Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis (TaPiOCA)

Seoul National University

Status and phase: Unknown; Phase 2

Conditions: Cardiac Amyloidosis

Treatments: Drug: Thalidomide; Drug: Dexamethasone

Study type: Interventional

Funder types: Other

Identifiers

NCT02966522

H-1512-129-730

Details and patient eligibility

About

To prove the organ-reversing potential of thalidomide for amyloidosis with cardiac involvement

Full description

Considering that dismal prognosis of amyloidosis is attributable to organ dysfunction, primary aim of amyloidosis treatment should be an organ reversal. However, due to various reasons, not much is known about organ reversal in amyloidosis. Almost all of the clinical trials evaluated hematologic response in amyloidosis. Meanwhile, besides autologous stem cell transplantation with high-dose melphalan conditioning, hematologic response rate of various agents such as bortezomib, melphalan, thalidomide and lenalidomide are similar for amyloidosis. However, organ reversing potential of these agents is not known. If there is a difference in organ reversing potential despite of similar hematologic response rate, drug with effective organ reversing potential should be a standard treatment for amyloidosis.

The investigators assume that thalidomide could make organ reversal in cardiac amyloidosis due to its specific mechanism of action. To prove this concept, the investigators propose a clinical trial that evaluates organ reversing potential of thalidomide in cardiac amyloidosis.

Enrollment: 30 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age : more than 18 years old

2. Cardiac amyloidosis (both AL or AH type) patient who meet both (A and B) of the following criteria A. Cardiac involvement: meet one of the following criteria

   • Echocardiography: mean wall thickness >12 mm, and no other cardiac cause
   • NTproBNP >332 ng/l in the absence of renal failure
   • Evidence of cardiac amyloidosis in cardiac MRI B. Treatment history: No history of exposure to thalidomide

3. ECOG(Eastern Cooperative Oncology Group) performance status ≤ 3

4. Tolerable major organ function determined by laboratory examination i. Serum creatinine ≤ 3.0 mg/dl ii. Absolute neutrophil count ≥ 1000/μl iii. Platelet ≥ 75000/ μl iv. Hemoglobin ≥ 8.0 mg/dl v. Bilirubin < 2 times or Alkaline phosphate < 4 times upper limit of normal

5. Expected survival > 3 months

6. Female participants of child-bearing potential must have a negative pregnancy test prior to treatment and agree to use dual methods of contraception for the duration of the study and for 30 days following completion of study. Male participants must also agree to use a barrier method of contraception for the duration of the study and for 30 days following completion of study if sexually active with a female of child-bearing potential.

Exclusion criteria

1. Amyloidosis without cardiac involvement
2. Patients who are planning to receive autologous stem cell transplantation
3. Patients who received autologous stem cell transplantation, remained in hematologic complete response
4. Pregnant, lactating or unwilling to use adequate contraception
5. Systemic infection unless specific anti-infective therapy is employed
6. Known allergies to thalidomide
7. Previous experimental agents or approved anti-tumor treatment within 1 months before the date of registration

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Thalidomide

Experimental group

Description:

Patient with cardiac amyloidosis receive thalilomide with dexamethasone

Treatment:

Drug: Dexamethasone

Drug: Thalidomide