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Cardiac AmyloidosisPhase 2TaPiOCANCT02966522

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

A Phase 2 study testing Thalidomide and Dexamethasone in people with Cardiac Amyloidosis, run by Seoul National University Hospital at 1 site in South Korea.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

To prove the organ-reversing potential of thalidomide for amyloidosis with cardiac involvement Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cardiac Amyloidosis
What is being tested?
Thalidomide and Dexamethasone
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 siteSouth Korea
Study dates
Oct 2016to Sep 2019
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 9Show fewer
  • 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.

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country.

Ryul Kim, MD contact

chrono0707@icloud.com

South Korea1 site, 1 recruiting

  • Seoul National University Hospital

    Seoul, 03080

    Youngil Koh, MD, PhD, go01@snu.ac.kr

    Ryul Kim, MD, chrono0707@icloud.com

    Recruiting

Other trials in South Korea

About this study

Official title: Frontline Thalidomide for Amyloidosis Involving Myocardium: Investigation of Organ Reversing Capacity of Lenalidomide

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

Read the detailed 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.

Keywords
Thalidoide
Medical subject headings
Amyloid Neuropathies, Familial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02966522
Study groupRoleWhat they receive
ThalidomideExperimentalPatient with cardiac amyloidosis receive thalilomide with dexamethasone
Receives: Drug: Thalidomide; Drug: Dexamethasone

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: through study completion, an average of 1 year

Secondary outcomes (8)

  1. Cardiac response

    > 30% and > 300 ng/l decrease in NTproBNP levels in patients with NTproBNP levels ≥ 650 ng/l at base-line or ≥ 2-class decrease in NYHA class in patients with NYHA class 3 or 4 at baseline

    Measured: through study completion, an average of 1 year

  2. Maximal LV myocardium-blood cavity ratio

    estimated by 11C-Pittsburge B PET imaging

    Measured: through study completion, an average of 1 year

  3. Overall survival

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

  4. Progression-free survival

    Measured: 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

  5. Toxicity profile related to thalidomide, according to CTCAE version 4.03

    Measured: through study completion, an average of 1 year

  6. Renal response

    > 50% (≥ 5.0 g/d) decrease in 24h urine protein levels in patients with urine protein levels > 0.5 g/l at baseline without ≥ 25% increase in serum creatinine levels or decrease in creatinine clearance from baseline

    Measured: through study completion, an average of 1 year

  7. Hepatic response

    ≥ 50% decrease in alkaline phosphatase levels and/or ≥ 2cm decrease in liver size (assessed by radiograph)

    Measured: through study completion, an average of 1 year

  8. Mean LV myocardium-blood cavity ratio

    estimated by 11C-Pittsburge B PET imaging

    Measured: through study completion, an average of 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Nov 2016 (estimated)
  2. Study startOct 2016
  3. Primary completionSep 2019 (estimated)
  4. Study completionSep 2019 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02966522
Sponsor's protocol ID
H-1512-129-730
Acronym
TaPiOCA
Status last verified
Oct 2017
First submitted
8 Nov 2016
Last updated
26 Oct 2017
Results posted
No

Publications

  • 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.

    PubMed 26491974

  • 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.

    PubMed 23294331

  • 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.

    PubMed 22493299

  • 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.

    PubMed 17008538

  • 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.

    PubMed 22517904

  • 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.

    PubMed 25499132

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Thalidomide and Dexamethasone in people with Cardiac Amyloidosis, run by Seoul National University Hospital at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 26 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Ryul Kim, MD (chrono0707@icloud.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hematologic response (time frame: through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02966522, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2017.

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