Extension Study of Inhaled Treprostinil in Subjects with Fibrotic Lung Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

United Therapeutics Corp.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

1 Mar 2024 → ongoing

Decision date (initial)

2023-10-13

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

UNITED THERAPEUTICS CORPORATION

External identifiers

EU CT number

2023-504471-25-00

ClinicalTrials.gov

NCT04905693

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the long-term safety and tolerability of inhaled treprostinil in subjects with IPF or PPF.

Secondary objectives 1

1. To provide or continue to provide inhaled treprostinil for eligible subjects who participated in RIN-PF-301, RIN-PF-303 or RIN-PF-305.

Conditions and MedDRA coding

Fibrotic Lung Disease

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Subject gives voluntary informed consent to participate in the study.
2. The subject participated in RIN-PF-301, RIN-PF-303 or RIN-PF-305 and had 1 of the following applied: a. Remained on study drug and completed all scheduled study visits b. Was enrolled in RIN-PF-301, RIN-PF-303 or RIN-PF-305 at the time that the study or study subject was discontinued by the Sponsor for reasons other than safety.
3. Women of childbearing potential must be non-pregnant (as confirmed by a urine pregnancy test at OLE Entry Visit) and non-lactating, and will agree to do 1 of the following: a. Abstain from intercourse (when it is in line with their preferred and usual lifestyle) b. Use 2 medically acceptable, highly effective forms of contraception for the duration of the study, and at least 30 days after discontinuing study drug. Medically acceptable, highly effective forms of contraception can include approved hormonal contraceptives (oral, injectable, and implantable) and barrier methods (such as a condom or diaphragm) when used with a spermicide. Women who are successfully sterilized or postmenopausal are not considered to be of reproductive potential.
4. Males with a partner of childbearing potential must agree to use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug.
5. In the opinion of the Investigator, the subject is able to communicate effectively with study personnel, and is considered reliable, willing, and likely to be cooperative with protocol requirements, including attending all study visits.

Exclusion criteria 3

1. Subject is pregnant or lactating.
2. In the opinion of the Investigator, enrollment in RIN-PF-302 would represent a risk to the subject’s overall health.
3. Use of any other investigational drug/device or participation in any investigational study in which the subject received a medical intervention (ie, procedure, device, medication/supplement). Subjects participating in non-interventional, observational, or registry studies are eligible.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 10

1. Efficacy will be assessed by evaluating the effect of continued long-term therapy with inhaled Treprostinil on the following parameters: 1. Change in absolute FVC
2. 2. Time to clinical worsening (including time to death, respiratory hospitalization, or ≥10% relative decline in % predicted FVC)
3. 3. Time to acute exacerbation of IPF or ILD
4. 4. Overall survival
5. 5. Change in % predicted FVC
6. 6. Change in K-BILD score
7. 7. Change in N-terminal pro-brain natriuretic peptide
8. 8. Change in DLCO
9. 9. Change in resting supplemental oxygen use
10. SAFETY ENDPOINTS Safety will be assessed by reviewing the following parameters: • AEs and serious adverse events (SAEs) • Clinical laboratory parameters •Vital signs, including resting saturation of peripheral capillary oxygenation (SpO2) • 12-lead electrocardiograms (ECGs)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

United Therapeutics Corp.

Sponsor organisation

United Therapeutics Corp.

Address

55 Tw Alexander Drive

City

Research Triangle Park

Postcode

27709-0152

Country

United States

Scientific contact point

Organisation

United Therapeutics Corp.

Contact name

Peter Smith

Public contact point

Organisation

United Therapeutics Corp.

Contact name

Regulatory Department

Third parties 6

Locations

7 EU/EEA countries · 52 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 48 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

15 submissions — initial application plus substantial / non-substantial modifications