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UMIN-CTR Clinical Trial |
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Name: | UMIN ID: |
Recruitment status | Main results already published |
Unique ID issued by UMIN | UMIN000018497 |
Receipt No. | R000021402 |
Scientific Title | Prospective study of I-131 3-iodobenzylguanidine radiotherapy for refractory pheochromocytoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs. |
Date of disclosure of the study information | 2015/07/31 |
Last modified on | 2020/02/13 |
Basic information | ||
Public title | Prospective study of I-131 3-iodobenzylguanidine radiotherapy for refractory pheochromocytoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs. | |
Acronym | Prospective study of I-131 3-iodobenzylguanidine radiotherapy for refractory pheochromocytoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs. | |
Scientific Title | Prospective study of I-131 3-iodobenzylguanidine radiotherapy for refractory pheochromocytoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs. | |
Scientific Title:Acronym | Prospective study of I-131 3-iodobenzylguanidine radiotherapy for refractory pheochromocytoma; in accordance with the Japanese Advanced Medical Care B program for the anticancer drug with high medical needs. | |
Region |
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Condition | ||||
Condition | Refractory pheochromocytoma (including paraganglioma) | |||
Classification by specialty |
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Classification by malignancy | Malignancy | |||
Genomic information | NO |
Objectives | |
Narrative objectives1 | The aim of this study is to evaluate the safety and the efficacy of I-131 3-iodobenzylguanidine (I-131 MIBG) radiotherapy for I-123 MIBG-avid refractory pheochromocytoma, along with the following primary and secondary endpoints. Additionally, this study was performed in accordance with the Japanese Advanced Medical Care B program ahead to sponsor initiated clinical trial, which intend to rationalize application for approval under the Japanese Pharmaceutical and Medical Device Act. |
Basic objectives2 | Safety,Efficacy |
Basic objectives -Others | |
Trial characteristics_1 | |
Trial characteristics_2 | |
Developmental phase |
Assessment | |
Primary outcomes | Dose limiting toxicity (DLT) |
Key secondary outcomes | Types and frequency of adverse event/reaction
Response rate under the RECIST criteria Response rate under scintigraphic evaluation of MIBG Overall survival (OS) Progression-free survival (PFS) |
Base | |
Study type | Interventional |
Study design | |
Basic design | Single arm |
Randomization | Non-randomized |
Randomization unit | |
Blinding | Open -no one is blinded |
Control | Uncontrolled |
Stratification | |
Dynamic allocation | |
Institution consideration | |
Blocking | |
Concealment |
Intervention | ||
No. of arms | 1 | |
Purpose of intervention | Treatment | |
Type of intervention |
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Interventions/Control_1 | I-131 3-iodobenzylguanidine (I-131 MIBG) radiotherapy
Administering the radiopharmaceutical in accordance with the following regimen every 24 weeks until satisfying withdrawal or dose-reduction criteria. Radiopharmaceutical: 131I-MIBG Dose: 7,400MBq (=200mCi)* i.v. in 1 hour *Administering maximal dose of permitted amount of radioisotopes in each facility, if permitted amount is lower than 7,400MBq. |
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Interventions/Control_2 | ||
Interventions/Control_3 | ||
Interventions/Control_4 | ||
Interventions/Control_5 | ||
Interventions/Control_6 | ||
Interventions/Control_7 | ||
Interventions/Control_8 | ||
Interventions/Control_9 | ||
Interventions/Control_10 |
Eligibility | ||||
Age-lower limit |
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Age-upper limit |
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Gender | Male and Female | |||
Key inclusion criteria | Satisfying all the following conditions.
1)Confirmed pheochromocytoma, paraganglioma, malignant pheochromocytoma, or malignant paraganglioma. 2)Not applicable any kind of surgical treatment and radical external irradiation. 3)I-123 MIBG-avid tumor. 4)Satisfying each condition: 4)-1 WBC >= 3,000[/mm3], Hb >= 9.0[g/dL] and platelets >= 100,000[/mm3] under G-CSF non-administraion. 4)-2 eGFR >= 30[mL/min/1.73m2] 4)-3 AST < 100[IU/L], ALT < 100[IU/L] and LDH < 400[IU/L] 4)-4 New York Heart Association (NYHA) Functional Classification class I or below. 4)-5 HbA1c < 8.0% 4)-6 Oxygen saturation >= 96[%] at room air. 5)Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 to 2 and Karnofsky Performance Scale (KPS) 80[%] or more. 6)Age 20 or older. 7)Independent feeding, excretion and sleeping. 8)Written consent to participate in this study and treatment. |
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Key exclusion criteria | Satisfying any of the following conditions.
