Sotatercept for Pulmonary Hypertension, Primary, 1

Phase-Based Estimates
2
Effectiveness
3
Safety
Hopitaux Universitaires de Strasbourg ( Site 1307), Strasbourg, France
Pulmonary Hypertension, Primary, 1+2 More
Sotatercept - Drug
Eligibility
18+
All Sexes
Eligible conditions
Pulmonary Hypertension, Primary, 1

Study Summary

This study is evaluating whether a new drug may help improve the progression of pulmonary hypertension.

See full description

Eligible Conditions

  • Pulmonary Hypertension, Primary, 1
  • Familial Primary Pulmonary Hypertension
  • Hypertension
  • Pulmonary Arterial Hypertension

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether Sotatercept will improve 1 primary outcome and 8 secondary outcomes in patients with Pulmonary Hypertension, Primary, 1. Measurement will happen over the course of From initiation of treatment to Week 24.

Week 24
Change from baseline in NT-proBNP levels.
Change in 6MWD.
Change in EuroQol - 5 dimensions scale 5 levels (EQ 5D 5L) index score.
Change in Pulmonary Arterial Hypertension Symptoms and Impact (PAH-SYMPACT)®
Multicomponent improvement endpoint of 6-minute walk distance (6MWD), NT-proBNP and WHO functional class (FC).
Proportion of participants who achieved a low Registry to Evaluate Early and Long Term PAH Disease Management (REVEAL) Lite 2 risk score.
Proportion of participants who improve in WHO FC or maintain WHO FC II at 24 weeks from baseline.
Proportion of participants who maintain or achieve a low simplified French risk score (FC)
Month 56
Time to Clinical Worsening, defined as the first confirmed morbidity event or death.

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Side Effects for

Sotatercept 0.1 mg/kg
Headache
43%
Oedema peripheral
29%
Thrombocytopenia
14%
Influenza
14%
Urinary tract infection
14%
Toothache
14%
Chest discomfort
14%
Back pain
14%
Ataxia
14%
Dizziness
14%
Cough
14%
Pruritus
14%
Vitreous floaters
14%
Bronchitis
14%
Abdominal pain upper
14%
Stomatitis
14%
Abdominal pain
0%
Alanine aminotransferase increased
0%
Cerebrovascular accident
0%
Neutropenia
0%
Nasopharyngitis
0%
Sinusitis
0%
Cholelithiasis
0%
Vertigo
0%
Blood creatinine increased
0%
Colitis
0%
Escherichia pyelonephritis
0%
Lung infection
0%
Spinal fracture
0%
Spinal compression fracture
0%
Subdural haematoma
0%
Diabetic foot
0%
Delirium
0%
Hemiparesis
0%
Haematuria
0%
Hypoxia
0%
Hypotension
0%
Aortic stenosis
0%
Haemolytic anaemia
0%
Sinus tachycardia
0%
Ear pain
0%
Eye haemorrhage
0%
Diarrhoea
0%
Salivary gland enlargement
0%
Dyspepsia
0%
Nausea
0%
Abdominal discomfort
0%
Dry mouth
0%
Gait disturbance
0%
Injection site reaction
0%
Face oedema
0%
Fatigue
0%
Pneumonia
0%
Tooth infection
0%
Contusion
0%
Upper respiratory tract infection
0%
Fall
0%
Peripheral swelling
0%
Blood bilirubin increased
0%
Creatinine renal clearance decreased
0%
Platelet count decreased
0%
Muscular weakness
0%
Tooth fracture
0%
Decreased appetite
0%
Blood alkaline phosphatase increased
0%
Muscle spasms
0%
Hyperkalaemia
0%
Myalgia
0%
Hypoglycaemia
0%
Iron overload
0%
Dehydration
0%
Neck pain
0%
Anxiety
0%
Arthralgia
0%
Musculoskeletal chest pain
0%
Osteopenia
0%
Basal cell carcinoma
0%
Osteoarthritis
0%
Dyspnoea
0%
Pain in extremity
0%
Insomnia
0%
Confusional state
0%
Gynaecomastia
0%
Rhinorrhoea
0%
Dry skin
0%
Ecchymosis
0%
Dysgeusia
0%
Depression
0%
Proteinuria
0%
Epistaxis
0%
Paraesthesia
0%
Pruritus generalised
0%
Rash
0%
Skin lesion
0%
Pyrexia
0%
Vessel puncture site swelling
0%
Lacrimation increased
0%
Eyelid oedema
0%
Anaemia
0%
Large intestine perforation
0%
Angina pectoris
0%
Cholangitis
0%
Non-cardiac chest pain
0%
Mass
0%
Clostridium difficile colitis
0%
Hip fracture
0%
Blood pressure increased
0%
International normalised ratio increased
0%
Peritoneal abscess
0%
Leukocytosis
0%
Transfusion reaction
0%
Oedema
0%
Constipation
0%
Asthenia
0%
Anaphylactic reaction
0%
Eye inflammation
0%
Pharyngitis
0%
Vomiting
0%
Influenza like illness
0%
Laryngitis
0%
Skin abrasion
0%
Muscle strain
0%
Aspartate aminotransferase increased
0%
Neutrophil count decreased
0%
Hyperglycaemia
0%
Lipase increased
0%
Hypercalcaemia
0%
Hyperuricaemia
0%
Arthritis
0%
Pollakiuria
0%
Dyspnoea exertional
0%
Nasal congestion
0%
Upper-airway cough syndrome
0%
Hypertension
0%
This histogram enumerates side effects from a completed 2018 Phase 2 trial (NCT01736683) in the Sotatercept 0.1 mg/kg ARM group. Side effects include: Headache with 43%, Oedema peripheral with 29%, Thrombocytopenia with 14%, Influenza with 14%, Urinary tract infection with 14%.

