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

NCT02966522 | Unknown Phase 2

• 1 other identifier: H-1512-129-730
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Cardiac Amyloidosis

Keywords

Thalidoide

Outcome Measures

Primary Outcomes (1)

• Hematologic response

  Complete response: Normalization of FLC levels and κ to λ ratio, with nega-tive serum and urine immunofixation Very good partial response: de-creased of dFLC to \< 40mg/l Partial response: \> 50% reduction of dFLC

  through study completion, an average of 1 year

Secondary Outcomes (8)

• Cardiac response

  through study completion, an average of 1 year

• Maximal LV myocardium-blood cavity ratio

  through study completion, an average of 1 year

• Overall survival

  From date of enrollment until the date of death from any cause, assessed up to 60 months

• Progression-free survival

  From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 60 months

• Toxicity profile related to thalidomide, according to CTCAE version 4.03

  through study completion, an average of 1 year

Study Arms (1)

Thalidomide

EXPERIMENTAL

Patient with cardiac amyloidosis receive thalilomide with dexamethasone

Drug: Thalidomide; Drug: Dexamethasone

Interventions

Thalidomide DRUG

Thalidomide

Dexamethasone DRUG

Thalidomide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age : more than 18 years old
• 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
• ECOG(Eastern Cooperative Oncology Group) performance status ≤ 3
• 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
• Expected survival \> 3 months
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Seoul National University Hospital: lead
• CW pharmaceutical company: collaborator

Study Sites (1)

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Related Publications (6)

• Kastritis E, Dimopoulos MA. Recent advances in the management of AL Amyloidosis. Br J Haematol. 2016 Jan;172(2):170-86. doi: 10.1111/bjh.13805. Epub 2015 Oct 22.

  PMID: 26491974 BACKGROUND

• Gatt ME, Palladini G. Light chain amyloidosis 2012: a new era. Br J Haematol. 2013 Mar;160(5):582-98. doi: 10.1111/bjh.12191. Epub 2013 Jan 7.

  PMID: 23294331 BACKGROUND

• Dispenzieri A, Buadi F, Laumann K, LaPlant B, Hayman SR, Kumar SK, Dingli D, Zeldenrust SR, Mikhael JR, Hall R, Rajkumar SV, Reeder C, Fonseca R, Bergsagel PL, Stewart AK, Roy V, Witzig TE, Lust JA, Russell SJ, Gertz MA, Lacy MQ. Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis. Blood. 2012 Jun 7;119(23):5397-404. doi: 10.1182/blood-2012-02-413161. Epub 2012 Apr 4.

  PMID: 22493299 BACKGROUND

• Dispenzieri A, Lacy MQ, Zeldenrust SR, Hayman SR, Kumar SK, Geyer SM, Lust JA, Allred JB, Witzig TE, Rajkumar SV, Greipp PR, Russell SJ, Kabat B, Gertz MA. The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis. Blood. 2007 Jan 15;109(2):465-70. doi: 10.1182/blood-2006-07-032987. Epub 2006 Sep 28.

  PMID: 17008538 BACKGROUND

• Kastritis E, Terpos E, Roussou M, Gavriatopoulou M, Pamboukas C, Boletis I, Marinaki S, Apostolou T, Nikitas N, Gkortzolidis G, Michalis E, Delimpasi S, Dimopoulos MA. A phase 1/2 study of lenalidomide with low-dose oral cyclophosphamide and low-dose dexamethasone (RdC) in AL amyloidosis. Blood. 2012 Jun 7;119(23):5384-90. doi: 10.1182/blood-2011-12-396903. Epub 2012 Apr 18.

  PMID: 22517904 BACKGROUND

• Lee SP, Lee ES, Choi H, Im HJ, Koh Y, Lee MH, Kwon JH, Paeng JC, Kim HK, Cheon GJ, Kim YJ, Kim I, Yoon SS, Seo JW, Sohn DW. 11C-Pittsburgh B PET imaging in cardiac amyloidosis. JACC Cardiovasc Imaging. 2015 Jan;8(1):50-59. doi: 10.1016/j.jcmg.2014.09.018. Epub 2014 Nov 4.

  PMID: 25499132 BACKGROUND

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

Thalidomide; Dexamethasone

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Study Officials

• Youngil Koh, MD, PhD

  Seoul National University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ryul Kim, MD

CONTACT

chrono0707@icloud.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical professor

Study Record Dates

• First Submitted: November 8, 2016
• First Posted: November 17, 2016
• Study Start: October 1, 2016
• Primary Completion: September 1, 2019
• Study Completion: September 1, 2019
• Last Updated: October 26, 2017

Record last verified: 2017-10

Locations

KR (1)