Pulmonary HypertensionPhase 2/3SAPHNCT01324999

Tadalafil for Sarcoidosis Associated Pulmonary Hypertension

A Phase 2/3 study testing Tadalafil in people with Pulmonary Hypertension, run by University of North Carolina, Chapel Hill at 2 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 study is designed to assess safety and efficacy of tadalafil in patients with Sarcoidosis Associated Pulmonary Hypertension. Hypothesis: Tadalafil is safe and effective in treating pulmonary hypertension in patients with Sarcoidosis Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Pulmonary Hypertension
What is being tested?: Tadalafil
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 12 participants
Where: 2 sitesUnited States
Study dates: Mar 2011to Oct 2013
Phase: Phase 2/3
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

Biopsy proven sarcoidosis
Mean pulmonary artery pressure > 25 mmHg at rest or greater than 30 mmHg with exercise by right heart catheterization within 1 year prior to entry into study
Pulmonary capillary wedge pressure ≤ 15 mmHg
PVR values ≥ 3.0 Woods units
Forced vital capacity (FVC) > 40% predicted
Forced expiratory volume in 1 second (FEV1) > 40% predicted

Show all 12Show fewer

WHO functional class II or III
Stable sarcoidosis treatment regimen for three months prior to entry into study
6 minute walk distance between 150-450 meters
Stable dose of antihypertensive medications
On no other medication to treat PAH (sildenafil, vardenafil, treprostinil, epoprostenol, iloprost, bosentan, ambrisentan) within one month prior to enrollment and during duration of the study
Non-pregnant females

Who cannot join

Exercise limitation related to a non-cardiopulmonary reason (e.g. arthritis)
Severe systemic hypertension > 170/95
Severe systemic hypotension < 90/50
History of priapism
Patients with congestive heart failure (left ventricular dysfunction) LVEF < 45% by echocardiogram
Anticipation by the investigator for escalation in sarcoidosis treatment during the course of the study

Show all 17Show fewer

Pulmonary hypertension related to etiology other than sarcoidosis (i.e. HIV, scleroderma, chronic thromboemboli)
Use within 1 month of an sildenafil or vardenafil
WHO functional class IV status
Patients with severe other organ disease felt by investigators to impact on survival during the course of the study
Subjects with liver function abnormalities (ALT or AST > 3 times the upper limit of normal at screening or at baseline) or chronic liver disease
Advanced kidney failure (GFR < 30 ml/min at screening or at baseline)
History of hypersensitivity reaction or adverse effect related to tadalafil
Pregnant or lactating women
Concomitant use of nitrates (any form) either regularly or intermittently
Concomitant use of potent CYP3A inhibitors (eg, ritonavir, ketoconazole, itraconazole)
Any additional contraindications and precautions specified in the package inserts for Tadalafil (Adcirca) not listed above

Trial locations and contacts

2 sites in 1 country.

United States2 sites

University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Cincinnati
Cincinnati, Ohio, 45267

Other trials in United States

About this study

This study is designed to assess safety and efficacy of tadalafil in patients with Sarcoidosis Associated Pulmonary Hypertension.

Hypothesis: Tadalafil is safe and effective in treating pulmonary hypertension in patients with Sarcoidosis

Conditions: Pulmonary Hypertension
Being tested: Tadalafil
Keywords: Sarcoidosis Associated Pulmonary Hypertension
Medical subject headings: Hypertension, Pulmonary

What participants receive

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

TadalafilDrug
20mg/day for one month then 40mg/day for additional 4 months

Study groups in NCT01324999
Study group	Role	What they receive
Sarcoid Associated Pulm. Hypertension	Experimental	Single-arm open-label proof of concept study of tadalafil in patients with sarcoidosis associated pulmonary hypertension. Receives: Drug: Tadalafil

What is being measured

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

6 Minute Walk Distance
Measured: Baseline, Week 8, Week 16, Week 24

Secondary outcomes (7)

