Detail

Basic Information Clinical Results Relationship Graph

Basic Information

Gene symbol	PMEL	Synonyms	D12S53E, HMB-45, HMB45, ME20, ME20-M, ME20M, P1, P100, PMEL17, SI, SIL, SILV, gp100	Type of gene	protein-coding
Chromosome	12	Map location	12q13.2	dbXrefs
Description	premelanosome protein

Gene symbol	MLANA	Synonyms	MART-1, MART1	Type of gene	protein-coding
Chromosome	9	Map location	9p24.1	dbXrefs
Description	melan-A

GTO ID	GTC3962
Disease	Melanoma
Altered gene	MART-1\|gp100
Therapeutic/Target gene	Target gene
Therapy	TCR-T cell
Treatment	gp100/MAGE-1 TCR-T cells
Recruitment status	Unknown
Title	Lack of specific gamma-retroviral vector long terminal repeat promoter silencing in patients receiving genetically engineered lymphocytes and activation upon lymphocyte restimulation
Company sponsor	National Cancer Institute (NCI)

Vector information

Vector	retrovirus
Vector type	γ-retroviral vector
Transgene/Inserted gene	the α-chain and β-chain of a MART-1-specific TCR

Clinical Result

Cohort 1

Administration route	intravenous infusion
Dosage	40 mg/kg/day continuous on days 1-4 of a 28-day cycle, for two cycles
Pts	35
Outcome	1/35(PR); 21/35(PD); 12/35(SD)
Adverse reactions	1/35(Arthralgia); 1/35(Anemia); 1/35(Upper abdominal pain)
References	PMID: 19589923

Cohort 2

Administration route	intravitreal injection
Dosage	0.3 mg
Pts	133
Outcome	the proportions of subjects with an improvement of >= 10 letters: at week 102 was 38.3% (41/107). Improvement of >=15 letters: from baseline to week 54 were 16.5% (22/133); at week 102 was 23.4%(25/107). The proportion of subjects with a decrease in the degree of retinopathy by >= 2 steps: at week 54 was 10.2%; at week 102 was 16.3%. The proportion of subjects with a >= 25% decrease in retinal thickness: at week 54 was 31.7% (39/123); at week 102 was 40.4%(40/99). A >= 50% decrease in retinal thickness: at week 54 was 14.6% (18/123); at week 102 was 19.2%(19/99).
Adverse reactions	2/144(angina pectoris; coronary artery disease); 1/144(vitreous hemorrhage); 32/144(conjunctival hemorrhage); 16/144(diabetic retinal edema)
References	PMID: 19589923

Cohort 3

Administration route	intravitreal injection
Dosage	0.3 mg
Pts	61
Outcome	The mean change in visual acuity decreased by 10.3 letters for up to 199 weeks (62-199 weeks; mean 140 weeks) during follow-up.
Adverse reactions	12/61(retinal hemorrhage; anterior chamber inflammation; macular degeneration; floaters; photopsia; retinal vessel aneurysm; vitreous hemorrhage; ocular hypertension; arteriosclerosis obliterans; hypertension)
References	PMID: 19589923

Cohort 4

Administration route	intravitreal injection
Dosage	3 mg/100 microL (every 6 weeks for minimum of 6 injections)
Pts	5
Outcome	These two patients had progressive decrease in retinal hard exudate and reduction in central retinal thickness measured by optical coherence tomography. One of these two patients had improvement in visual acuity of 3 lines.
Adverse reactions	1/5(tractional retinal detachment); 3/5(transient postinjection hypotony in two eyes)
References	PMID: 19589923

Cohort 5

Administration route	infusion
Dosage	0.1~8.6E10 cells
Donor type	autologous
Pts	17
Lymph depletion	Yes
Outcome	2/17(PR); 15/17(SD)
Adverse reactions	not mentioned
References	PMID: 19589923

Cohort 6

Administration route	injection
Dosage	0.266~2.66E11 cells
Donor type	Allogeneic
Pts	15
Outcome	1/15(PR)
Adverse reactions	1(septicemia)
References	PMID: 19589923

Cohort1: Eye001

Administration route	intravitreal injection
Dosage	3 mg/100 μl, 3 times at 28-day intervals
Pts	10
Outcome	87.5% of patients had stabilized (<0 lines increase) or improved vision; 60% of patients showed a 3 line improvement of vision on the ETDRS chart at 3 months
Adverse reactions	vitreous floaters; mild anterior chamber inflammation; ocular irritation; increased intraocular pressure; intraocular air; vitreous haze; subconjunctival hemorrhage; eye pain; lid edema/erythema; dry eye; conjunctival injection
References	PMID: 19589923

Cohort2: Eye001_Photodynamic Therapy

Administration route	intravitreal injection
Dosage	3 mg/100 μl, 3 times at 28-day intervals
Pts	11
Outcome	90% of patients had stabilized (<0 lines increase) or improved vision; 60% of patients showed a 3 line improvement of vision on the ETDRS chart at 3 months
Adverse reactions	ptosis; mild anterior chamber inflammation; corneal abrasion; eye pain; foreign body sensation; chemosis; subconjunctival hemorrhage; vitreous prolapse
References	PMID: 19589923

Cohort1: dose level 1

Administration route	intravitreal injection
Dosage	0.3 mg
Pts	295
Age	Adult, Older_Adult
Adverse reactions	eye pain; vitreous floaters; punctate keratitis; cataracts; vitreous opacities; anterior-chamber inflammation; visual disturbance; eye discharge; corneal edema
References	PMID: 19589923

