Interstitial PneumonitisPhase 2/3IMPPACTNCT00504348

Investigation in Myositis-associated Pneumonitis of Prednisolone And Concomitant Tacrolimus

A Phase 2/3 study testing Tacrolimus in people with Interstitial Pneumonitis and Polymyositis, run by Tokyo Medical and Dental University at 11 sites in Japan.

Completed

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

Official record

Search all trials

Trial summary

The purpose of the study is to evaluate the efficacy and safety of the combination treatment of tacrolimus and corticosteroid in polymyositis/dermatomyositis patients with interstitial pneumonitis with comparison against corticosteroid-treated historical controls. Read more about this study

Who is it for?: Ages 16 to 74 years
Conditions the study is for: Interstitial Pneumonitis, Polymyositis, Dermatomyositis
What is being tested?: Tacrolimus
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 25 participants
Where: 11 sitesJapan
Study dates: Jul 2007to Jan 2011
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 16 to 74 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Experimental treatment group
Diagnosis of definite or probable polymyositis or dermatomyositis by criteria of Bohan et al, or of clinically-amyopathic dermatomyositis by the definition proposed by Sontheimer et al
High-resolution CT findings consistent with interstitial pneumonitis, confirmed by a radiologist. If consolidation is the only abnormal findings, the patient must have pathologically documented evidence of interstitial pneumonitis of other histological type than cryptogenic organizing pneumonia/bronchiolitis obliterans organizing pneumonia (the patient could have more than one histological type including cryptogenic organizing pneumonia/bronchiolitis obliterans organizing pneumonia)
Meet two or more of the following criteria (must include 1) 1. Serum KL-6 above the upper normal limit 2. Presence of dyspnea on exertion (grade 2 on the Magnitude of Task component of the Mahler Modified Dyspnea Index 3. PaO2 of less than 80 mmHg while breathing ambient air at rest, not accompanied by abnormal increase of PaCO2 4. Vital capacity < 80% predicted, or diffusing capacity for carbon monoxide < 65% predicted 5. Meet at least one of the following condition over the 12-week period (84 days) prior to the initiation of the study drug
Decrease in either % forced vital capacity or % diffusing capacity for carbon monoxide of 10% or more
Worsening of interstitial pneumonitis findings by chest CT, confirmed by a radiologist

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16 to 74 years of age
Historical control group
Diagnosis of definite or probable polymyositis or dermatomyositis by criteria of Bohan et al, or of clinically-amyopathic dermatomyositis by the definition proposed by Sontheimer et al
High-resolution CT findings consistent with interstitial pneumonitis, confirmed by a radiologist. If consolidation is the only abnormal findings, the patient must have pathologically documented evidence of interstitial pneumonitis of other histological type than cryptogenic organizing pneumonia/bronchiolitis obliterans organizing pneumonia (the patient could have more than one histological type including cryptogenic organizing pneumonia/bronchiolitis obliterans organizing pneumonia)
Meet two or more of the following criteria (must include 1) 1. Serum KL-6 above the upper normal limit 2. Presence of dyspnea on exertion (grade 2 on the Magnitude of Task component of the Mahler Modified Dyspnea Index 3. PaO2 of less than 80 mmHg while breathing ambient air at rest, not accompanied by abnormal increase of PaCO2 4. Vital capacity < 80% predicted, or diffusing capacity for carbon monoxide < 65% predicted 5. Meet at least one of the following condition over the 12-week period (84 days) prior to the initiation of the study drug
Decrease in either % forced vital capacity or % diffusing capacity for carbon monoxide of 10% or more
Worsening of interstitial pneumonitis findings by chest CT, confirmed by a radiologist
Use of corticosteroids at doses equivalent to between 0.6 to 1.0mg/kg/day of prednisolone for 14 days or longer to treat interstitial pneumonitis on or after the day when the inclusion criteria (3) was met (up to two courses of pulse IV corticosteroid therapy within the first 28 days are allowed)
16 to 74 years of age

