An Open-Label Extension Study for Participants with PAH Who Have Previously Participated in a Clinical Trial with PF-07868489

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Pfizer Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

19 Dec 2025 → ongoing

Decision date (initial)

2025-12-02

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Pfizer Inc., 66 Hudson Boulevard East, New York, NY 10001, USA

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the long-term safety and tolerability of repeat subcutaneous (SC) doses of PF-07868489 in participants with PAH.

Secondary objectives 3

1. To characterize change in blood concentration of NT-pro-BNP following repeat SC dose administration of PF-07868489 in participants with PAH.
2. To characterize serum exposure following repeat SC doses of PF-07868489 in participants with PAH.
3. To evaluate the immunogenicity profile of PF-07868489 following repeat SC doses in participants with PAH.

Conditions and MedDRA coding

Pulmonary Arterial Hypertension

Regulatory references

Scientific advice from competent authorities

Paul-Ehrlich-Institut

Plan to share IPD

Yes

IPD plan description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 18 years of age or older at screening of the qualifying (parent) study (or the minimum age of consent in accordance with local regulations), who have completed at least 20-week treatment period and week 24 study procedures of the qualifying study.
2. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
3. Capable of giving signed informed consent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol.

Exclusion criteria 11

1. Any medical or psychiatric condition including any active suicidal ideation in the past year or suicidal behavior in the past 5 years or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
2. Participants with clinical worsening or hospitalization for worsening PAH during the qualifying study, placement on a transplant list, and planned surgical procedure for PAH.
3. Participants with an ongoing clinically significant safety event in the qualifying parent study (C5001001 Part B) based on CTCAE Grade 3 or higher (Grade 4 for PAH-related) or in the opinion of the investigator that would preclude treatment with open label PF-07868489.
4. History or currently established hepato-pulmonary syndrome.
5. Use of any prohibited concomitant medication(s) during the qualifying or current study or unwillingness or inability to use a required concomitant medication(s) or change in stable background PAH medications used in the qualifying study. Refer to Section 6.9.
6. Current use of GLP-1 agonist.
7. Participation in studies of other investigational products (drug or vaccine) at any time during participation in this study.
8. Any of the following clinical chemistry values at baseline:  ALT or AST > 3× ULN (> 5 ULN if solely due to right heart failure) or total bilirubin ≥2 × ULN (For Gilbert’s syndrome, direct bilirubin >ULN [or ≥ 2 × ULN if solely due to right heart failure] is exclusionary).
9. Poor compliance with study visits or procedures during the qualifying C5001001 study, as assessed by the investigator or the Pfizer Medical Monitor.
10. Participants who met discontinuation criteria at any point during the participating qualifying C5001001 study.
11. Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly involved in the conduct of the study and their family members.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. Incidence and severity of treatment emergent Adverse Events (AEs) and Serious Adverse Events (SAEs).
2. Incidence of related treatment emergent AEs (TEAEs) & treatment emergent SAEs (TESAEs).
3. Change from baseline in vital signs.
4. Change from baseline in clinical laboratory values.
5. Change from baseline in electrocardiogram (ECG) parameters (heart rate, QT, QTc corrected using Fridericia’s formula (QTcF), PR, and QRS intervals).

Secondary endpoints 3

1. Change from baseline in NT-pro-BNP.
2. PF-07868489 PK parameters after repeat doses; as data permit: minimum plasma concentration (Cmin).
3. Incidence of the development of antidrug antibody (ADA) against PF-07868489 following repeat doses.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pfizer Inc.

Sponsor organisation

Pfizer Inc.

Address

66 Hudson Boulevard East

City

New York

Postcode

10001-2189

Country

United States

Scientific contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Public contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Third parties 8

Locations

7 EU/EEA countries · 21 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

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