Primary Systemic AmyloidosisPhase 1NCT00030381
Iododoxorubicin in Treating Patients With Primary Systemic Amyloidosis
A Phase 1 study testing 4'-iodo-4'-deoxydoxorubicin and pharmacological study in people with Primary Systemic Amyloidosis, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Iododoxorubicin may dissolve protein deposits and be an effective treatment for primary systemic amyloidosis. Phase I trial to determine the effectiveness of iododoxorubicin in treating patients who have primary systemic amyloidosis Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Systemic Amyloidosis
- What is being tested?
- 4'-iodo-4'-deoxydoxorubicin and pharmacological study
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2001to Jan 2003
- Phase
- Phase 1
- 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
- Histochemically confirmed amyloidosis by polarizing microscopy of greenbirefringent material in Congo red-stained tissue specimens
- At least one of the following:
- Demonstrable M-protein in serum or urine
- Clonal population of plasma cells in bone marrow
- Immunohistochemical stain with anti-light chain antisera of amyloid fibrils
- Symptomatic organ involvement, including liver involvement, mild cardiac involvement, renal involvement, grade 1 or 2 peripheral neuropathy, or soft tissue involvement (including tongue)
Show all 42Show fewer
- No purpura or carpal tunnel syndrome as sole manifestation of disease
- No clinically overt multiple myeloma defined as monoclonal bone marrow platelet concentration greater than 20% and at least one of the following:
- Bone lesions
- Anemia
- Hypercalcemia
- Performance status - ECOG 0-3 (3 allowed only if related to muscular infiltration by amyloid or peripheral neuropathy)
- Platelet count at least 100,000/mm^3
- Absolute neutrophil count at least 1,500/mm^3
- Total bilirubin no greater than 2.0 mg/dL
- Direct bilirubin no greater than 1.0 mg/dL
- Alkaline phosphatase no greater than 4 times upper limit of normal (ULN)
- AST or ALT no greater than 3 times ULN
- Creatinine clearance at least 40 mL/min
- Ejection fraction at least 50% by echocardiogram
- No New York Heart Association class III or IV heart disease
- No enzyme-documented myocardial infarction within the past 3 years
- No chronic atrial fibrillation
- No grade 2 or 3 atrioventricular block (Mobitz type I allowed)
- No sustained (greater than 30 seconds) ventricular tachycardia, more than 1 episode of non-sustained ventricular tachycardia (3 consecutive ventricular beats), or frequent (more than 20 in 24 hours) ventricular pairs by 24-hour ambulatory electrocardiographic monitoring
- No intraventricular septum greater than 16 mm by echocardiogram
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No uncontrolled infection
- No other active malignancy except nonmelanoma skin cancer or cervical cancer
- No psychiatric illness or social situation that would preclude study
- No severe diarrhea (greater than grade 3) that is not controllable with medication or that requires total parenteral nutrition
- More than 4 weeks since prior interferon alfa
- No concurrent immunotherapy
- More than 4 weeks since prior melphalan or other alkylating agents
- No prior anthracycline exposure greater than 120 mg/m^2
- Recovered from prior chemotherapy
- No other concurrent chemotherapy
- More than 4 weeks since prior high-dose dexamethasone
- No concurrent radiotherapy
- No concurrent investigational ancillary therapy
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I Trial of 4'-IODO-4'-Deoxydoxorubicin in Primary Amyloidosis (AL)
Iododoxorubicin may dissolve protein deposits and be an effective treatment for primary systemic amyloidosis. Phase I trial to determine the effectiveness of iododoxorubicin in treating patients who have primary systemic amyloidosis
Read the detailed description
PRIMARY OBJECTIVES:
- Determine the maximum tolerated dose of iododoxorubicin in patients with primary systemic amyloidosis.
SECONDARY OBJECTIVES:
- Determine the safety, especially cardiac safety, of this drug in these patients.
II. Determine the survival rate of patients treated with this drug. III. Determine, preliminarily, the clinical efficacy of this drug in these patients.
IV. Determine the pharmacokinetics of this drug in these patients.
OUTLINE: This is a dose-escalation study.
Patients receive iododoxorubicin IV over 15 minutes on days 1, 8, 15, and 22. Treatment repeats every 12 weeks for a total of 4 courses or a cumulative dose of 400 mg/m^2 in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of iododoxorubicin until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed at 3 months.
- Conditions
- Primary Systemic Amyloidosis
- Being tested
- 4'-iodo-4'-deoxydoxorubicin, pharmacological study
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4'-iodo-4'-deoxydoxorubicinDrug
Also called IDOX, iododoxorubicin
Given IV
Also called pharmacological studies
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (iododoxorubicin) | Experimental | Patients receive iododoxorubicin IV over 15 minutes on days 1, 8, 15, and 22. Treatment repeats every 12 weeks for a total of 4 courses or a cumulative dose of 400 mg/m^2 in the absence of disease progression or unacceptable toxicity. Receives: Drug: 4'-iodo-4'-deoxydoxorubicin; Other: pharmacological study |
What is being measured
The primary outcome is the main question the study is designed to answer.