Who cannot join

Experimental treatment group
Use of corticosteroids at doses equivalent to or higher than prednisolone 0.6mg/kg/day within 4 weeks (28 days) prior to the initiation of the study drug
Use of immunosuppressive agents other than corticosteroids within 12 weeks (84 days) prior to the initiation of the study drug
Could not exclude the following conditions on clinical ground: drug-induced pneumonitis, occupational lung disease, hypersensitivity pneumonitis, radiation-induced lung injury
Presence of end-stage interstitial pneumonitis as identified on the basis of a vital capacity < 45% predicted, diffusing capacity for carbon monoxide < 30% predicted, or lung CT with predominantly honeycombing appearance
Presence of pancreatitis

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Presence of diabetes mellitus with the exception of glucocorticoid-induced one that is well-controlled (HbA1c < 6.5%)
Serum creatinine of 1.5 mg/dL or above
Presence of liver dysfunction (AST(GOT) or ALT (GPT) greater than 2.5 times the upper limit of normal) with the exception of the one that is considered to be due to myositis and is accompanied by the elevation of muscle enzymes above the upper limit of normal
Serum potassium above the upper limit of normal
Presence of ischemic heart disease, arrhythmia requiring treatment, congestive heart failure, or pulmonary hypertension requiring treatment
Presence or history of malignancy with the exception of those without relapse off treatment for 5 years or longer
Presence of serious active infection
Presence of active hepatitis B, hepatitis C, or HIV infection
History of severe drug hypersensitivity reaction
Patients who are pregnant or breast-feeding, or patients who intend to or whose spouses intend to conceive during the course of the study, including the follow-up period
Participation in another clinical trial or post-marketing clinical study within 26 weeks (182 days) prior to screening
Other medical condition which, in the investigator's judgment, may be associated with increased risk to the subject or may interfere with study assessments or outcomes
Historical control group
Use of immunosuppressive agents other than corticosteroids within 12 weeks (84 days) prior to or 2 weeks (14 days) after the corticosteroid treatment as defined by the inclusion criteria (4) is initiated
Could not exclude the following conditions on clinical ground: drug-induced pneumonitis, occupational lung disease, hypersensitivity pneumonitis, radiation-induced lung injury
Presence of end-stage interstitial pneumonitis as identified on the basis of a vital capacity < 45% predicted, diffusing capacity for carbon monoxide < 30% predicted, or lung CT with predominantly honeycombing appearance
Presence of pancreatitis
Presence of diabetes mellitus with the exception of glucocorticoid-induced one that is well-controlled (HbA1c < 6.5%)
Serum creatinine of 1.5 mg/dL or above
Presence of liver dysfunction (AST(GOT) or ALT (GPT) greater than 2.5 times the upper limit of normal) with the exception of the one that is considered to be due to myositis and is accompanied by the elevation of muscle enzymes above the upper limit of normal
Serum potassium above the upper limit of normal
Presence of ischemic heart disease, arrhythmia requiring treatment, congestive heart failure, or pulmonary hypertension requiring treatment
Presence or history of malignancy with the exception of those without relapse off treatment for 5 years or longer
Presence of serious active infection including active hepatitis B, hepatitis C, or HIV infection
Other medical condition which, in the investigator's judgment, may be associated with increased risk to the subject or may interfere with study assessments or outcomes

Trial locations and contacts

11 sites in 1 country.

