First-in-Human Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy

• Status: Recruiting
• Trial end date: 2022-06-15
• Target enrollment: 0
• Participant gender: All

Summary

A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Neurimmune AG

Criteria

Key Inclusion Criteria:

1. Age ≥18 years

2. Confirmed diagnosis of ATTR-Cardiomyopathy

3. Known genotype (wild-type or hereditary form)

4. Chronic Heart Failure with LVEF ≥40%, LVWT ≥14 mm, NT-proBNP ≥ 600 pg/mL, 6-MWT ≥150 meter, no hospitalizations for cardiac disease for at least 30 calendar days prior to screening

5. Karnofsky Performance Status score ≥60%

Key Exclusion Criteria:

1. Amyloid light-chain amyloidosis or any other non ATTR amyloidosis

2. New York Heart Association class IV

3. NT-proBNP ≥6000 pg/mL

4. Heart failure not predominantly caused by ATTR-Cardiomyopathy

5. Any severe uncorrected valve disease

6. Chronic liver disease with liver function test abnormalities

7. Respiratory insufficiency requiring oxygen therapy

8. Renal insufficiency

9. Active malignancy with exception of: adequately treated basal cell carcinoma, squamous cell carcinoma of the skin, in situ cervical cancer, low risk prostate cancer with Gleason score < 7 and prostate specific antigen < 10 mg/mL, any other cancer from which the subject has been disease-free for ≥ 2 years

10. Uncontrolled infection, HIV infection, seropositivity for HIV, hepatitis B and C, active hepatitis A

11. Autoimmune disease requiring immunosuppressive/modulating treatment in the last 2 years

12. History of organ transplantation or ventricular assist device

13. Polyneuropathy disability score > IIIA

14. Suspected or known drug or alcohol abuse, serious psychiatric or any other medical condition, which, in the opinion of the Investigator, makes the subject unsuitable