Trial Outcomes & Findings for Folate Receptor Alpha Peptide Vaccine With GM-CSF Versus GM-CSF Alone in Patients With Platinum Sensitive Ovarian Cancer (NCT NCT02978222)

Last Updated: 2022-12-15

Results Overview

Time to disease progression or recurrence of ovarian cancer defined as disease progression by RECIST 1.1., disease recurrence, death without progression or CA125 progression

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

120 participants

Primary outcome timeframe

2 years

Results posted on

2022-12-15

Participant Flow

The first subject screened for trial 20 Jul 2017 and the last subject enrolled was screened 29Nov2018. Seventeen sites (academic and private clinics) in the United States enrolled and treated patients in the trial. The trial was terminated on 18 Oct 2019, after a blinded analysis by the DSMB determined that the futility requirements were not met. While the trial was terminated on 10/18/2019, the last patient could not return for a final visit/safety evaluation until January 15, 2020.

Enrolled subjects were only excluded from the trial if it was found that inclusion/exclusion criteria were not met for continued participation.

Participant milestones

Participant milestones
Measure	FRα Peptide Plus Adjuvant (GM-CSF) FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Overall Study STARTED	62	58
Overall Study COMPLETED	1	2
Overall Study NOT COMPLETED	61	56

Reasons for withdrawal

Reasons for withdrawal
Measure	FRα Peptide Plus Adjuvant (GM-CSF) FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Overall Study Withdrawal by Subject	2	2
Overall Study Physician Decision	2	0
Overall Study Adverse Event	0	1
Overall Study Death	1	0
Overall Study Disease Progression	32	32
Overall Study Early Study Termination	24	21

Baseline Characteristics

Folate Receptor Alpha Peptide Vaccine With GM-CSF Versus GM-CSF Alone in Patients With Platinum Sensitive Ovarian Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=62 Participants FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=58 Participants GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF	Total n=120 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	40 Participants n=93 Participants	34 Participants n=4 Participants	74 Participants n=27 Participants
Age, Categorical >=65 years	22 Participants n=93 Participants	24 Participants n=4 Participants	46 Participants n=27 Participants
Sex: Female, Male Female	62 Participants n=93 Participants	58 Participants n=4 Participants	120 Participants n=27 Participants
Sex: Female, Male Male	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=93 Participants	6 Participants n=4 Participants	9 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	58 Participants n=93 Participants	52 Participants n=4 Participants	110 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	2 Participants n=93 Participants	3 Participants n=4 Participants	5 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	3 Participants n=93 Participants	1 Participants n=4 Participants	4 Participants n=27 Participants
Race (NIH/OMB) White	55 Participants n=93 Participants	53 Participants n=4 Participants	108 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=93 Participants	1 Participants n=4 Participants	3 Participants n=27 Participants
Region of Enrollment United States	62 participants n=93 Participants	58 participants n=4 Participants	120 participants n=27 Participants
More than 6 months to last date of platinum therapy	9 Participants n=93 Participants	3 Participants n=4 Participants	12 Participants n=27 Participants

PRIMARY outcome

Timeframe: 2 years

Time to disease progression or recurrence of ovarian cancer defined as disease progression by RECIST 1.1., disease recurrence, death without progression or CA125 progression

Outcome measures

Outcome measures
Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=61 Participants FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=58 Participants GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Progression Free Survival	11.1 Months Interval 8.1 to The confidence interval upper bound is not able to be estimated because the Kaplan-Meier curve representing the upper confidence limits of the survival function lies above 0.5	10.9 Months Interval 8.0 to 16.8

SECONDARY outcome

Timeframe: 2 years

Death with or without ovarian cancer progression

Outcome measures

Outcome measures
Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=61 Participants FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=58 Participants GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Overall Survival (OS)	NA Months Point estimates are not able to be estimates because the Kaplan-Meier does not cross 0.5. Confidence interval upper bounds cannot be estimated because the Kaplan-Meier curve representing the upper confidence limits of the survival function lies above 0.5. Additionally, the confidence interval lower bound for the FRα Peptide Plus Adjuvant (GM-CSF) Alone group cannot be estimated because the Kaplan-Meier curve representing the lower confidence limits of the survival function lies above 0.5.	NA Months Interval 22.8 to Point estimates are not able to be estimates because the Kaplan-Meier does not cross 0.5. Confidence interval upper bounds cannot be estimated because the Kaplan-Meier curve representing the upper confidence limits of the survival function lies above 0.5. Additionally, the confidence interval lower bound for the FRα Peptide Plus Adjuvant (GM-CSF) Alone group cannot be estimated because the Kaplan-Meier curve representing the lower confidence limits of the survival function lies above 0.5.