Cohort2: dose level 2

Administration route	intravitreal injection
Dosage	1 mg
Pts	301
Age	Adult, Older_Adult
Adverse reactions	eye pain; vitreous floaters; punctate keratitis; cataracts; vitreous opacities; anterior-chamber inflammation; visual disturbance; eye discharge; corneal edema
References	PMID: 19589923

Cohort3: dose level 3

Administration route	intravitreal injection
Dosage	3 mg
Pts	296
Age	Adult, Older_Adult
Adverse reactions	eye pain; vitreous floaters; punctate keratitis; cataracts; vitreous opacities; anterior-chamber inflammation; visual disturbance; eye discharge; corneal edema
References	PMID: 19589923

Cohort1: dose level 1

Administration route	intravitreal injection
Dosage	0.3 mg
Pts	44
Adverse reactions	6/44(Serious Adverse reactions)
References	PMID: 19589923

Cohort2: dose level 2

Administration route	intravitreal injection
Dosage	1 mg
Pts	44
Adverse reactions	2/44(Serious Adverse reactions)
References	PMID: 19589923

Cohort3: dose level 3

Administration route	intravitreal injection
Dosage	3 mg
Pts	42
Adverse reactions	13/42(Serious Adverse reactions)
References	PMID: 19589923

Cohort 15

Administration route	injection
Dosage	0.266~2.66E11 cells
Donor type	Allogeneic
Pts	15
Outcome	1/15(PR)
Adverse reactions	1(septicemia)
References	PMID: 19589923

Cohort 16

Administration route	intravitreal injection
Dosage	3 mg/100 microL (every 6 weeks for minimum of 6 injections)
Pts	5
Outcome	These two patients had progressive decrease in retinal hard exudate and reduction in central retinal thickness measured by optical coherence tomography. One of these two patients had improvement in visual acuity of 3 lines.
Adverse reactions	1/5(tractional retinal detachment); 3/5(transient postinjection hypotony in two eyes)
References	PMID: 19589923

Cohort1: dose level 1_RB006_RB007

Administration route	intravenous infusion
Dosage	15 mg
Pts	6
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort2: dose level 2_RB006_RB007

Administration route	intravenous infusion
Dosage	30 mg
Pts	6
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort3: dose level 3_RB006_RB007

Administration route	intravenous infusion
Dosage	50 mg
Pts	8
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort4: dose level 4_RB006_RB007

Administration route	intravenous infusion
Dosage	75mg
Pts	8
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort5: dose level 1_RB006_placebo

Administration route	intravenous infusion
Dosage	15 mg
Pts	3
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort6: dose level 2_RB006_placebo

Administration route	intravenous infusion
Dosage	30 mg
Pts	3
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort7: dose level 3_RB006_placebo

Administration route	intravenous infusion
Dosage	50 mg
Pts	4
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort8: dose level 4_RB006_placebo

Administration route	intravenous infusion
Dosage	75mg
Pts	4
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort1: MART-1 TCR-T

Administration route	infusion
Dosage	0.1~8.6E10 cells
Donor type	autologous
Pts	10
Lymph depletion	Yes
Outcome	1/10(CR); 1/10(PR); 8/10(SD)
Adverse reactions	not mentioned
References	PMID: 19589923

Cohort2: gp100 TCR-T

Administration route	infusion
Dosage	0.1~8.6E10 cells
Donor type	autologous
Pts	8
Lymph depletion	Yes
Outcome	8/8(SD)
Adverse reactions	not mentioned
References	PMID: 19589923

Cohort1: melanoma_gp100 TCR-T

Administration route	infusion
Dosage	0.18~11E10 mcells
Donor type	autologous
Pts	18
Lymph depletion	Yes
Outcome	1/18(CAR); 2/18(PR); 16/18(SD)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort2: Cholangiocarcinoma_P53 TCR-T

Administration route	infusion
Dosage	0.8E9 cells
Donor type	autologous
Pts	1
Lymph depletion	Yes
Outcome	1/1(SD)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort3: esophageal cancer_P53 TCR-T

Administration route	infusion
Dosage	1.2E9 mcells
Donor type	autologous
Pts	1
Lymph depletion	Yes
Outcome	1/1(SD)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort4: melanoma_p53 TCR-T

Administration route	infusion
Dosage	2.2~27.7E9 mcells
Donor type	autologous
Pts	2
Lymph depletion	Yes
Outcome	2/2(SD)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort5: breast cancer_P53 TCR-T

Administration route	infusion
Dosage	0.8~9.2E9 mcells
Donor type	autologous
Pts	4
Lymph depletion	Yes
Outcome	4/4(SD)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort6: Salivary gland adenocarcinoma_P53 TCR-T

Administration route	infusion
Dosage	0.5E9 cells
Donor type	autologous
Pts	1
Lymph depletion	Yes
Outcome	1/1(PR)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort7: Small bowel adenocarcinoma_P53 TCR-T

Administration route	infusion
Dosage	3.7E9 mcells
Donor type	autologous
Pts	1
Lymph depletion	Yes
Outcome	1/1(SD)
Adverse reactions	No serious clinical adverse events
References	PMID: 19589923

Cohort 34

Administration route	None
References	PMID: 19589923

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