task begins with the first presentation phase in which 18 words are shown in turn on the screen. The participant is then asked to recall as many words as possible during the first immediate recall phase. The same 18 words are then shown in a second presentation phase which is followed by a second immediate recall phase. After a delay of approximately 20-30 minutes, a delayed recall stage is completed. The possible range of correct responses is 0 (worst) to 18 (best). Higher number of correct responses in the test indicates a better outcome. A negative change from baseline indicates a worsening of the score.

Change From Baseline in the Verbal Recall Memory (VRM) Total Number of Correct Responses for Immediate Recall at 1 Hour After Scopolamine Administration
Baseline and 1 hour after scopolamine administration on Day 1 of each treatment period.

VRM measures the ability to encode and subsequently retrieve verbal information. This task begins with the first presentation phase in which 18 words are shown in turn on the screen. The participant is then asked to recall as many words as possible during the first immediate recall phase. The possible range of correct responses is 0 (worst) to 18 (best). Higher number of correct responses in the test indicates a better outcome. A negative change from baseline indicates a worsening of the score.

Change From Baseline in Postprandial Area Under the Curve From Time 0 to 8 Hours (AUC[0-8]) for Active Glucagon-like Peptide-1
Baseline and Day 11; samples were taken at -15 min and -5 min (pre-meal), and 15 min, 30 min, and 1, 2, 3, 4, 6, and 8 hours (post-meal).

The concentration of glucagon-like peptide-1 (GLP-1) in blood before and up to 8 hours after eating (postprandial) was plotted and the area under the curve calculated using the linear trapezoidal rule at Baseline and on Day 11. Least squares means of the change from Baseline to Day 11 were obtained using an analysis of covariance (ANCOVA) model with treatment as fixed effect, and Baseline postprandial AUC (0-8) of active GLP-1 as a continuous covariate.

To determine the pharmacokinetic profiles after single and repeated dose of roflumilast 500 µg administered to healthy Chinese subjects.
Period I: single dose, PK-Investigation over 8 days, Period II: repeated dose for 14 days, PK-Investigation on days 14 and 15

PK samples at pre-dose, 0.25h, 0.5h, 0.75h, 1h, 1.5h, 2h, 2.5h, 3h, 4h, 5h, 6h, 8h, 10h, 12h, 16h and 24h, 36h, 48h, 72h, 96h, 120h, 144h and 168h post dose after single dose and at the time points pre-dose, 0.25h, 0.5h, 0.75h, 1h, 1.5h, 2h, 2.5h, 3h, 4h, 5h, 6h, 8h, 10h, 12h, 16h and 24h after administration of a repeated dose

Potential cardiovascular interaction (exploratory)

Secondary Endpoints

Percentage of Participants With Adverse Events of Interest
Baseline to Week 12 (Main Period)
Change From Baseline (V0DT) in Pre-bronchodilator Forced Expiratory Volume in First Second (FEV1) to Final Visit of the Down-Titration Period
Baseline (V0DT) [assessment at end of main period] and Final Visit of Down-Titration Period (Up to Day 56)
Percentage of Participants Prematurely Discontinuing Study Treatment Due to Any Reason During Down-Titration Period
Baseline DT (Day 1 of Down-Titration Period) to Week 8 (Down-Titration Period)
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Roflumilast 500 μg once dailyEXPERIMENTALRoflumilast 500 μg tablets, orally, once daily for 12 weeks. Any participants not tolerating study treatment will be prematurely discontinued and will receive roflumilast 250 μg, tablets, orally, once daily for 8 weeks.
Roflumilast 500 μg every other dayEXPERIMENTALRoflumilast 500 μg, tablets, orally, every other day, and roflumilast placebo-matching tablets, orally, every other day on non-treatment days, for 4 weeks, followed by roflumilast 500 μg, tablets, orally, once daily, for 8 weeks. Any participants not tolerating study treatment will be prematurely discontinued and will receive roflumilast 250 μg, tablets, orally, once daily for 8 weeks.
Roflumilast 250 μg once dailyEXPERIMENTALRoflumilast 250 μg, tablets, orally, once daily for 4 weeks, followed by roflumilast 500 μg, tablets, orally, once daily, for 8 weeks. Any participants not tolerating study treatment will be prematurely discontinued and will receive roflumilast 250 μg, tablets, orally, once daily for 8 weeks.
RoflumilastACTIVE_COMPARATOR500 μg tablet, once daily, oral administration in the morning after breakfast
PlaceboPLACEBO_COMPARATORtablet, once daily, oral administration in the morning after breakfast
1ACTIVE_COMPARATORRoflumilast 500 µg
2PLACEBO_COMPARATORPlacebo
Roflumilast plus montelukast, then placebo plus montelukastEXPERIMENTALParticipants in sequence 1 received roflumilast 500 μg plus montelukast 10 mg orally once daily for 4 weeks followed by a 4-week washout period and then received placebo plus montelukast 10 mg orally once daily for 4 weeks.
Placebo plus montelukast, then roflumilast plus montelukastEXPERIMENTALParticipants in sequence 2 received placebo plus montelukast 10 mg orally once daily for 4 weeks followed by a 4-week washout period and then received roflumilast 500 μg plus montelukast 10 mg orally once daily for 4 weeks.
Sequence 1 (ABDC)EXPERIMENTALRoflumilast placebo-matching tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 1, followed by roflumilast placebo-matching tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 2, followed by roflumilast Dose A tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 3, followed by roflumilast Dose A tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 4. Each treatment period is separated by a 14-day washout period.
Sequence 2 (BCAD)EXPERIMENTALRoflumilast placebo-matching tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 1, followed by roflumilast Dose A tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 2, followed by roflumilast placebo-matching tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 3, followed by roflumilast Dose A tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 4. Each treatment period is separated by a 14-day washout period.
Sequence 3 (CDBA)EXPERIMENTALRoflumilast Dose A tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 1, followed by roflumilast Dose A tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 2, followed by roflumilast placebo-matching tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 3, followed by roflumilast placebo-matching tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 4. Each treatment period is separated by a 14-day washout period.
Sequence 4 (DACB)EXPERIMENTALRoflumilast Dose A tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 1, followed by roflumilast placebo-matching tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 2, followed by roflumilast Dose A tablets, orally, donepezil placebo-matching overencapsulated tablets, orally, and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 3, followed by roflumilast placebo-matching tablets, orally, donepezil 10 mg, overencapsulated tablets, orally and scopolamine 0.5 mg subcutaneous injection, once, Day 1, Period 4. Each treatment period is separated by a 14-day washout period.
Roflumilast + alogliptinEXPERIMENTALRoflumilast 500 μg, tablets, orally and alogliptin 25 mg, tablets, orally, once a day for 11 days.
Alogliptin aloneEXPERIMENTALPlacebo to roflumilast, tablets, orally and alogliptin, 25 mg, tablets, orally, once a day for 11 days.
Roflumilast aloneEXPERIMENTALRoflumilast 500 μg, tablets, orally and placebo to alogliptin, tablets, orally, once a day, for 11 days.
ExenatideACTIVE_COMPARATORExenatide 5 μg subcutaneous injection twice a day for 11 days.
Treatment AACTIVE_COMPARATOR -
Treatment BACTIVE_COMPARATOR -