SECONDARY outcome

Timeframe: 2 years

Population: Subject in MITT with measurable disease at baseline.

Best overall response defined as sum of Complete Responses and Partial Responses in the subset of patients with measurable tumor lesions at baseline. Best overall response is a binary endpoint and defined as a best overall response of either CR or PR among subjects with measurable lesions at baseline, using RECIST v1.1. Disease control rate is the percentage of subjects with a response of CR, PR, or SD or non-CR/non-PR vs PD among subjects with measurable lesions at baseline.

Outcome measures

Outcome measures
Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=6 Participants FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=4 Participants GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Best Overall Response Rate	0 Participants	3 Participants

SECONDARY outcome

Timeframe: 2 years

Population: Subjects in MITT with measurable lesions at baseline.

Disease control rate defined as the sum of Complete Responses, Partial Responses and Stable Disease. Best overall response is a binary endpoint and defined as a best overall response of either CR or PR among subjects with measurable lesions at baseline, using RECIST v1.1. Disease control rate is the percentage of subjects with a response of CR, PR, or SD or non-CR/non-PR vs PD among subjects with measurable lesions at baseline.

Outcome measures

Outcome measures
Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=6 Participants FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=4 Participants GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Disease Control Rate	6 Participants	4 Participants

Adverse Events

FRα Peptide Plus Adjuvant (GM-CSF)

Serious events: 8 serious events

Other events: 61 other events

Deaths: 1 deaths

Adjuvant (GM-CSF) Alone

Serious events: 8 serious events

Other events: 56 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Gerald Garrett

Marker Therapeutics

Phone: 713.400.6400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=62 participants at risk FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=58 participants at risk GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Respiratory, thoracic and mediastinal disorders dyspnea	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders colitis	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Cardiac disorders acute myocardial infarction	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Nervous system disorders Dizziness	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Injury, poisoning and procedural complications Closed Right Hip Fracture	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders small intestinal obstruction	1.6% 1/62 • Number of events 2 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Unspecified (incl) cysts and polyps; ovarian cancer	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Nervous system disorders Headache	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) malignant melanoma in situ	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Infections and infestations Pneumonia-unknown etiology	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Ear and labyrinth disorders vertigo	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders Abdominal Pain	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.
Vascular disorders thromboembolic event	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Blood and lymphatic system disorders anemia	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Infections and infestations Pneumonia	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders oral mucositis	1.6% 1/62 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.	0.00% 0/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders small bowel obstruction	0.00% 0/62 • Adverse events were collected for two years after the first vaccine administration.	1.7% 1/58 • Number of events 1 • Adverse events were collected for two years after the first vaccine administration.