1)Previous or current malignancies of other histologies within the last 5 years. 2)History of tumor deterioration under the condition of I-131 MIBG radiotherapy before this study. 3)History of Common Terminology Criteria for Adverse Events (CTCAE) grade 2 or more in non-hematotoxicity, or grade 3 or more in hematotoxicity under the condition of I-131 MIBG radiotherapy before this study. 4)CTCAE grade 2 or more toxicity currently treated under the condition of any kind of anticancer treatments before this study. 5)Diagnosed as Hepatitis B virus, Hepatitis C virus, Human Immunodeficiency virus (HIV) or any other infections currently treated. 6)Episodes of severe symptoms due to uncontrollable increase of catecholamines. 7)Episodes of fatal arrhythmia or asystole. 8)Diagnosed as any of the following diseases or conditions out of control. 8)-1 symptomatic arrhythmia 8)-2 thyroid dysfunction (hyperthyroidism or hypothyroidism) 8)-3 respiratory disease 8)-4 pleural effusion or ascites 9)Diagnosed as any of the following disease or conditions. 9)-1 coronary artery disease 9)-2 administration of amiodarone 9)-3 severe valvular disease of the heart 9)-4 aortic disease 9)-5 bleeding disorder 10)Pregnant or lactating women, or desire to bear children. 11)Diagnosed as psychosis. 12)Diagnosed as any diseases currently treated with adrenal corticosteroids or immunosuppressants. 13)Not applicable isolation due to radiation control. 14)Episodes of allergic reaction to potassium iodide. 15)Any symptomatic lesions currently treated with palliative external irradiation. |
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Target sample size | 20 |
Research contact person | |||||||
Name of lead principal investigator |
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Organization | Kanazawa University | ||||||
Division name | Institute of Medical, Pharmaceutical and Health Sciences | ||||||
Zip code | 920-8641 | ||||||
Address | 13-1, Takara-machi, Kanazawa | ||||||
TEL | 076-265-2333 | ||||||
kinuya@med.kanazawa-u.ac.jp |
Public contact | |||||||
Name of contact person |
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Organization | Kanazawa University | ||||||
Division name | Institute of Medical, Pharmaceutical and Health Sciences | ||||||
Zip code | 920-8641 | ||||||
Address | 13-1, Takara-machi, Kanazawa | ||||||
TEL | 076-265-2333 | ||||||
Homepage URL | |||||||
henri@staff.kanazawa-u.ac.jp |
Sponsor | |
Institute | Kanazawa University |
Institute | |
Department |
Funding Source | |
Organization | Japan Agency for Medical Research and Development |
Organization | |
Division | |
Category of Funding Organization | Other |
Nationality of Funding Organization |
Other related organizations | |
Co-sponsor | |
Name of secondary funder(s) |
IRB Contact (For public release) | |
Organization | Innovative Clinical Research Center, Kanazawa University |
Address | 13-1, Takara-machi, Kanazawa |
Tel | 076-265-2090 |
crc.irb-knz@esct.jp |
Secondary IDs | |
Secondary IDs | NO |
Study ID_1 | |
Org. issuing International ID_1 | |
Study ID_2 | |
Org. issuing International ID_2 | |
IND to MHLW |
Institutions | |
Institutions | 金沢大学附属病院(石川県)
鹿児島大学病院(鹿児島県) 北海道大学病院(北海道) 群馬大学医学部附属病院(群馬県) |
Other administrative information | |||||||
Date of disclosure of the study information |
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Related information | |
URL releasing protocol | https://www.jstage.jst.go.jp/article/jmi/64/3.4/64_205/_article |
Publication of results | Published |
Result | |||||||
URL related to results and publications | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6527850/ | ||||||
Number of participants that the trial has enrolled | 20 | ||||||
Results | No DLT was found. There was no death event within 6 months after enrollment. However, one patient died due to disease progression.
No grade 4 adverse event or unexpected adverse reactions were found. The response based on RECIST was 2 in CR, 13 in SD, 3 in PD, and 2 in NE. RR was 10%. The scintigraphic response in the first course was 2 in CR, 5 in PR, 8 in SD, 4 in PD, and one in non-CR/non-PD. RR was 35%. There was no death within 6 months of enrollment and OS and PFS were 100% and 80%, respectively. |
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Results date posted |
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Results Delayed | |||||||
Results Delay Reason | |||||||
Date of the first journal publication of results |
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Baseline Characteristics | Sex (F:M) 6:14
Age 51.2 +- 14.4 (range: 21-76) years Severe local invasion at initial diagnosis (25%, 5/20) Metastasis at initial diagnosis (25%, 5/20) Local recurrence after surgical resection (20%, 4/20) Metastasis after surgical resection (65%, 13/20) |
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Participant flow | We enrolled 20 patients between February 2016 and July 2017. | ||||||
Adverse events | Adverse reactions by PT
Thrombocytopenia 15/20, 75.0% Loss of appetite 14/20, 70.0% Lymphopenia 13/20, 65.0% Nausea 10/20, 50.0% Leukopenia 10/20, 50.0% |
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Outcome measures | The best overall response rate based on RECIST was 10% (2/20) in complete response (CR), 65% (13/20) in stable disease (SD), 15% (3/20) in progressive disease (PD), and 10% (2/20) in not evaluated (NE). The response rate [partial response (PR) + CR] was 10%.
The scintigraphic response in the first course was 10%(2/20) in CR, 25% (5/20) in PR, 40% in SD (8/20), 20% in PD (4/20), and 5% in non-CR/non-PD (1/20). Response rate (PR + CR) was 35% (95% CI: 15.4-59.2%). There was no death within 6 months of enrollment and overall survival (OS) rate was 100%. Four patients had progression events during the 6 months since enrollment and progression-free survival (PFS) at 6 months was 80.0%. |
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Recruitment status | Main results already published | ||||||
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Last modified on |
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Link to view the page | |
URL(English) | https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000021402 |
Research Plan | |
Registered date | File name |
2016/04/03 | 01_160115_MIBG_protocol_1.3.pdf |
Research case data specifications | |
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Research case data | |
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