Neoplasms

Blood and Lymph Conditions

All Conditions

Immune System Diseases

Behaviors and Mental Disorders

Nervous System Diseases

Skin and Connective Tissue Diseases

Rare Diseases

Leukemia

Mastocytosis

Aggression

Mastocytosis, Systemic

Leukemia, Mast-Cell

Neoplasms by Histologic Type

Hematologic Diseases

Dementia

Aberrant Motor Behavior in Dementia

Behavioral Symptoms

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Mast Cell Activation Disorders

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Systemic Mastocytosis

Acute Graft Versus Host Disease

All Drugs and Chemicals

Antineoplastic Agents

Amino Acids

Tyrosine Kinase Inhibitors

Midostaurin

Protein Kinase Inhibitors

Enzyme Inhibitors

Tyrosine

Midostaurin was administered at a dose of 100 mg twice daily (bid) in continuous cycles of 28 days until disease progression, intolerable toxicity or withdrawal due to any cause, whichever occurred first.

Midostaurin was provided as 25 mg soft gelatin capsules for oral administration.

Midostaurin (PKC412)

Novartis Pharmaceuticals

The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated Hematological clonal Non-Mast cell lineage Disease (AHNMD).

Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia

A Single Arm, Phase II, Open-Label Study to Determine the Efficacy of 100mg Twice Daily Oral Dosing of Midostaurin Administered to Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Overall Response Rate (ORR) was defined as the percentage of participants who classified as confirmed responders (Major Response (MR) or Partial Response (PR)) by the adjudication of the SSC and based on a Modified Valent Criteria.

A major responder had complete resolution of at least one C-Finding and no progression in other C-Findings. A partial responder showed a measurable improvement in one or more C-Finding(s) without confirmed progression in other C-Findings. A C-Finding was a Clinical Finding, which was considered by the investigator and corroborated by the Study Steering Committee (SSC) Chairperson or designee, attributable to the mast cell disease component and not the associated hematological clonal non-mast cell lineage disease (AHNMD) component or any other cause.

The Duration of response (DoR) was defined as the time from first onset of confirmed response (MR or PR) to the date of first documented and confirmed progression or death due to ASM/MCL.

The Time to response (TTR) was defined as the time from start of treatment until the date of onset of confirmed response (MR or PR).

The Progression-free survival (PFS) is defined as the time from start of treatment to the date of the first documented and confirmed progression or death due to any cause.

The Overall Survival (OS) is defined as the time from start of treatment to the date of death due to any cause.

Analysis of frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC)

Histopathologic response was summarized to demonstrate the change from baseline in percentage of mast cell infiltrations in the Bone Marrow (BM) and related serum tryptase levels.

Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events fields "number of deaths resulting from adverse events" all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

Age Continuous (PEP)

PEP (n=89) - Female

PEP (n=89) - Male

Sex: Female, Male (PEP)

PEP (n=89) - Black

PEP (n=89) - White

PEP (n=89) - Other/Missing

Race (PEP)

The Participant Flow was on the Full Analysis Set (FAS), the Baseline Characteristics were done on the FAS and Primary Efficacy Population (PEP). The Efficacy analysis was done on the PEP (except for the Overall Survival which was done on FAS and PEP). The Safety analysis was done on the Safety Set (SS).

Study Director

Primary Efficacy Population: participants assigned to study treatment who met diagnostic criteria for aggressive systemic mastocytosis or mast cell leukemia and presented with at least 1 measurable C-Finding at study entry and/or participants with transfusion dependent anemia due to their underlying disease at study entry as confirmed by the SCC.

Percentage of Participants With Overall Response Rate (ORR)

Primary Efficacy Population (PEP), which consisted of all participants with a confirmed response, was considered.

Median Time to Duration of Response (DoR)

Median Time to Response (TTR)

Median Time to Progression-Free Survival (PFS)

Primary Efficacy Population (PEP) (n=89)

Full Analysis Set (FAS) (n=116)

Primary Efficacy Population (PEP), which consisted of all participants with a confirmed response and the Full Analysis Set (FAS), which consisted of all patients who received at least one dose of study drug were considered.