Measure	FRα Peptide Plus Adjuvant (GM-CSF) n=62 participants at risk FRα peptide vaccine with GM-CSF adjuvant ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years FRα peptide plus Adjuvant (GM-CSF): Intradermal injection FRα peptides, 500μg each - plus GM-CSF 125 μg	Adjuvant (GM-CSF) Alone n=58 participants at risk GM-CSF adjuvant alone ID administration monthly for 6 months followed by booster administrations every 3 months for up to 1.5 years Adjuvant (GM-CSF) Alone: Intradermal injection 125 μg GM-CSF
Gastrointestinal disorders abdominal distension	8.1% 5/62 • Adverse events were collected for two years after the first vaccine administration.	12.1% 7/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders abdominal pain	19.4% 12/62 • Adverse events were collected for two years after the first vaccine administration.	24.1% 14/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders consipation	6.5% 4/62 • Adverse events were collected for two years after the first vaccine administration.	10.3% 6/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders diarrhea	17.7% 11/62 • Adverse events were collected for two years after the first vaccine administration.	8.6% 5/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders Nausea	14.5% 9/62 • Adverse events were collected for two years after the first vaccine administration.	12.1% 7/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders vomiting	11.3% 7/62 • Adverse events were collected for two years after the first vaccine administration.	8.6% 5/58 • Adverse events were collected for two years after the first vaccine administration.
Gastrointestinal disorders Gastroesophageal Reflux Disease	4.8% 3/62 • Adverse events were collected for two years after the first vaccine administration.	5.2% 3/58 • Adverse events were collected for two years after the first vaccine administration.
General disorders Fatigue	21.0% 13/62 • Adverse events were collected for two years after the first vaccine administration.	20.7% 12/58 • Adverse events were collected for two years after the first vaccine administration.
General disorders injection site reaction	64.5% 40/62 • Adverse events were collected for two years after the first vaccine administration.	37.9% 22/58 • Adverse events were collected for two years after the first vaccine administration.
General disorders pyrexia	6.5% 4/62 • Adverse events were collected for two years after the first vaccine administration.	5.2% 3/58 • Adverse events were collected for two years after the first vaccine administration.
Infections and infestations sinusitis	4.8% 3/62 • Adverse events were collected for two years after the first vaccine administration.	5.2% 3/58 • Adverse events were collected for two years after the first vaccine administration.
Infections and infestations upper respiratory tract infection	4.8% 3/62 • Adverse events were collected for two years after the first vaccine administration.	8.6% 5/58 • Adverse events were collected for two years after the first vaccine administration.
Infections and infestations urinary tract infection	11.3% 7/62 • Adverse events were collected for two years after the first vaccine administration.	6.9% 4/58 • Adverse events were collected for two years after the first vaccine administration.
Injury, poisoning and procedural complications fall	3.2% 2/62 • Adverse events were collected for two years after the first vaccine administration.	5.2% 3/58 • Adverse events were collected for two years after the first vaccine administration.
Investigations white blood cell count decreased	1.6% 1/62 • Adverse events were collected for two years after the first vaccine administration.	5.2% 3/58 • Adverse events were collected for two years after the first vaccine administration.
Musculoskeletal and connective tissue disorders arthralgia	17.7% 11/62 • Adverse events were collected for two years after the first vaccine administration.	17.2% 10/58 • Adverse events were collected for two years after the first vaccine administration.
Musculoskeletal and connective tissue disorders back pain	11.3% 7/62 • Adverse events were collected for two years after the first vaccine administration.	10.3% 6/58 • Adverse events were collected for two years after the first vaccine administration.
Musculoskeletal and connective tissue disorders myalgia	8.1% 5/62 • Adverse events were collected for two years after the first vaccine administration.	10.3% 6/58 • Adverse events were collected for two years after the first vaccine administration.
Musculoskeletal and connective tissue disorders pain in extremity	6.5% 4/62 • Adverse events were collected for two years after the first vaccine administration.	6.9% 4/58 • Adverse events were collected for two years after the first vaccine administration.
Nervous system disorders headache	8.1% 5/62 • Adverse events were collected for two years after the first vaccine administration.	8.6% 5/58 • Adverse events were collected for two years after the first vaccine administration.
Psychiatric disorders insomnia	4.8% 3/62 • Adverse events were collected for two years after the first vaccine administration.	6.9% 4/58 • Adverse events were collected for two years after the first vaccine administration.
Respiratory, thoracic and mediastinal disorders cough	14.5% 9/62 • Adverse events were collected for two years after the first vaccine administration.	10.3% 6/58 • Adverse events were collected for two years after the first vaccine administration.
Skin and subcutaneous tissue disorders alopecia	4.8% 3/62 • Adverse events were collected for two years after the first vaccine administration.	5.2% 3/58 • Adverse events were collected for two years after the first vaccine administration.
Skin and subcutaneous tissue disorders pruritus	9.7% 6/62 • Adverse events were collected for two years after the first vaccine administration.	6.9% 4/58 • Adverse events were collected for two years after the first vaccine administration.
Skin and subcutaneous tissue disorders rash maculo-papular	8.1% 5/62 • Adverse events were collected for two years after the first vaccine administration.	13.8% 8/58 • Adverse events were collected for two years after the first vaccine administration.
Vascular disorders hypertension	1.6% 1/62 • Adverse events were collected for two years after the first vaccine administration.	6.9% 4/58 • Adverse events were collected for two years after the first vaccine administration.