thalidomide and Amyloidosis

thalidomide has been researched along with Amyloidosis in 84 studies

Thalidomide: A piperidinyl isoindole originally introduced as a non-barbiturate hypnotic, but withdrawn from the market due to teratogenic effects. It has been reintroduced and used for a number of immunological and inflammatory disorders. Thalidomide displays immunosuppressive and anti-angiogenic activity. It inhibits release of TUMOR NECROSIS FACTOR-ALPHA from monocytes, and modulates other cytokine action.
thalidomide : A racemate comprising equimolar amounts of R- and S-thalidomide.
2-(2,6-dioxopiperidin-3-yl)-1H-isoindole-1,3(2H)-dione : A dicarboximide that is isoindole-1,3(2H)-dione in which the hydrogen attached to the nitrogen is substituted by a 2,6-dioxopiperidin-3-yl group.

Amyloidosis: A group of sporadic, familial and/or inherited, degenerative, and infectious disease processes, linked by the common theme of abnormal protein folding and deposition of AMYLOID. As the amyloid deposits enlarge they displace normal tissue structures, causing disruption of function. Various signs and symptoms depend on the location and size of the deposits.

Research

Studies (84)

Authors

Clinical Trials (14)

Trial Overview

Trial Outcomes

Duration of Response

Assessed as the median value for the time from first partial response until progression; death; or last follow-up. (NCT00890552)
Timeframe: 32 months

Event-free Survival (EFS)

Assessed as the median value for EFS 12 months after starting MDR treatment (NCT00890552)
Timeframe: 12 months

Overall Survival (OS)

Participants alive 12 months after starting MDR treatment. (NCT00890552)
Timeframe: 12 months

Hematologic Response Rate

At the end of each treatment cycle (4 weeks), hematologic response rate as assessed. Hematologic response was considered to be amyloid complete response (normal FLC ratio and negative serum and urine immunofixation); very good partial response (difference between involved and uninvolved FLCs [dFLC] < 40 mg/L); or partial response (dFLC decrease > 50%). (NCT00890552)
Timeframe: 8 weeks

Number of Patients Removed From Study Treatment Due to Toxicities

(NCT00091260)
Timeframe: 1 year

Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response

(NCT00091260)
Timeframe: 1 year

Number of Patients With Hematologic Response With Single-agent CC-5013

"Complete response = Absence of detectable monoclonal protein in serum or urine by immunofixation electrophoresis, less than 5% plasma cells on bone marrow biopsy without clonal dominance of kappa or lambda isotype, and normal serum free light chain assay.~Partial response= For patients with detectable and quantifiable monoclonal marrow plasmacytosis= a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. For patients with a detectable monoclonal peak on serum or urine protein electrophoresis= a reduction in the peak height of 50% or more.~For patients with quantifiable urinary kappa or lambda chain concentration= a 50% reduction in daily light chain excretion in 24 hour urine.~For patients with an elevated serum free light chain assay, a reduction of 50% or more." (NCT00091260)
Timeframe: 3 months

Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose

Number of patients in Phase I cohort 1 experiencing dose-limiting toxicity at the 2 milligram dose of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain amyloidosis (NCT01570387)
Timeframe: one month

Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 3 Milligram Dose

Number of patients in Phase I cohort 2 experiencing dose limiting toxicity in the 3 milligram dose level, cohort 2. (NCT01570387)
Timeframe: One month

Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 4 Milligram Dose

Number of patients in Phase I cohort 3 experiencing dose-limiting toxicity at the 4 milligram dose for participants within the third dose cohort (NCT01570387)
Timeframe: One month

Response to the Maximal Tolerated Dose

Number of participants with a response to treatment at that maximal tolerated dose (including partial, very good, or complete responses) (NCT01570387)
Timeframe: one year

Duration of Response

Duration of response was calculated from the documentation (date) of first response (CR, VGPR, or PR) until the date of progression or last follow-up in the subset of patients who responded. Kaplan Meier method was used to compute this outcome. (NCT00558896)
Timeframe: Duration of study (up to 5 years)

Progression Free Survival (PFS)

"PFS was defined as the time from registration to progression or death due to any cause. PFS was analyzed using Kaplan Meier method.~Progression was defined as any one or more of the following:~25% increase in serum M-component (absolute increase >= 0.5g/dl)~25% increase in urine M-component (absolute increase >= 200mg/24hour~25% increase in the difference between involved and uninvolved Free Light Chain levels (absolute increase >= 10mg/dl)~25% increase in bone marrow plasma cell percentage (absolute increase of >=10%)~Definite development of new bone lesion or soft tissue plasmacytomas" (NCT00558896)
Timeframe: Duration of study (up to 5 years)

The Number of Confirmed Hematologic Responses (Complete, Partial, or Very Good Partial Response)