Median Time to Overall Survival (OS)

AEs by Primary System Organ Class (SOC) (n=116)

SAEs by Primary System Organ Class (SOC) (n=88)

Deaths by Primary System Organ Class (SOC) (n=25)

Long-term Safety and Tolerability of Midostaurin

>50% decrease in mast cell (MC) (n=41)

>0-<=50% decrease in mast cell (MC) (n=19)

>50% decrease in serum tryptase (n=52)

>0-<=50% decrease in serum tryptase (n=25)

Primary Efficacy Population (PEP); for each patient, the best improvement relative to Baseline was considered.

Histopathologic Response

Overall Study

masitinib (AB1010)

Olivier Lortholary, MD, PhD

This is a 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap.

Efficacy will be assessed based on:

Pruritus score Number of flush per day Pollakyuria (on a daily basis) Number of stools per day QLQ-C30 score Hamilton Rating Scale for depression

Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

Phase IIa, Open-label, Randomized Study of Oral AB1010 in Patients With Systemic Indolent Mastocytosis With Handicap and Not Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)

AB Science

Melanoma

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms, Nerve Tissue

Nevus, Pigmented

Nevus

Nevi and Melanomas

Skin Neoplasms

Skin Diseases

Neuroendocrine Tumor

Neuroepithelioma

Vaccines

Intralesional injection for chemoablation

PV-10 (10% rose bengal disodium)

John F Thompson, MD

The primary objective of this study is to investigate the effectiveness of intralesional (IL) PV-10 for locoregional treatment of metastatic melanoma. This study will also include assessment of response in untreated bystander lesions following intralesional injection of PV-10 into targeted lesions. Additional objectives are to determine the safety profile of PV-10 following intralesional injection, and assess the pharmacokinetic profile of PV-10 in the bloodstream following intralesional injection.

This is a multicenter, open-label, single-agent study. Subjects with at least one melanoma lesion ≥ 0.2 cm in diameter that can be accurately measured by ruler/caliper or ultrasound will receive intralesional injection of PV-10 into each of up to twenty (20) Study Lesions. Additionally, one to two measurable Bystander Lesions may remain untreated and will be followed for assessment of bystander response.

To accurately reflect anticipated clinical use, repeat dosing of treated lesions will be allowed at the Investigator's discretion at weeks 8, 12 and 16 following initial treatment for those lesions not exhibiting complete response. Subjects will be followed for 52 weeks following initial treatment with PV-10.

Phase 2 Study of Intralesional PV-10 for Metastatic Melanoma

A Phase 2 Study of Intralesional PV-10 in the Treatment of Metastatic Melanoma

Using modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for cutaneous or subcutaneous target lesions assessed by ruler, caliper or ultrasound: Complete Response (CR), disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Objective Response Rate (ORR) = %CR + %PR.

Using modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for designated, untreated cutaneous or subcutaneous bystander lesions assessed by ruler, caliper or ultrasound: Complete Response (CR), disappearance of all bystander lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of bystander lesions; Objective Response Rate (ORR) = %CR + %PR.

Progression is defined using modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or significant worsening of non-target disease (e.g., a measurable increase in non-target lesions or the appearance of new lesions) indicative of disease progression.

Provectus Pharmaceuticals

PV-10 (10% rose bengal disodium): Intralesional injection for chemoablation.

PV-10

United States

Australia

Region of Enrollment

IIIB

IIIC

Participants were staged clinically at baseline based on cutaneous and subcutaneous, nodal and visceral tumor burden. Stage III participants had disease confined to cutaneous, subcutaneous and/or nodal sites. Stage IV participants had distant metastatic disease (i.e., distant cutaneous or subcutaneous metastases or visceral metastases).

American Joint Committee on Cancer (AJCC) Stage at Baseline

Less than 10 Lesions

10 or more Lesions

Too Numerous to Count

Tumor Burden in Skin

Excision

Nodal Biopsy

Regional Chemotherapy

Immunotherapy

Radiotherapy

Investigational Agents

Systemic Chemotherapy

Distal Amputation

Other

Participants may have had more than one prior intervention.

Prior Interventions

Number of Prior Interventions

Participants were staged clinically at baseline using the ECOG Performance Status scale:

GRADE 0: Fully active, able to carry on all pre-disease performance without restriction. GRADE 1: Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work. GRADEe 2: Ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours.