Resting Oxygen Saturation
Percent oxygen saturation of hemoglobin as measured by pulse oximetry in the resting state.
Measured: Baseline, Week 24
Oxygen Desaturation During 6 Minute Walk Test
Change (decrease) in oxygen saturation from start of 6 minute walk to nadir during the 6 minute walk test
Measured: Baseline, Week 24
Maximum Borg Dyspnea Score During 6 Minute Walk Test
The modified Borg scale consists of a vertical scale labelled 0 to10 with corresponding verbal expressions of progressively increasing sensation (shortness of breath) intensity. 0 represents no dyspnea, and 10 is the highest level of perceived dyspnea.
Measured: Baseline, Week 8, Week 16, Week 24
Brain Natriuretic Peptide Level
Measured: Baseline, Week 8, Week 16, Week 24
Short Form-36 Global Score
SF- 36 investigates the standard of quality of life through a general health assessment and not specific to a particular disease, age or treatment group. It is a 36-item questionnaire measuring 8 domains (physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting better health-related functional status. The global score is determined by taking an average of the individual domain scores. The global score range is from 0 (worst) to 100 (best).
Measured: Baseline, Week 8, Week 16, Week 24
St. George's Respiratory Questionnaire (SGRQ) Score
The SGRQ ranges from 0 (no impairment of quality of life) to 100 (highest impairment of quality of life)
Measured: Baseline, Week 8, Week 16, Week 24
Number of Participants With Change in WHO Functional Class (WHO FC)
The WHO functional classification ranges from I (patient's disease does not affect daily activities) to IV (patient's disease causes severe impairment). Higher scores indicate more severe impairment.
Measured: Baseline, Week 24

Timeline

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

Mar 2011Oct 2013

First posted29 Mar 2011 (estimated)
Study startMar 2011
Primary completionJul 2013
Study completionOct 2013

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 Pulmonary HypertensionStudies of the same condition, open and under way.
More trials run by University of North Carolina, Chapel HillThe 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 2/3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 12 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01324999
Sponsor's protocol ID: 09-2326
Acronym: SAPH
Status last verified: Jun 2017
First submitted: 24 Mar 2011
Last updated: 24 Jul 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Ford HJ, Baughman RP, Aris R, Engel P, Donohue JF. Tadalafil therapy for sarcoidosis-associated pulmonary hypertension. Pulm Circ. 2016 Dec;6(4):557-562. doi: 10.1086/688775.
PubMed 28090299

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

Not yet recruitingPhase 2/3
An Open-Label Extension Study to Evaluate Inhaled Treprostinil SMI in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
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Not yet recruitingPhase 2
Study of Treprostinil Administered Via Soft Mist Inhaler (SMI) in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Active, not recruitingPhase 1
A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
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RecruitingObservational study
Natural History Study of Biomarkers in Pulmonary Arterial Hypertension
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Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH

All Pulmonary Hypertension trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Tadalafil for Sarcoidosis Associated Pulmonary Hypertension
ClinicalTrials.gov ID: NCT01324999
Acronym / sponsor ID: SAPH / 09-2326
Conditions: Pulmonary Hypertension
Keywords: Sarcoidosis Associated Pulmonary Hypertension
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Completed
Enrolment: 12 participants (actual)
Lead sponsor: University of North Carolina, Chapel HillUniversity, hospital or other
Collaborators: Eli Lilly and Company, United Therapeutics, University of Cincinnati
Study start: Mar 2011
Primary completion: Jul 2013 (actual)
Study completion: Oct 2013 (actual)
First posted: 29 Mar 2011 (estimated)
Results: Posted 24 Jul 2017

Clinical summary

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

This study is designed to assess safety and efficacy of tadalafil in patients with Sarcoidosis Associated Pulmonary Hypertension.

Hypothesis: Tadalafil is safe and effective in treating pulmonary hypertension in patients with Sarcoidosis

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Sarcoid Associated Pulm. Hypertension	Experimental	Drug: TadalafilSingle-arm open-label proof of concept study of tadalafil in patients with sarcoidosis associated pulmonary hypertension.