Trial Design

2 Treatment Groups

Placebo plus background PAH therapy
Sotatercept plus background PAH therapy
Placebo group

This trial requires 662 total participants across 2 different treatment groups

This trial involves 2 different treatments. Sotatercept is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Sotatercept plus background PAH therapy
Drug
Administered at a starting dose of 0.3 mg/kg, with a target dose of 0.7 mg/kg, subcutaneously (SC) every 21 days plus background PAH therapy
Placebo plus background PAH therapy
Other
Administered subcutaneously (SC) every 21 days plus background PAH therapy
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sotatercept
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from screening to the first clinical worsening event, up to 56 months.
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly from screening to the first clinical worsening event, up to 56 months. for reporting.

Closest Location

University of California San Diego Medical Center - La Jolla, CA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Pulmonary Hypertension, Primary, 1 or one of the other 2 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
REVEAL Lite 2 Risk Score ≥ 6
Diagnosis of PAH within 6 months of screening and on stable doses of a double combination of background PAH therapies for at least 90 days prior to screening. A triple combination of therapies, with stable doses for 90 days, may be allowed per local standard-of-care guidelines, but is restricted to 10% of the study population.
Six-minute walk distance ≥ 150 m repeated twice at screening at least 4 hours apart, but no longer than 1 week apart, and both values are within 15% of each other (calculated from the highest value)
Age ≥ 18 years
Idiopathic PAH
Drug/toxin-induced PAH
PAH associated with connective tissue disease
PAH associated with simple, congenital systemic to pulmonary shunts at least 1 year following repair
Heritable PAH
Symptomatic PAH classified as WHO FC II or III

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes pulmonary hypertension, primary, 1?

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Pulmonary hypertension is a chronic disease frequently accompanied by pulmonary-systemic shunts. Shunting occurs at various locations throughout the pulmonary circulation. The most common site, as determined by autopsy, is in the systemic to systemic-pulmonary vein anastomoses, and the superior and inferior pulmonary veins and the pulmonary capillary lobules. The main problem for the treatment of pulmonary hypertension, primary, 1, is that the arterial pressure of the pulmonary circulation is very low and fluctuates. Treatment should be aimed at reducing the number of shunts and at the arterial pressures to minimize symptoms. This can be achieved surgically by balloon angioplasty or by embolotherapy.

Unverified Answer

What is pulmonary hypertension, primary, 1?

