A Long-term Follow-up Study of Sotatercept for PAH Treatment

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Acceleron Pharma Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

26 Aug 2021 → ongoing

Decision date (initial)

2024-11-12

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ, USA

External identifiers

EU CT number

2023-509138-21-00

EudraCT number

2020-005061-13

WHO UTN

U1111-1309-6218

ClinicalTrials.gov

NCT04796337

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Therapy, Safety, Pharmacodynamic

The primary objective of this open-label, LTFU study is to evaluate the long-term safety and tolerability of sotatercept when added to background PAH therapy in adult participants with PAH.

Secondary objectives 1

1. The secondary objective is to follow participants from parent sotatercept studies that were treated with sotatercept or placebo and assess continued efficacy.

Conditions and MedDRA coding

Pulmonary Arterial Hypertension (PAH)

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Participants must have completed their current respective PAH sotatercept clinical study, must have completed the parent study requirements, and must not have discontinued early. 2. Participants must be willing to adhere to the study visit schedule and understand and comply with all protocol requirements. 3. Participants must have the ability to understand and provide documented consent 4. Females of childbearing potential must: a. Have a negative pregnancy tests as verified by the investigator prior to starting study drug administration; she must agree to ongoing pregnancy testing during the course of the study and until 8 weeks after the last dose of the study drug. b. If sexually active, have used, and agree to continue to use highly effective contraception in combination with a barrier method without interruption, for at least 28 days prior to starting the investigational product, during the study (including dose interruptions), and for 16 weeks (112 days) after discontinuation of study drug. c. Refrain from breastfeeding a child or donating blood, eggs, or ovum for the duration of the study and for at least 16 weeks (112 days) after the last dose of study drug. 5. Male participants must: a. Agree to use a condom, defined as a male latex condom or non-latex condom NOT made out of natural (animal) membrane (e.g., polyurethane), during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions, and for at least 16 weeks (112 days) following investigational product discontinuation, even if he has undergone a successful vasectomy b. Refrain from donating blood or sperm for the duration of the study and for 16 weeks (112 days) after the last dose of study drug. 6. Participants must agree not to participate in any other trials of investigational drugs/devices while they are enrolled in the A011-12 study, except for MK-7962-038 study.

Exclusion criteria 1

1. Did not participate in a sotatercept PAH parent trial. 2. Missed more than the equivalent of 4 consecutive doses between the end of parent study and the start of this study. 3. Presence of an ongoing Serious Adverse Event (SAE) that occurred during a PAH sotatercept clinical study that is assessed to be possibly or probably related to sotatercept. 4. Pregnant or breastfeeding females.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. • Adverse events (AEs) • Anti-drug antibodies (ADA) • Clinical laboratory assessments (hematology) • Vital signs • 12-lead electrocardiogram (ECG)

Secondary endpoints 1

1. • 6-minute walk distance (6MWD) • N-terminal pro-hormone B-type natriuretic peptide • World Health Organization (WHO) functional class • Pulmonary vascular resistance • Simplified French Risk score

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Acceleron Pharma Inc.

Sponsor organisation

Acceleron Pharma Inc.

Address

126 East Lincoln Avenue

City

Rahway

Postcode

07065-4607

Country

United States

Scientific contact point

Organisation

Acceleron Pharma Inc.

Contact name

Howard Julien

Public contact point

Organisation

Acceleron Pharma Inc.

Contact name

Howard Julien

Third parties 12

Locations

14 EU/EEA countries · 58 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 121 files

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

Application history

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