City, postcode or hospital

11 sites in 1 country

Japan11 sites

Juntendo University Hospital
Bunkyo-ku, Tokyo, 113-8431
Tokyo Medical and Dental University Hospital
Bunkyo-ku, Tokyo, 113-8519
The University of Tokyo Hospital
Bunkyo-ku, Tokyo, 113-8655
Chiba University Hospital
Chiba, 260-8677
Osaka Minami Medical Center
Kawachi-Nagano, Osaka, 586-8521
Nagasaki University Hospital of Medicine and Dentistry
Nagasaki, 852-8501
Hokkaido University Hospital
Sapporo, Hokkaido, 060-8648
Keio University Hospital
Shinjuku-ku, Tokyo, 160-8582
International Medical Center of Japan
Shinjuku-ku, Tokyo, 162-8655
Tokushima University Hospital
Tokushima, 770-8503
Tsukuba University Hospital
Tsukuba, Ibaraki, 305-8576

Other trials in Japan

About this study

Official title: An Open-label Clinical Trial of the Combination Treatment of Tacrolimus and Corticosteroid in Polymyositis/Dermatomyositis Patients With Interstitial Pneumonitis, With Comparison Against Corticosteroid-treated Historical Controls

Read the detailed description

Interstitial pneumonia (IP) is a common complication of and has a significant impact on the prognosis of patients with polymyositis (PM) and dermatomyositis (DM). Reported prevalence of IP in PM/DM patients varies between 23 to 65% depending on criteria applied as well as on clinical settings of studied cohorts, and an earlier overview and a later study reported its high short-term mortality.

However, treatment for this grave complication has not yet been either established or even been prospectively investigated. Glucocorticoids, while long been considered as the first-line drugs, is effective in less than 50% of patients. Furthermore, the mortality of these glucocorticoids-resistant patients does not improve even if immunosuppressive drugs are later added.

Recently, we and others reported retrospective data which suggest that either an early addition of immunosuppressive drugs to glucocorticoids or the combined use of glucocorticoids and immunosuppressive drugs from the initial treatment may improve the survival of PM/DM patients. To save lives of PM/DM-IP patients, desperate treating physicians have started using this approach, strongly urging the conduct of prospective studies to investigate the superiority of this approach over glucocorticoids alone. At the same time, it was considered not ethically appropriate to conduct a prospective study with a concurrent controlled group receiving glucocorticoids alone given the presence of the PM/DM-IP subtype with rapidly progressive course and high short-term mortality if treated with glucocorticoids alone and the absence of useful demographic or bio-markers which could distinguish patients with this subtype early. Among immunosuppressive drugs used in the treatment of PM/DM-IP, tacrolimus has recently been suggested to be effective even for those patients who are resistant to cyclosporine or cyclosphosphamide.

To investigate whether the combined initial treatment of glucocorticoids and tacrolimus is superior to glucocorticoids alone in PM/DM-IP patients, we conducted a multicenter clinical trial to evaluate the efficacy and safety of a combination treatment of glucocorticoids and tacrolimus for 1 year in patients with newly developed active PM/DM-IP or its relapse by comparing against clinical outcome of historical control patients who were treated with glucocorticoid alone as an initial treatment.

Conditions: Interstitial Pneumonitis, Polymyositis, Dermatomyositis
Being tested: Tacrolimus
Keywords: Interstitial pneumonitis, Polymyositis, Dermatomyositis, Tacrolimus, Corticosteroids
Medical subject headings: Lung Diseases, Interstitial, Polymyositis, Dermatomyositis

What participants receive

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

TacrolimusDrug
Also called Prograf
Start at the standard starting dose of 0.075mg/kg/day divided into two doses, then adjust doses based on clinical response and tolerability, but maintain whole blood trough levels between 5 to 10 ng/mL and total daily doses equal to or below 0.3mg/kg.

