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

  • STATUS
    Recruiting
  • End date
    Feb 28, 2023
  • participants needed
    36
  • sponsor
    Neurimmune AG
Updated on 21 April 2022

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).

Description

This phase 1, randomized, placebo-controlled, double-blind trial in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM) consists of single-ascending dose (SAD) and multiple-ascending dose (MAD) phases, followed by an open-label extension (OLE) phase.

In the SAD phase subjects are randomized in a 4:2 ratio to receive a single infusion of NI006 or placebo.

Subjects completing the SAD phase will be enrolled in the MAD phase upon evaluation of all available safety data and receive a maximum of 3 additional infusions of NI006 or placebo every 28 days.

Subjects completing the MAD phase will have the possibility to continue in an OLE phase with treatment up-titrations and switch from placebo to NI006 and receive up to 8 infusions of NI006 every 28 days.

Subjects of cohort 1 to 5 who received at least one dose of NI006 during the OLE phase will have the possibility for a second OLE phase (OLE2) after completing the OLE phase and receive up to 10 additional infusions of NI006 every 28 days.

In total, about 30 to 36 subjects are planned to be enrolled in 5 to 6 ascending dose cohorts of 6 subjects each.

Details
Condition Amyloid Transthyretin Cardiomyopathy
Treatment Placebo, NI006
Clinical Study IdentifierNCT04360434
SponsorNeurimmune AG
Last Modified on21 April 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Age ≥18 years
Confirmed diagnosis of ATTR-Cardiomyopathy
Known genotype (wild-type or hereditary form)
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
Karnofsky Performance Status score ≥60%

Exclusion Criteria

Amyloid light-chain amyloidosis or any other non ATTR amyloidosis
New York Heart Association class IV
NT-proBNP ≥6000 pg/mL
Heart failure not predominantly caused by ATTR-Cardiomyopathy
Any severe uncorrected valve disease
Chronic liver disease with liver function test abnormalities
Respiratory insufficiency requiring oxygen therapy
Renal insufficiency
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
Uncontrolled infection, HIV infection, seropositivity for HIV, hepatitis B and C, active hepatitis A
Autoimmune disease requiring immunosuppressive/modulating treatment in the last 2 years
History of organ transplantation or ventricular assist device
Polyneuropathy disability score > IIIA
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
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