MTD of IDOX defined as the highest safely-tolerated dose where =< 1 patient experiences DLT with the next higher dose having at least 2 patients who experience DLT
The number and severity of toxicity incidents will indicate the level of tolerance of IDOX in the treatment of primary amyloidosis. Non-hematologic toxicities will be evaluated via the ordinal CTC standard toxicity grading. Hematologic toxicity measures of thrombocytopenia, neutropenia and leukopenia will be assessed using continuous variables as the outcome measures (primarily nadir and percent change from baseline values) as well as categorization via CTC standard toxicity grading.
Measured: 12 weeks
Secondary outcomes (1)
Laboratory correlates
Descriptive statistics and simple scatterplots will form the basis of presentation of these data. Correlations between these laboratory values and other outcome measures will be carried out by standard parametric and non-parametric correlation procedures (Pearson's and Spearman's coefficients). Prerequisite normality testing of these data will be carried out via standard Shapiro and Wilk (25) testing.
Measured: Up to 3 months post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2003 (estimated)
- Study startDec 2001
- Primary completionJan 2003
Why it stopped: Administratively complete.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Systemic AmyloidosisStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Angela Dispenzieri, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00030381
- Sponsor's protocol ID
- NCI-2012-02443
- Other IDs
- MC0113, U01CA069912, CDR0000069160
- Status last verified
- Jan 2013
- First submitted
- 14 Feb 2002
- Last updated
- 16 Jan 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial of 4'-IODO-4'-Deoxydoxorubicin in Primary Amyloidosis (AL)
- ClinicalTrials.gov ID
- NCT00030381
- Acronym / sponsor ID
- NCI-2012-02443
- Conditions
- Primary Systemic Amyloidosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Administratively complete.
- Enrolment
- 22 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Dec 2001
- Primary completion
- Jan 2003 (actual)
- First posted
- 17 Sep 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Iododoxorubicin may dissolve protein deposits and be an effective treatment for primary systemic amyloidosis. Phase I trial to determine the effectiveness of iododoxorubicin in treating patients who have primary systemic amyloidosis
Detailed description
PRIMARY OBJECTIVES:
- Determine the maximum tolerated dose of iododoxorubicin in patients with primary systemic amyloidosis.
SECONDARY OBJECTIVES:
- Determine the safety, especially cardiac safety, of this drug in these patients.
II. Determine the survival rate of patients treated with this drug. III. Determine, preliminarily, the clinical efficacy of this drug in these patients.
IV. Determine the pharmacokinetics of this drug in these patients.
OUTLINE: This is a dose-escalation study.
Patients receive iododoxorubicin IV over 15 minutes on days 1, 8, 15, and 22. Treatment repeats every 12 weeks for a total of 4 courses or a cumulative dose of 400 mg/m^2 in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of iododoxorubicin until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed at 3 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (iododoxorubicin) | Experimental | Drug: 4'-iodo-4'-deoxydoxorubicin, Other: pharmacological studyPatients receive iododoxorubicin IV over 15 minutes on days 1, 8, 15, and 22. Treatment repeats every 12 weeks for a total of 4 courses or a cumulative dose of 400 mg/m^2 in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (42 listed)
- Histochemically confirmed amyloidosis by polarizing microscopy of greenbirefringent material in Congo red-stained tissue specimens
- At least one of the following:
- Symptomatic organ involvement, including liver involvement, mild cardiac involvement, renal involvement, grade 1 or 2 peripheral neuropathy, or soft tissue involvement (including tongue)
- No clinically overt multiple myeloma defined as monoclonal bone marrow platelet concentration greater than 20% and at least one of the following:
- Performance status - ECOG 0-3 (3 allowed only if related to muscular infiltration by amyloid or peripheral neuropathy)
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4'-iodo-4'-deoxydoxorubicinAlso: IDOX, iododoxorubicinGiven IV | Drug | Treatment (iododoxorubicin) |
| pharmacological studyAlso: pharmacological studiesCorrelative studies | Other | Treatment (iododoxorubicin) |
Primary outcome
MTD of IDOX defined as the highest safely-tolerated dose where =< 1 patient experiences DLT with the next higher dose having at least 2 patients who experience DLT
Time frame: 12 weeks
The number and severity of toxicity incidents will indicate the level of tolerance of IDOX in the treatment of primary amyloidosis. Non-hematologic toxicities will be evaluated via the ordinal CTC standard toxicity grading. Hematologic toxicity measures of thrombocytopenia, neutropenia and leukopenia will be assessed using continuous variables as the outcome measures (primarily nadir and percent change from baseline values) as well as categorization via CTC standard toxicity grading.
1 secondary outcome
Laboratory correlates
Time frame: Up to 3 months post treatment
Results and safety
The study was stopped early (reason given: Administratively complete.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Angela Dispenzieri principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 4'-iodo-4'-deoxydoxorubicin and pharmacological study in people with Primary Systemic Amyloidosis, run by National Cancer Institute (NCI) at 1 site in United States.
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 42 inclusion and 0 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: stopped early, as of the sponsor's last update on 16 Jan 2013. 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Administratively complete.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MTD of IDOX defined as the highest safely-tolerated dose where =< 1 patient experiences DLT with the next higher dose having at least 2 patients who experience DLT (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 16 Jan 2013.