Who may qualify

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

Key inclusion criteria (12 listed)

Biopsy proven sarcoidosis
Mean pulmonary artery pressure > 25 mmHg at rest or greater than 30 mmHg with exercise by right heart catheterization within 1 year prior to entry into study
Pulmonary capillary wedge pressure ≤ 15 mmHg
PVR values ≥ 3.0 Woods units
Forced vital capacity (FVC) > 40% predicted

Key exclusion criteria (17 listed)

Exercise limitation related to a non-cardiopulmonary reason (e.g. arthritis)
Severe systemic hypertension > 170/95
Severe systemic hypotension < 90/50
History of priapism
Patients with congestive heart failure (left ventricular dysfunction) LVEF < 45% by echocardiogram

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
Tadalafil20mg/day for one month then 40mg/day for additional 4 months	Drug	Sarcoid Associated Pulm. Hypertension

Primary outcome

6 Minute Walk Distance
Time frame: Baseline, Week 8, Week 16, Week 24

7 secondary outcomes

Resting Oxygen Saturation
Time frame: Baseline, Week 24
Oxygen Desaturation During 6 Minute Walk Test
Time frame: Baseline, Week 24
Maximum Borg Dyspnea Score During 6 Minute Walk Test
Time frame: Baseline, Week 8, Week 16, Week 24
Brain Natriuretic Peptide Level
Time frame: Baseline, Week 8, Week 16, Week 24
Short Form-36 Global Score
Time frame: Baseline, Week 8, Week 16, Week 24
St. George's Respiratory Questionnaire (SGRQ) Score
Time frame: Baseline, Week 8, Week 16, Week 24
Number of Participants With Change in WHO Functional Class (WHO FC)
Time frame: Baseline, Week 24

Results and safety

Results posted to the registry on 24 Jul 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Tadalafil	12	7

Primary outcome: 6 Minute Walk Distance

Time frame: Baseline, Week 8, Week 16, Week 24. Mean (Standard Deviation). Unit: meters

Measure	Tadalafil n = 6
Baseline	419.1 (170.1)
Week 8	418.1 (143.5)
Week 16	436.5 (178.1)
Week 24	377.1 (140.9)

Tadalafil: p = 0.68, t-test, 2 sided

Population analysed: Only 6 of the 7 study completers performed 6 minute walk test through week 24

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: 24 weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Tadalafil	0 of 12 (0%)	5 of 12 (41.7%)	0 of 12

Limitations and caveats (from the sponsor): Since this was a proof of concept study evaluating safety and tolerability of the intervention in this population, it was not powered to achieve statistical significance in changes observed from baseline to week 24 in any of the endpoints.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 28090299Ford HJ, Baughman RP, Aris R, Engel P, Donohue JF. Tadalafil therapy for sarcoidosis-associated pulmonary hypertension. Pulm Circ. 2016 Dec;6(4):557-562. doi: 10.1086/688775.

Related trials for Pulmonary Hypertension

Not yet recruitingPhase 2/3
An Open-Label Extension Study to Evaluate Inhaled Treprostinil SMI in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Not yet recruitingPhase 2
Study of Treprostinil Administered Via Soft Mist Inhaler (SMI) in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Active, not recruitingPhase 1
A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
Pulmonary Arterial Hypertension
RecruitingObservational study
Natural History Study of Biomarkers in Pulmonary Arterial Hypertension
Pulmonary Disease, Pulmonary Hypertension
Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH

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.

Hubert J Ford, M.D. principal investigator

University of North Carolina, Chapel Hill

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Tadalafil in people with Pulmonary Hypertension, run by University of North Carolina, Chapel Hill at 2 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 12 inclusion and 17 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 24 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 24 Jul 2017). 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?

6 Minute Walk Distance (time frame: Baseline, Week 8, Week 16, Week 24). 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 NCT01324999, 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 24 Jul 2017.