Trial Outcomes & Findings for Study of Pralatrexate With Vitamin B12 and Folic Acid Supplementation for Previously Treated Recurrent or Metastatic Head and Neck Squamous Cell Cancer (HNSCC) (NCT NCT01183065)

NCT ID: NCT01183065

Last Updated: 2015-11-20

Results Overview

by RECIST version 1.1 criteria

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 13 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2015-11-20

Participant Flow

Participant milestones

Measure	Pralatrexate and Vitamin Supplementation This will be an open-label, single arm, Simon optimal two-stage design phase II study. Pralatrexate With Vitamin B12 and Folic Acid: Patients will be treated with 30 mg/m2 of pralatrexate intravenously once weekly for 3 weeks in a 4 week cycle with vitamin supplementation. Patients will take 1.0-1.25 mg oral folic acid on a daily basis. Folic acid should be initiated during the 10-day period preceding the first dose of pralatrexate and dosing will be continued during the full course of therapy and for 30 days after the last dose of pralatrexate. Patients will also receive a vitamin B12 (1 mg) intramuscular injection no more than 10 weeks prior to the first dose of pralatrexate and every 8-10 weeks thereafter. Subsequent vitamin B12 injections may be given the same day as treatment with pralatrexate.
Overall Study STARTED	13
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure

Pralatrexate and Vitamin Supplementation This will be an open-label, single arm, Simon optimal two-stage design phase II study. Pralatrexate With Vitamin B12 and Folic Acid: Patients will be treated with 30 mg/m2 of pralatrexate intravenously once weekly for 3 weeks in a 4 week cycle with vitamin supplementation. Patients will take 1.0-1.25 mg oral folic acid on a daily basis. Folic acid should be initiated during the 10-day period preceding the first dose of pralatrexate and dosing will be continued during the full course of therapy and for 30 days after the last dose of pralatrexate. Patients will also receive a vitamin B12 (1 mg) intramuscular injection no more than 10 weeks prior to the first dose of pralatrexate and every 8-10 weeks thereafter. Subsequent vitamin B12 injections may be given the same day as treatment with pralatrexate.

Overall Study Patient no longer fits eligibility crite

1

Baseline Characteristics

Study of Pralatrexate With Vitamin B12 and Folic Acid Supplementation for Previously Treated Recurrent or Metastatic Head and Neck Squamous Cell Cancer (HNSCC)

Baseline characteristics by cohort

Measure	Pralatrexate and Vitamin Supplementation n=13 Participants This will be an open-label, single arm, Simon optimal two-stage design phase II study. Pralatrexate With Vitamin B12 and Folic Acid: Patients will be treated with 30 mg/m2 of pralatrexate intravenously once weekly for 3 weeks in a 4 week cycle with vitamin supplementation. Patients will take 1.0-1.25 mg oral folic acid on a daily basis. Folic acid should be initiated during the 10-day period preceding the first dose of pralatrexate and dosing will be continued during the full course of therapy and for 30 days after the last dose of pralatrexate. Patients will also receive a vitamin B12 (1 mg) intramuscular injection no more than 10 weeks prior to the first dose of pralatrexate and every 8-10 weeks thereafter. Subsequent vitamin B12 injections may be given the same day as treatment with pralatrexate.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

by RECIST version 1.1 criteria

Outcome measures

Measure

Pralatrexate and Vitamin Supplementation n=8 Participants This will be an open-label, single arm, Simon optimal two-stage design phase II study. Pralatrexate With Vitamin B12 and Folic Acid: Patients will be treated with 30 mg/m2 of pralatrexate intravenously once weekly for 3 weeks in a 4 week cycle with vitamin supplementation. Patients will take 1.0-1.25 mg oral folic acid on a daily basis. Folic acid should be initiated during the 10-day period preceding the first dose of pralatrexate and dosing will be continued during the full course of therapy and for 30 days after the last dose of pralatrexate. Patients will also receive a vitamin B12 (1 mg) intramuscular injection no more than 10 weeks prior to the first dose of pralatrexate and every 8-10 weeks thereafter. Subsequent vitamin B12 injections may be given the same day as treatment with pralatrexate.

