Clinical trial NCT06321705: Safety and Efficacy of Continued CS1 Treatment of Patients With Pulmonary Arterial Hypertension
Official title: Expanded Access, Open-Label, Safety Extension Study for Patients That Have Completed Parent Study CS1-003 and Who Are Judged by the Investigator to Benefit From Continued CS1 Treatment
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Pulmonary Arterial Hypertension
Keywords: Pulmonary Arterial Hypertension
CardioMEMS
Lead sponsor: Cereno Scientific AB
Sponsor class: INDUSTRY
Collaborators: name: Worldwide Clinical Trials
name: Abbott
First posted: 2024-03-20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT

Brief summary: CS1-004 will be an extension of the CS1-003 Study. The primary objective of the CS1-004 study is to evaluate long-term safety and tolerability of continued treatment with CS1.

Detailed description: CS1-004 will be an extension of the CS1-003 Study. The primary objective of the CS1-004 study is to evaluate long-term safety and tolerability of continued treatment with CS1. The exploratory objectives are to evaluate clinical benefit by using Cardio-Microelectromechanical system (CardioMEMS), echocardiography, cardiac magnetic resonance imaging, right heart catheterization, quality of life assessments, and actigraphy to measure changes in clinical response with continued treatment with CS1 in patients with Pulmonary Arterial Hypertension (PAH).

Up to 30 patients are planned for enrollment in the ongoing phase 2 Study CS1-003; thus up to 30 patients could participate in Study CS1-004, if they completed the parent study, tolerated CS1 treatment, and in the investigator's judgement the benefits of continued treatment with CS1 outweigh the risk. Note that as the parent study is ongoing; thus, some patients will directly roll- over into Study CS1-004 with no disruption in CS1 treatment, while other patients that have already completed Study CS1-003, will need to be restarted on CS1. On a yearly basis, the principal investigator will determine if a patient continues the expanded access study for a subsequent year based on safety, tolerability, and clinical benefit of CS1. Continuation of the expanded access study will also be evaluated on a yearly basis by Cereno Scientific (Sponsor).

Eligibility criteria: Inclusion Criteria:

1. Patient has derived benefit from CS1 treatment in parent Study CS1-003 based on the Investigator's judgement and the benefit of continued CS1 treatment outweighs the risk or the patient could benefit from continued treatment with CS1.
2. Patient must be willing and able to sign a written informed consent prior to any study-related procedures and able to understand and follow instructions; return to the study unit for specified study visits; and able to participate in the study for the entire period.
3. Patient is classified as New York Heart Association/World Health Organisation (NYHA/WHO) functional Class I, II or III.
4. Patient has CardioMEMS PA Sensor implanted and has completed treatment per parent protocol in Phase 2 Study CS1-003.
5. Female patient of childbearing potential must be willing and able to practice effective contraception during the study and continuing contraception for 30 days after their last dose of study drug. A female patient of non-childbearing potential is defined as being surgically sterilized by bilateral tubal ligation, bilateral oophorectomy, or hysterectomy. A female patient 45 to 60 years of age, who is post-menopausal for at least 1 year, and has a follicle-stimulating hormone level confirmation indicating post menopausal status will be considered of non-childbearing potential. A female patient \>60 years of age is considered post menopausal and of non childbearing potential.

Exclusion Criteria:

1. Patient has a concomitant medical disorder that is expected to limit the patient's life-expectancy to ≤1 year.
2. Patient has thrombocytopenia: platelets \<100,000/mm3.
3. Patient has a recent history of pancreatic disease.
4. Patient is a pregnant or lactating female.
5. Patient has active coronavirus disease 19 (COVID-19); however, those with previous COVID 19 are permitted.
6. Patient is participating in another investigational study which, in the opinion of the Investigator, would interfere with the study compliance or outcome assessments.
7. Patient is on regular treatment with other anti-epilepsy drugs or other prohibited medications (See Section 6.8.1) that cannot be discontinued at Roll-over or Restart Visit (V1).
8. Patient is on regular anticoagulation or on antiplatelet therapy that cannot be discontinued. A low daily dose aspirin (\<125 mg) is allowed, ie, "baby" aspirin.
9. Patient has significant liver dysfunction as measured by any one of the following at Roll-over or Restart Visit (V1) (including patients with acute or chronic hepatitis as well as patients with own or family history of serious hepatitis, especially drug related):

   1. Alanine aminotransferase (ALT) \>2.0 × upper limit of normal (ULN).
   2. Aspartate aminotransferase (AST) \>2.0 × ULN.
   3. Serum bilirubin ≥1.6 mg/dL or \>2.0 × ULN.
10. Patient with any major surgical procedure or trauma within 30 days prior to Roll-over or Restart Visit (V1) or planned surgical procedure during the study period.
11. Patient has known hypersensitivity to study drug or any of the excipients of the drug formulation.
12. Patient prematurely discontinued CS1 treatment in parent Study CS1-003 due to safety concerns of the Investigator.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Gothenburg
country: Sweden
facility: Fredrik Frick
Study officials:
References: