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

NCT ID: NCT02966522

Last Updated: 2017-10-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-31

Study Completion Date

2019-09-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cardiac Amyloidosis

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Thalidoide

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Thalidomide

Patient with cardiac amyloidosis receive thalilomide with dexamethasone

Group Type EXPERIMENTAL

Thalidomide

Intervention Type DRUG

Dexamethasone

Intervention Type DRUG

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Thalidomide

Intervention Type DRUG

Dexamethasone

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

CW pharmaceutical company

UNKNOWN

Sponsor Role collaborator

Seoul National University Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Youngil Koh

Clinical professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Youngil Koh, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Seoul National University Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Seoul National University Hospital

Seoul, , South Korea

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

South Korea

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Ryul Kim, MD

Role: CONTACT

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Youngil Koh, MD, PhD

Role: primary

Ryul Kim, MD

Role: backup

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type BACKGROUND
PMID: 26491974 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 23294331 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 22493299 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17008538 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 22517904 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 25499132 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

H-1512-129-730

Identifier Type: -

Identifier Source: org_study_id