Study groups in NCT00504348
Study group	Role	What they receive
Prospective investigation group	Experimental	Tacrolimus treatment is to be initiated at the starting dose of 0.075mg/kg/day, adjusted to maintain its whole blood trough levels between 5 and 10 ng/mL for 52 weeks. All patients are to receive glucocorticoids with the starting doses equivalent to between 0.6 and 1.0 mg/kg/day of prednisolone which are to be continued for the first 28 days after which be subsequently tapered according to a predefined guideline. Up to two courses of pulse intravenous glucocorticoid therapy are allowed during that period. Receives: Drug: Tacrolimus

What is being measured

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

Overall Survival
Overall survival (OS) was calculated from the day on which the protocol treatment was started until death due to any cause. Participants still alive were censored at the date they were last known to be alive.
Measured: 52 weeks

Secondary outcomes (1)

Progression-free Survival
Patients were considered to have reached the progression if they died, or if they met all the following criteria; (1) ≥10% decline from baseline FVC or ≥15mmHg increase in baseline resting P(A-a)O2, (2) a worsening of interstitial pneumonitis findings by chest CT compared to the most recent study, confirmed by a radiologist, and (3) exclusion of pneumocystis pneumonia, cytomegalovirus pneumonia, and other pulmonary infection on clinical ground.
Measured: 52 weeks

Timeline

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

Jul 2007Jan 2011

First posted20 Jul 2007 (estimated)
Study startJul 2007
Primary completionJan 2011
Study completionJan 2011

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 Interstitial PneumonitisStudies 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 2/3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 25 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00504348
Sponsor's protocol ID: IICT-FK506-01
Acronym: IMPPACT
Status last verified: Mar 2020
First submitted: 19 Jul 2007
Last updated: 24 Mar 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Takada K, Katada Y, Ito S, Hayashi T, Kishi J, Itoh K, Yamashita H, Hirakata M, Kawahata K, Kawakami A, Watanabe N, Atsumi T, Takasaki Y, Miyasaka N. Impact of adding tacrolimus to initial treatment of interstitial pneumonitis in polymyositis/dermatomyositis: a single-arm clinical trial. Rheumatology (Oxford). 2020 May 1;59(5):1084-1093. doi: 10.1093/rheumatology/kez394.
PubMed 31539061

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?

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Study at a glance

From the registry record, last updated by the sponsor on 24 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label Clinical Trial of the Combination Treatment of Tacrolimus and Corticosteroid in Polymyositis/Dermatomyositis Patients With Interstitial Pneumonitis, With Comparison Against Corticosteroid-treated Historical Controls
ClinicalTrials.gov ID: NCT00504348
Acronym / sponsor ID: IMPPACT / IICT-FK506-01
Conditions: Interstitial Pneumonitis, Polymyositis, Dermatomyositis
Keywords: Interstitial pneumonitis, Polymyositis, Dermatomyositis, Tacrolimus, Corticosteroids
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Completed
Enrolment: 25 participants (actual)
Lead sponsor: Tokyo Medical and Dental UniversityUniversity, hospital or other
Collaborators: Japan Medical Association, Astellas Pharma Inc
Study start: Jul 2007
Primary completion: Jan 2011 (actual)
Study completion: Jan 2011 (actual)
First posted: 20 Jul 2007 (estimated)
Results: Posted 24 Mar 2020

Clinical summary

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

Detailed description

Study design

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

Arm	Type	Receives
Prospective investigation group	Experimental	Drug: TacrolimusTacrolimus treatment is to be initiated at the starting dose of 0.075mg/kg/day, adjusted to maintain its whole blood trough levels between 5 and 10 ng/mL for 52 weeks. All patients are to receive glucocorticoids with the starting doses equivalent to between 0.6 and 1.0 mg/kg/day of prednisolone which are to be continued for the first 28 days after which be subsequently tapered according to a predefined guideline. Up to two courses of pulse intravenous glucocorticoid therapy are allowed during that period.