"Response that was confirmed on 2 consecutive evaluations~Complete Response(CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and <5% plasma cells in bone marrow.~Very Good Partial Response(VGPR): >=90% reduction in serum M-component; Urine M-Component <100mg per 24hours; <=5% plasma cells in bone marrow.~Partial Response(PR): >=50% reduction in serum M-component and/or Urine M-Component >=90% reduction or <200mg per 24hours; or >=50% decrease in difference between involved and uninvolved FLC levels." (NCT00558896)
Timeframe: Duration of study (up to 3 years)

Number of Participants Who Achieved a Confirmed Response Defined as a Complete Response (CR), Very Good Partial Response (VGPR) or Partial Response (PR)

"Response that was confirmed on 2 consecutive evaluations during treatment.~Complete Response(CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and <5% plasma cells in bone marrow.~Very Good Partial Response(VGPR): >=90% reduction in serum M-component; Urine M-Component <=100 mg per 24 hours.~Partial Response(PR): >=50% reduction in serum M-component and/or Urine M-Component >=90% reduction or <200 mg per 24 hours; or >=50% decrease in difference between involved and uninvolved FLC levels." (NCT00564889)
Timeframe: Duration on study (up to 3 years)

Number of Participants With Severe Adverse Events

Severe adverse events were defined as grade 3 or higher, at least possibly related to study drugs. Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 3. (NCT00564889)
Timeframe: Duration of study (up to 3 years)

Number of Patients With Organ Response

"Organ response was evaluated on the basis of improvement of one or more affected organ; only one parameter was required to satisfy the criteria. Response needed to be maintained for a minimum of 3 months to be considered valid.~Renal response required a 50% reduction in 24-hour urine protein excretion (at least 0.5 g/d) with stable creatinine. Cardiac response required one of >= 2-mm reduction in the interventricular septal (IVS) thickness by echocardiogram, or improvement of ejection fraction by >= 20%, or improvement by 2 NYHA classes without an increase in diuretic use. Hepatic response required either >= 50% decrease in (or normalization of) an initially elevated alkaline phosphatase level or reduction in the size of the liver by at least 2 cm by radiographic determination. Gastrointestinal tract improvement was defined as normalization of a low serum carotene level, or reduction of diarrhea to < 50% of previous movements/day, or decrease in fecal fat excretion by 50%." (NCT00564889)
Timeframe: Duration of study (up to 3 years)

Overall Survival (OS)

Overall survival (OS) was defined as the time from registration to death of any cause. Surviving patients were censored at the date of last follow-up. The median OS with 95% CI was estimated using the Kaplan Meier method. (NCT00564889)
Timeframe: Duration of study (up to 3 years)

Progression Free Survival (PFS)

Progression free survival (PFS) was defined as the time from registration to hematologic progression or death of any cause. Progression free and alive patients were censored at the date of last follow-up. The median PFS with 95% CI was estimated using the Kaplan Meier method. (NCT00564889)
Timeframe: Duration of study (up to 3 years)

Number of Organs Improved or Stable Based on Description Below:

"Renal response - > 50% decrease in daily 24 hour proteinuria, without worsening renal insufficiency.~Hepatic response - decrease of 2 centimeters or more of the liver span and/or decrease of the alkaline phosphatase by 50% if elevated at baseline.~Cardiac response - decrease of 2 millimeters or more in mean left ventricular wall thickness in patients with baseline wall thickness > 11 mm or a decrease in New York Heart Association heart failure class.~Autonomic nervous system response - resolution of orthostatic vital signs and symptoms, and resolution of symptoms of gastric atony or of functional ileus.~Gastrointestinal response - a greater than one grade improvement in diarrhea due to biopsy proven amyloid.~Peripheral nervous system response - resolution of clinical signs of peripheral neuropathy." (NCT00679367)
Timeframe: one year

Number of Participants Removed From Study Due to Toxicities

Number of study participants removed from study treatment due to toxicities (NCT00679367)
Timeframe: One year

Number of Participants With Hematologic Response

"Complete hematologic response: Absence of detectable monoclonal protein in serum or urine by immunofixation electrophoresis, bone marrow biopsy with less than 5% plasma cells without clonal dominance of kappa or lambda isotype, and normal serum free light chain assay.~Partial hematologic response: Amyloid patients have highly individualized measures of disease burden. For patients with detectable and quantifiable monoclonal marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. For patients with a detectable monoclonal peak on serum or urine protein electrophoresis, a reduction in the peak height of 50% or more. For patients with quantifiable urinary kappa or lambda chain concentration, a 50% reduction in daily light chain excretion (concentration x 24 hour urine volume). For patients with an elevated serum free light chain assay, reduction of 50% or more." (NCT00679367)
Timeframe: one year

Reviews

19 reviews available for thalidomide and Amyloidosis

Trials

22 trials available for thalidomide and Amyloidosis

Other Studies

43 other studies available for thalidomide and Amyloidosis