Eastern Cooperative Oncology Group (ECOG) Performance Status

Untreated Visceral Disease

One or More Lesions Not Measured and Untreated

All Lesions Treated Except Bystanders

All Lesions Treated

Participants were categorized at baseline according to tumor burden that was not injected with PV-10.

Untreated Disease Burden

Eric Wachter, Chief Technology Officer

Objective Response Rate (ORR) of PV-10 Treated Lesions

ITT participants having at least one uninjected dermal bystander lesion designated at baseline (some ITT participants did not have at least one bystander lesion).

Objective Response Rate of Untreated Bystander Lesions

Participants reporting Stage III disease at baseline

Stage III

Participants reporting Stage IV disease at baseline

Stage IV

Progression Free Survival (PFS)

Using modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for cutaneous or subcutaneous target lesions assessed by ruler, caliper or ultrasound: Complete Response (CR), disappearance of all target lesions; Complete Response Rate (CRR) = %CR.

ITT participants having all baseline disease injected with PV-10

Rate of Complete Response

Overall Survival

PV-10 (10% rose bengal disodium): Intralesional injection for chemoablation. In the treatment phase, participants received a single IL injection of PV-10 into each of up to 20 study lesions on day 0 (i.e., one cycle). Treatment cycles could be repeated at weeks 8, 12 and 16 for new non-target lesions or existing target or non-target lesions not exhibiting complete response (i.e., complete disappearance). Participants were observed for 52 weeks. Radiologic assessments of visceral disease status were performed every 12 weeks throughout the study and patients were transitioned into survival follow-up if at any time the investigator identified clinical or radiologic evidence of distant progression. No other melanoma therapy was permitted during the study interval.

Treatment Phase

Observation Phase

Eye Diseases

Diseases and Abnormalities at or Before Birth

Retinoblastoma

Neoplasms, Neuroepithelial

Neoplasms, Glandular and Epithelial

Retinal Neoplasms

Eye Neoplasms

Eye Diseases, Hereditary

Retinal Diseases

Carboplatin

Etoposide

Vincristine

Melphalan

Etoposide phosphate

Tubulin Modulators

Antimitotic Agents

Antineoplastic Agents, Alkylating

Alkylating Agents

Immunosuppressive Agents

Immunologic Factors

Patients will receive alternating treatments beginning with systemic chemotherapy then followed by intra-arterial (IA) therapy.

Bilateral retinoblastoma patients will be in Cohort 1.

For bilateral Bilateral retinoblastoma patients where one eye is stage A or B and the other eye is C, D, or E, only the higher stage eye (C, D, E) will be treated with IA chemotherapy unless the stage A or B eye is not amenable or has failed local therapy.

Patients will receive only intra-arterial (IA) therapy for more limited disease.

Anuradha Banerjee, MD

The purpose of this study is to test the safety of the treatment combination of alternating standard chemotherapy and another (melphalan) chemotherapy at different interval schedules. Researchers want to find out what effects, good and/or bad, the treatment combination has on the patients and their retinoblastoma.

Alternating Systemic Chemotherapy and Intra-Arterial Melphalan (IAM) Chemotherapy in Children With Intra-Ocular Retinoblastoma

A Pilot Study Evaluating the Safety of Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Newly Diagnosed Advanced Intra-Ocular Retinoblastoma

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia	NCT00782067	Leukemia	Midostaurin (PK...	18 Years -	Novartis
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis	NCT00831974	Mastocytosis	masitinib (AB10... masitinib (AB10...	18 Years -	AB Science
Phase 2 Study of Intralesional PV-10 for Metastatic Melanoma	NCT00521053	Melanoma	PV-10 (10% rose...	18 Years -	Provectus Pharmaceuticals
Alternating Systemic Chemotherapy and Intra-Arterial Melphalan (IAM) Chemotherapy in Children With Intra-Ocular Retinoblastoma	NCT02116959	Advanced Intra-... Retinoblastoma	Melphalan Carboplatin Etoposide Vincristine	3 Months - 18 Years	University of California, San Francisco

Spots Global Cancer Trial Database for systemic

Every month we try and update this database with for systemic cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for systemic

Every month we try and update this database with for systemic cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for systemic cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.