An Open-label Extension Study Evaluating the Long-term Safety and Efficacy of Seralutinib Orally Inhaled for the Treatment of Pulmonary Arterial Hypertension (PAH)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

GB002 Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

12 Jul 2024 → ongoing

Decision date (initial)

2024-06-24

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

GB002, Inc.

External identifiers

EU CT number

2023-506334-75-00

ClinicalTrials.gov

NCT06274801

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the long-term safety and tolerability of seralutinib in subjects with PAH

Secondary objectives 1

1. To evaluate the long-term effect of seralutinib on exercise capacity when added to background PAH disease-specific medication. To evaluate the long-term effect of seralutinib on NT-proBNP when added to background PAH disease-specific medication.

Conditions and MedDRA coding

Pulmonary Arterial Hypertension

Study design 1 period

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. "1. Subjects must have completed a prior seralutinib PAH study on investigational product (IP) and in accordance with the protocol. 2. Women of childbearing potential (WOCBP) must have a negative urine or serum pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin [hCG]) at Enrollment visit before first administration of seralutinib. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test is required, and results must be negative. 3. WOCBP who are not abstinent and intend to be sexually active with a non-sterilized male partner must be willing to use a highly effective method of contraception from consent through 30 days following the last administration of seralutinib; acceptable methods include hormonal contraception (oral contraceptives – provided stable dose, patch, implant, or injection), intrauterine devices, or other form of highly effective contraception. Female subjects must refrain from egg donation throughout this period. 4. Women of nonchildbearing potential (WONCBP), classified by one of the following: a. Surgical sterilization (bilateral oophorectomy, bilateral salpingectomy, tubal ligation, Falope® rings, clips, or bands; segmental destruction of the fallopian tubes with electrocoagulation; suture ligation, a microinsert such as the Essure® system, or hysterectomy). b. Evidence of post-menopausal status. Women will be considered post-menopausal if they have been amenorrheic for 12 months or more following cessation of exogenous hormonal treatments and have luteinizing hormone (LH), follicle stimulating hormone (FSH), and estradiol levels in the post-menopausal range at the Enrollment visit. 5. Male subjects: Non-sterilized male subjects who are not abstinent and intend to be sexually active with a female partner of childbearing potential must use a male condom from consent through 90 days after the last dose of seralutinib. Male subjects must refrain from sperm donation throughout this period. 6. Evidence of an informed consent document, signed and dated by the subject, indicating that the subject has been informed of all pertinent aspects of the study prior to initiation of any subject-mandated procedures. 7. Willingness and ability to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. "

Exclusion criteria 1

1. "1. Severe acute or chronic medical or laboratory abnormality that may increase the risk associated with study participation or seralutinib administration, unless associated with an ongoing AE and discussed with the Sponsor’s MM (or designee). 2. Have any other condition or reason that, in the opinion of the Investigator would prohibit the subject from participating in the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of treatment-emergent adverse events (TEAEs)

Secondary endpoints 2

1. 1. Changes in distance achieved on the six-minute walk test (6MWT), (Δ6MWD)
2. 2. Changes in NT-proBNP

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

GB002 Inc.

Sponsor organisation

GB002 Inc.

Address

3013 Science Park Road

City

San Diego

Postcode

92121-1101

Country

United States

Scientific contact point

Organisation

GB002 Inc.

Contact name

Conor O'Donovan

Public contact point

Organisation

GB002 Inc.

Contact name

Conor O'Donovan

Third parties 14

Locations

16 EU/EEA countries · 53 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 251 files

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

Application history

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