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Patients with PVOD are commonly misdiagnosed as having pulmonary hypertension, primary, 1. The current criteria are inaccurate and unnecessarily specific. The current criteria should be revised. Pulmonary hypertension, primary, 1 is an easy diagnosis with a low complication rate. The prognosis is excellent when the treatment is prompt.

Unverified Answer

Can pulmonary hypertension, primary, 1 be cured?

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There is no evidence that pulmonary hypertension, primary, 1 can be cured. In order to determine whether pulmonary hypertension can be managed more efficiently, strategies that prevent its development and treatments to prevent death of the patient need to be developed and tried in future studies.

Unverified Answer

What are the signs of pulmonary hypertension, primary, 1?

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The signs of pulmonary hypertension include fatigue, lack of feeling in limbs, shortness of breath and wheezing. The symptoms vary according to the severity of the PH. If the PH is detected early, proper treatment and timely management can slow or reverse its progress.

Unverified Answer

What are common treatments for pulmonary hypertension, primary, 1?

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Pulmonary hypertension often presents with advanced degrees of heart failure and can lead to a sudden death from cardiac or pulmonary failure. Most cases are not diagnosed, mainly because of the unspecific symptoms (pulmonary pain, dyspnea, cough, and fatigue) or the reluctance of patients to visit a pulmonary specialist. There is no definitive test to determine the degree of PH. Nevertheless, PH can be defined differently based on its different causes (idiopathic, ischemic, or thromboembolic). It takes on an intermediate stage between primary (severe forms) and secondary (moderate and very severe forms) forms.

Unverified Answer

How many people get pulmonary hypertension, primary, 1 a year in the United States?

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Around 18,000 cases of idiopathic pulmonary hypertension are diagnosed each year in the United States, making it the most common disease of the lung in this population.

Unverified Answer

Have there been any new discoveries for treating pulmonary hypertension, primary, 1?

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Currently there is not a singular treatment for pulmonary hypertension, even though it is a very serious disease. There are many different treatment plans for this disease such as lung transplants, mechanical lung support, and new and more traditional therapies. Some of these therapies can be obtained through clinical trials, others can not. For those in clinical trials, it is essential that they meet all of the eligibility standards and criteria to join the trial. It is imperative for participants and doctors to be aware that the treatments and treatments have their own risks and benefits. It is important to weigh up the chances and outcomes of participating and receiving treatments.

Unverified Answer

Does pulmonary hypertension, primary, 1 run in families?

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Patients with a family history of pulmonary hypertension had a higher prevalence of familial pulmonary hypertension. This lends credence to the notion that mutations in BHLH gene are associated with primary familial pulmonary hypertension.

Unverified Answer

What is the latest research for pulmonary hypertension, primary, 1?

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The literature on treatment for pulmonary hypertension, primary, 1 is limited. No effective treatment for pulmonary hypertension, primary, 1 has been found to date. If there was an effective treatment, it would not be worthwhile to treat those with symptomatic pulmonary hypertension, primary, 1 because it could not be defined as preventing the morbidity and mortality from this disease.

Unverified Answer

What are the latest developments in sotatercept for therapeutic use?

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In March 2010, sotatercept (trade name Avapro) entered U.S. clinical practice and demonstrated safety and efficacy when used for treatment of pDMD. There are some intriguing data from clinical trials that suggest sotatercept may have unique properties compared to other approved agents. For example, patients with pDMD and non-diabetic kidney disease (creatinine clearance ≥30 ml/min) showed significantly greater improvements in renal function with sotatercept treatment than with placebo (37% decrease in eGFR with sotatercept vs 7% with placebo, p=0.002; 44% decrease in serum creatinine with sotatercept vs 16% with placebo, p=0.

Unverified Answer

Is sotatercept safe for people?

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In people with pulmonary arterial hypertension (PAP >60 mm Hg), (a) simeprevir-based triple therapy or a placebo-based regimen had comparable 6-month safety and efficacy. (b) a greater improvement in PAP was observed in simeprevir-treated vs. placebo-treated subjects.

Unverified Answer

Is sotatercept typically used in combination with any other treatments?

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Sotatercept monotherapy is commonly used to treat non-responders to pirfenidone or bosentan in patients with PH and PBC. Other treatments may need to be added either before or at the same time as sotatercept.

Unverified Answer
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