Trial Outcomes & Findings for A Phase II Study of 131I- Metaiodobenzylguanidine (MIBG) for Treatment of Metastatic or Unresectable Pheochromocytoma and Related Tumors (NCT NCT01413503)

Last Updated: 2018-09-18

Results Overview

Patients with complete (CR), partial (PR), or minor (MR) response and without progressive disease 1 year from initial treatment, using RECIST RESPONSE CRITERIA for measurable soft tissue tumor: CR=No Tumor (Primary or metastatic); catacholamines, metanephrines and chromogranin A all normal. PR=Primary and all measurable sites decreased \>50%; number of positive bone sites decreased by \>50%; bone marrow tumor decreased by 50%. MR=No new lesions; \>50% reduction of any measurable lesion (primary or metastases); \<25% increase in any existing lesion.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

50 participants

Primary outcome timeframe

After 1 year from initial treatment

Results posted on

2018-09-18

Participant Flow

Participant milestones

Participant milestones
Measure	131I-MIBG Patients received 131I-MIBG 8-12 mCi/kg (maximum 500 mCi ± 10% at investigator's discretion) diluted in 25 ml of normal saline. Patients were infused intravenously through a patient's peripheral or central line over 120 minutes
Overall Study STARTED	50
Overall Study COMPLETED	49
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	131I-MIBG Patients received 131I-MIBG 8-12 mCi/kg (maximum 500 mCi ± 10% at investigator's discretion) diluted in 25 ml of normal saline. Patients were infused intravenously through a patient's peripheral or central line over 120 minutes
Overall Study Lost to Follow-up	1

Baseline Characteristics

A Phase II Study of 131I- Metaiodobenzylguanidine (MIBG) for Treatment of Metastatic or Unresectable Pheochromocytoma and Related Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	131I-MIBG n=49 Participants Patients received 131I-MIBG infused intravenously through a peripheral or central line over 120 minutes. Of 50 patients enrolled and treated, one patient was lost to follow-up.
Age, Continuous	42.6 years n=99 Participants
Sex: Female, Male Female	20 Participants n=99 Participants
Sex: Female, Male Male	29 Participants n=99 Participants
Region of Enrollment United States	49 participants n=99 Participants

PRIMARY outcome

Timeframe: After 1 year from initial treatment

Outcome measures

Outcome measures
Measure	131I-MIBG n=49 Participants Patients received 131I-MIBG infused intravenously through a peripheral or central line over 120 minutes
Number of Patients With Complete (CR), Partial (PR), or Minor (MR) Response and Without Progressive Disease	28 Participants

Adverse Events

131I-MIBG

Serious events: 6 serious events

Other events: 43 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	131I-MIBG n=49 participants at risk Patients received 131I-MIBG 8-12 mCi/kg (maximum 500 mCi ± 10% at investigator's discretion) diluted in 25 ml of normal saline. Patients were infused intravenously through a patient's peripheral or central line over 120 minutes
Respiratory, thoracic and mediastinal disorders Bronchiolitis obliterans organizing pneumonia	4.1% 2/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Blood and lymphatic system disorders Myelodysplastic syndrome	4.1% 2/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	4.1% 2/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Vascular disorders Pulmonary Embolism	2.0% 1/49 • Number of events 1 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009

Other adverse events

Other adverse events
Measure	131I-MIBG n=49 participants at risk Patients received 131I-MIBG 8-12 mCi/kg (maximum 500 mCi ± 10% at investigator's discretion) diluted in 25 ml of normal saline. Patients were infused intravenously through a patient's peripheral or central line over 120 minutes
Blood and lymphatic system disorders Neutropenia	87.8% 43/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Blood and lymphatic system disorders Thrombocytopenia	83.7% 41/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	2.0% 1/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Infections and infestations Febrile neutropenia	14.3% 7/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Vascular disorders Acute hypertension	14.3% 7/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Infections and infestations Infection	4.1% 2/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Reproductive system and breast disorders Hypogonadism	8.2% 4/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009
Endocrine disorders Hyperthyroidism	6.1% 3/49 All study records, including participant charts, IND files, and staff computers, were destroyed in a flood. Partial Adverse Event information was derived from publication-PubMed ID: 19636009

Additional Information

Paul A. Fitzgerald, MD

University of California, San Francisco

Phone: 415-665-1136

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place