What side effects are associated with this type of intervention?

Common treatments for Pulmonary Arterial Hypertension (PAH) include endothelin receptor antagonists (ERAs), phosphodiesterase-5 inhibitors (PDE5Is), and soluble guanylate cyclase stimulators. Known side effects of these treatments can include headache, nausea, vomiting, diarrhea, flushing, and jaw pain. Specifically, ERAs may cause liver function abnormalities and fluid retention, while PDE5Is can lead to hypotension and visual disturbances. Soluble guanylate cyclase stimulators may also cause hypotension and dizziness. Treatments like Sotatercept, which target the TGF-beta superfamily, aim to reduce pulmonary vascular resistance and may have unique side effects that are still being studied.

What other types of research exist?

Promising novel alternatives to Sotatercept for PAH treatment include: 1) AT-877ER (fasudil hydrochloride), a Rho-kinase inhibitor, which has shown clinical benefits in PAH patients. 2) Evinacumab, an ANGPTL3 monoclonal antibody, which significantly lowers LDL-C and may have potential benefits in PAH. 3) Getagozumab, an antagonistic monoclonal antibody against Endothelin Receptor A, which has demonstrated efficacy in lowering pulmonary arterial pressure in PAH models.

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Sotatercept for Pulmonary Arterial Hypertension (MOONBEAM Trial)

Q: What prior FDA approvals exist?

The FDA has approved several treatments for Pulmonary Arterial Hypertension (PAH), including endothelin receptor antagonists (e.g., bosentan, ambrisentan), phosphodiesterase-5 inhibitors (e.g., sildenafil, tadalafil), and prostacyclin analogs (e.g., epoprostenol, treprostinil). These treatments work by different mechanisms to reduce pulmonary vascular resistance and improve symptoms. While Sotatercept, a ligand trap for TGF-beta superfamily members, is still under investigation, it represents a novel approach by targeting pathways involved in vascular remodeling. Other advanced therapies like riociguat, a soluble guanylate cyclase stimulator, have also been approved for PAH management.

Phase 2

Recruiting

Research Sponsored by Merck Sharp & Dohme LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Documented, historic diagnostic right heart catheterization (RHC) confirming the diagnosis of PAH WHO Group 1 in specified subtypes

Be younger than 18 years old

Must not have

Severe congenital or developmental abnormalities of the lung, thorax, and/or diaphragm

History of left-sided heart disease, including valvular disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up predose day 1, day 21, day 42, day 63, day 84, day105, day 126, day 147, day 168, day 189. postdose day 7, day 14, day 64, day 69 and day 76

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medication called sotatercept to see if it is safe and how it acts in children with high blood pressure in the lungs, who are already on standard treatments. The study will last for several months. Sotatercept has shown promise in improving exercise tolerance in adults with pulmonary arterial hypertension.

Who is the study for?

This trial is for children aged 1 to under 18 with Pulmonary Arterial Hypertension (PAH) who are already on stable PAH therapy. They must have a confirmed diagnosis and agree to contraception rules if applicable. It's not for those with certain heart or lung conditions, prior severe allergic reactions, or a family history of sudden cardiac death.

What is being tested?

The study tests the safety, tolerability, and how the body processes Sotatercept over 24 weeks in kids with PAH while they continue their standard treatments. There's no specific hypothesis; it's more about understanding these aspects of the drug in young patients.

What are the potential side effects?

While side effects aren't detailed here, typically such trials look out for any adverse reactions ranging from mild symptoms like headaches or nausea to more serious issues related to heart and lung function due to the nature of PAH treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a confirmed diagnosis of PAH Group 1 through a right heart catheterization.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have severe birth defects in my lung, chest, or diaphragm.

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I have had heart disease affecting the left side of my heart.

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I have a history of lung or heart conditions.

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I have a history of heart conditions.

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I have had allergic reactions to certain medications before.

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My family has a history of sudden heart deaths or long QT syndrome.

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I have a known abnormal connection between arteries and veins.

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I have been diagnosed with a specific lung condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ predose day 1, day 21, day 42, day 63, day 84, day105, day 126, day 147, day 168, day 189. postdose day 7, day 14, day 64, day 69 and day 76

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~predose day 1, day 21, day 42, day 63, day 84, day105, day 126, day 147, day 168, day 189. postdose day 7, day 14, day 64, day 69 and day 76

This trial's timeline: 3 weeks for screening, Varies for treatment, and predose day 1, day 21, day 42, day 63, day 84, day105, day 126, day 147, day 168, day 189. postdose day 7, day 14, day 64, day 69 and day 76 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Area Under the Curve at Steady State (AUCss) of Sotatercept

Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept

Blood Pressure (BP)

+9 more

Secondary study objectives

Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2)

Mean Change from Baseline in Eccentricity Index

Nesiritide

+8 more

Side effects data

From 2022 Phase 3 trial • 324 Patients • NCT04576988

20%

Headache

15%

COVID-19

12%

Diarrhoea

12%

Epistaxis

10%

Telangiectasia

10%

Nausea

10%

Fatigue

10%

Dizziness

Injection site pain

Hypokalaemia

Rash

Flushing

Oedema peripheral

Thrombocytopenia

Nasopharyngitis

Urinary tract infection

Dyspnoea

Iron deficiency

Bronchitis

Fall

Pulmonary arterial hypertension

Inguinal hernia

Atrial flutter

Supraventricular tachycardia

Abdominal pain

Pancreatitis

Cellulitis

Pneumonia

Pneumonia influenzal

Respiratory tract infection

Sepsis

Upper respiratory tract infection

Osteoporotic fracture

Acute kidney injury

Haemoptysis

Pulmonary artery aneurysm

Upper gastrointestinal haemorrhage

Joint injury

Sjogren's syndrome

Cerebral haematoma

Device malfunction

Nephritis

Sarcoidosis

Gastroenteritis viral

100%

80%

60%

40%

20%

Study treatment Arm

Placebo Plus Background PAH Therapy (LTDB Period)

Placebo Plus Background PAH Therapy (DBPC Period)

Sotatercept Plus Background PAH Therapy (DBPC Period)

Sotatercept Plus Background PAH Therapy (LTDB Period)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Children ≥1 to <18 years oldExperimental Treatment1 Intervention

Participants will receive a subcutaneous (SC) injection every 3 weeks (Q3W) of 0.3 mg/kg. Dosage may be adjusted based on protocol-specific guidelines.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Sotatercept

2019

Completed Phase 3

~690

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Pulmonary Arterial Hypertension (PAH) target various pathways to reduce pulmonary vascular resistance and improve patient outcomes. Endothelin receptor antagonists block the effects of endothelin-1, a potent vasoconstrictor, thereby reducing blood vessel constriction. Phosphodiesterase-5 inhibitors increase nitric oxide availability, leading to vasodilation and improved blood flow. Prostacyclin analogs mimic the effects of prostacyclin, promoting vasodilation and inhibiting platelet aggregation. Rho-kinase inhibitors, such as fasudil, reduce vasoconstriction by inhibiting the Rho-kinase pathway. Treatments like Sotatercept, which acts as a ligand trap for TGF-beta superfamily members, specifically target and reduce pulmonary vascular resistance. These mechanisms are crucial for PAH patients as they help alleviate symptoms, improve exercise capacity, and potentially slow disease progression.

Sorafenib as a potential strategy for refractory pulmonary arterial hypertension.Regulation of Pulmonary Vascular Smooth Muscle Contractility in Pulmonary Arterial Hypertension: Implications for Therapy.Double-blind, placebo-controlled clinical trial with a rho-kinase inhibitor in pulmonary arterial hypertension.