Interventions

NameTypeDescription
RoflumilastDRUGRoflumilast tablets
Roflumilast PlaceboDRUGRoflumilast placebo-matching tablets
Standard of Care COPD TreatmentDRUGThe participant is on standard of care COPD maintenance treatment including LABAs, long-acting anticholinergics, or any combination thereof taken on a constant daily dose within 12 weeks prior to Screening (Visit V0) Examples of LABA containing products are: Formoterol, Salmeterol, Indacaterol, Formoterol/Budesonide, Salmeterol/Fluticasone, Treatment including lon-acting anticholinergics: Tiotropium, Aclidinium.
PlaceboDRUGtablet, once daily, oral administration in the morning after breakfast
SalbutamolDRUGSalbutamol (given by MDI and spacer) used as rescue medication on an ass needed basis throughout the trial, and was used for post-bronchodilator spirometry tests at all study visits.
MontelukastDRUGMontelukast was supplied in tablets.
DonepezilDRUGDonepezil overencapsulated tablets
Donepezil placeboDRUGDonepezil placebo-matching overencapsulated tablets
ScopolamineDRUGScopolamine subcutaneous injection
AlogliptinDRUGAlogliptin tablets
ExenatideDRUGExenatide solution
Placebo to roflumilastDRUGPlacebo-matching roflumilast tablets
Placebo to alogliptinDRUGPlacebo-matching alogliptin tablets
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Eligibility Criteria

Age Range40 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: 1. In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements. 2. The participant or, when applicable, the participant's legally acceptable representative signs and dates a written, informed consent form and any requi...

Countries:SlovakiaDenmarkGermanyPolandSwedenUnited KingdomChinaHong KongSingaporeAustriaFranceHungaryItalySpainBelgiumCanadaNetherlandsSouth AfricaUnited StatesIndiaAustraliaBrazilNew ZealandRomaniaRussiaMalaysiaPhilippinesSouth KoreaTaiwanJapanUkraineArgentinaColombiaMexicoPeruCroatiaCzechiaFinlandGreeceIrelandNorwayPakistanPortugalThailandSwitzerland
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Recent Changes (Last 90 Days)

MEDIUMJul 11, 2026NCT00242307TRIAL_REMOVED: changed
MEDIUMJul 11, 2026NCT00246922TRIAL_REMOVED: changed
MEDIUMJul 11, 2026NCT00242307TRIAL_REMOVED: changed
MEDIUMJul 11, 2026NCT00246922TRIAL_REMOVED: changed
MEDIUMJul 11, 2026NCT00242307TRIAL_REMOVED: changed
MEDIUMJul 11, 2026NCT00246922TRIAL_REMOVED: changed

Frequently asked questions about Roflumilast

What is Roflumilast used for?

Roflumilast is an investigational small molecule being studied for respiratory conditions including bronchial asthma and chronic obstructive pulmonary disease (COPD). It is also being evaluated in type 2 diabetes mellitus. The drug is taken orally once daily and is currently in Phase 2 clinical development.

Who makes Roflumilast?

Roflumilast is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker AZN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in respiratory and metabolic indications.

What phase is Roflumilast in?

Roflumilast is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The development program includes completed Phase 2 and Phase 3 trials, with the most advanced ongoing studies being Phase 2.

What clinical trials is Roflumilast in?

Roflumilast has been studied in several clinical trials, including NCT00242307, a Phase 2 trial in Japanese patients with bronchial asthma, and NCT00242320, a Phase 3 trial in patients with COPD. Other trials include NCT01313494 and NCT01509677, both Phase 3 studies in COPD.

Is Roflumilast the same as Daxas?

Roflumilast is also known by the brand name Daxas. It is a phosphodiesterase-4 inhibitor being developed for respiratory conditions. The drug is being studied for its anti-inflammatory effects in chronic obstructive pulmonary disease and asthma.