Who may qualify

Ages: 16 to 74 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (15 listed)

Experimental treatment group
Diagnosis of definite or probable polymyositis or dermatomyositis by criteria of Bohan et al, or of clinically-amyopathic dermatomyositis by the definition proposed by Sontheimer et al
High-resolution CT findings consistent with interstitial pneumonitis, confirmed by a radiologist. If consolidation is the only abnormal findings, the patient must have pathologically documented evidence of interstitial pneumonitis of other histological type than cryptogenic organizing pneumonia/bronchiolitis obliterans organizing pneumonia (the patient could have more than one histological type including cryptogenic organizing pneumonia/bronchiolitis obliterans organizing pneumonia)
Meet two or more of the following criteria (must include 1) 1. Serum KL-6 above the upper normal limit 2. Presence of dyspnea on exertion (grade 2 on the Magnitude of Task component of the Mahler Modified Dyspnea Index 3. PaO2 of less than 80 mmHg while breathing ambient air at rest, not accompanied by abnormal increase of PaCO2 4. Vital capacity < 80% predicted, or diffusing capacity for carbon monoxide < 65% predicted 5. Meet at least one of the following condition over the 12-week period (84 days) prior to the initiation of the study drug
16 to 74 years of age

Key exclusion criteria (31 listed)

Experimental treatment group
Use of corticosteroids at doses equivalent to or higher than prednisolone 0.6mg/kg/day within 4 weeks (28 days) prior to the initiation of the study drug
Use of immunosuppressive agents other than corticosteroids within 12 weeks (84 days) prior to the initiation of the study drug
Could not exclude the following conditions on clinical ground: drug-induced pneumonitis, occupational lung disease, hypersensitivity pneumonitis, radiation-induced lung injury
Presence of end-stage interstitial pneumonitis as identified on the basis of a vital capacity < 45% predicted, diffusing capacity for carbon monoxide < 30% predicted, or lung CT with predominantly honeycombing appearance

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
TacrolimusAlso: PrografStart at the standard starting dose of 0.075mg/kg/day divided into two doses, then adjust doses based on clinical response and tolerability, but maintain whole blood trough levels between 5 to 10 ng/mL and total daily doses equal to or below 0.3mg/kg.	Drug	Prospective investigation group

Primary outcome

Overall Survival
Time frame: 52 weeks
Overall survival (OS) was calculated from the day on which the protocol treatment was started until death due to any cause. Participants still alive were censored at the date they were last known to be alive.

1 secondary outcome

Progression-free Survival
Time frame: 52 weeks

Results and safety

Results posted to the registry on 24 Mar 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Prospective Investigation Group	25	17

Primary outcome: Overall Survival

Time frame: 52 weeks. Number (95% Confidence Interval). Unit: percentage of participants

Measure	Prospective Investigation Group n = 25
Value	88.0 (67.3 to 96.0)

1 secondary outcome is reported in the full results.

Adverse events

Time frame: All adverse events observed between the first dose and 28 days after the last dose of tacrolimus.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Prospective Investigation Group	11 of 25 (44%)	25 of 25 (100%)	4 of 25

Limitations and caveats (from the sponsor): Conclusion is not based on meaningful statistical comparison, will not apply to asymptomatic or subclinical IP, or to the exacerbation of IP, or will not separately provide the efficacy of the treatment in the anti-MDA-5 positive subgroup.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 31539061Takada K, Katada Y, Ito S, Hayashi T, Kishi J, Itoh K, Yamashita H, Hirakata M, Kawahata K, Kawakami A, Watanabe N, Atsumi T, Takasaki Y, Miyasaka N. Impact of adding tacrolimus to initial treatment of interstitial pneumonitis in polymyositis/dermatomyositis: a single-arm clinical trial. Rheumatology (Oxford). 2020 May 1;59(5):1084-1093. doi: 10.1093/rheumatology/kez394.

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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.

Nobuyuki Miyasaka, MD, PhD principal investigator

Tokyo Medical and Dental University

11 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 Tacrolimus in people with Interstitial Pneumonitis and Polymyositis, run by Tokyo Medical and Dental University at 11 sites in Japan.

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 15 inclusion and 31 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 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in Japan. 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 Mar 2020). 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?

Overall Survival (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 NCT00504348, 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 Mar 2020.