To Determine the Overall Response Rate (CR+PR)

8 participants

Adverse Events

Pralatrexate and Vitamin Supplementation

Serious events: 8 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	Pralatrexate and Vitamin Supplementation n=12 participants at risk This will be an open-label, single arm, Simon optimal two-stage design phase II study. Pralatrexate With Vitamin B12 and Folic Acid: Patients will be treated with 30 mg/m2 of pralatrexate intravenously once weekly for 3 weeks in a 4 week cycle with vitamin supplementation. Patients will take 1.0-1.25 mg oral folic acid on a daily basis. Folic acid should be initiated during the 10-day period preceding the first dose of pralatrexate and dosing will be continued during the full course of therapy and for 30 days after the last dose of pralatrexate. Patients will also receive a vitamin B12 (1 mg) intramuscular injection no more than 10 weeks prior to the first dose of pralatrexate and every 8-10 weeks thereafter. Subsequent vitamin B12 injections may be given the same day as treatment with pralatrexate.
Injury, poisoning and procedural complications Injury/poison & proced complications Other, spec	8.3% 1/12 • Number of events 1
General disorders Fatigue	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Hypoxia	8.3% 1/12 • Number of events 1
General disorders Death NOS	33.3% 4/12 • Number of events 4
Respiratory, thoracic and mediastinal disorders Aspiration	8.3% 1/12 • Number of events 1
Nervous system disorders Depressed level of consciousness	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Mucositis oral	16.7% 2/12 • Number of events 2
Investigations Neutrophil count decreased	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Stridor	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Vomiting	8.3% 1/12 • Number of events 1

Other adverse events

Measure	Pralatrexate and Vitamin Supplementation n=12 participants at risk This will be an open-label, single arm, Simon optimal two-stage design phase II study. Pralatrexate With Vitamin B12 and Folic Acid: Patients will be treated with 30 mg/m2 of pralatrexate intravenously once weekly for 3 weeks in a 4 week cycle with vitamin supplementation. Patients will take 1.0-1.25 mg oral folic acid on a daily basis. Folic acid should be initiated during the 10-day period preceding the first dose of pralatrexate and dosing will be continued during the full course of therapy and for 30 days after the last dose of pralatrexate. Patients will also receive a vitamin B12 (1 mg) intramuscular injection no more than 10 weeks prior to the first dose of pralatrexate and every 8-10 weeks thereafter. Subsequent vitamin B12 injections may be given the same day as treatment with pralatrexate.
Investigations Alkaline phosphatase increased	8.3% 1/12 • Number of events 3
Metabolism and nutrition disorders Anorexia	8.3% 1/12 • Number of events 1
Investigations Aspartate aminotransferase increased	8.3% 1/12 • Number of events 1
Musculoskeletal and connective tissue disorders Back pain	8.3% 1/12 • Number of events 2
Musculoskeletal and connective tissue disorders Chest wall pain	8.3% 1/12 • Number of events 2
Gastrointestinal disorders Dry mouth	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Dysphagia	16.7% 2/12 • Number of events 2
General disorders Fatigue	25.0% 3/12 • Number of events 3
Nervous system disorders Headache	8.3% 1/12 • Number of events 1
Metabolism and nutrition disorders Hyperglycemia	16.7% 2/12 • Number of events 3
Metabolism and nutrition disorders Hyponatremia	8.3% 1/12 • Number of events 1
Metabolism and nutrition disorders Hypophosphatemia	8.3% 1/12 • Number of events 2
Investigations Lymphocyte count decreased	25.0% 3/12 • Number of events 5
Infections and infestations Mucosal infection	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Mucositis oral	33.3% 4/12 • Number of events 4
Gastrointestinal disorders Nausea	16.7% 2/12 • Number of events 2
Investigations Neutrophil count decreased	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Oral pain	8.3% 1/12 • Number of events 1
Investigations Platelet count decreased	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pleural effusion	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Vomiting	8.3% 1/12 • Number of events 2
Investigations Weight loss	16.7% 2/12 • Number of events 2
Investigations White blood cell decreased	16.7% 2/12 • Number of events 2

Additional Information

Ho, Alan, MD, PhD (Assistant Attending)

Memorial Sloan Kettering Cancer Center

Phone: +1646-888-4235

Email: HoA@mskcc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place