Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

All Drugs and Chemicals

Micronutrients

Deferasirox

Iron

Chelating Agents

Iron Chelating Agents

All patients are already on commercial deferasirox before entering the study.

Patients experiencing a hematological response and patients not experiencing a hematological response while on deferasirox treatment received a new bone marrow aspirate in order to investigate the presence of differential gene expression between those two groups

Bone marrow aspirate

Patients are already on commercial deferasirox before entering the study.

Several previous studies (clinical and non-clinical) hypothesize that treatment with deferasirox causes a hematological improvement in transfused patients with low and intermediate-1 risk myelodysplastic syndrome. The purpose of this study was to assess the presence of genetic biomarkers predictive for hematologic response by the use of gene expression profiling of bone marrow aspirates obtained from MDS patients with or without hematological response.

This trial was terminated due to low enrollment.

A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy.

A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy in Hematological Centers in Belgium Using Gene Expressing Profiling From Baseline Bone Marrow.

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

Using next-generation sequencing, gene expression profiling in responder and non-responder patients were to be performed on existing bone marrow aspirate samples. Gene transcription were then to be compared between the two groups and the fold increase/decrease in differentially expressed genes were to be calculated.

The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group.

From baseline to time of response (responder group) or time to last follow up (non-responders)

Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the non-responder group.

Novartis Pharmaceuticals

There was no treatment administration specific to this study. The trial was terminated due to low enrollment. The patient did not experience any Adverse Events during the trial.

Age, Customized

Sex/Gender, Customized

Due to low recruitment (1 patient after 12 months of recruitment) the study has been cancelled. Because there was only 1 patient who enrolled, it's best that the baseline characteristics of this patient be kept confidential, respecting the privacy of this patient.

The trial was terminated due to low enrollment.

Study Director

There was no treatment administration specific to this study. The trial was terminated due to low enrollment. Because of the limited number of data collected an efficacy analysis was not possible.

Fold Increase/Decrease in Gene Transcription From Baseline Bone Marrow Aspirate of Responders Versus Non-responders'

Time to Response

Changes in Serum Ferritin Levels

Deferasirox Dose Used

Changes in Serum Transferrin Levels

Changes in Transferrin Saturation Levels

Overall Study

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Prostatic Neoplasms

Genital Neoplasms, Male

Urogenital Neoplasms

Genital Diseases

Genital Diseases, Male

Urogenital Diseases

Prostatic Diseases

Male Urogenital Diseases

Vitamins

Ascorbic Acid

Antioxidants

Trace Elements

Protective Agents

Vitamin C

Each subjects receive 12 weeks of 1 weekly treatment with intravenous vitamin c.

5grams are given at week 1, 30 grams at week 2 and 60 grams at week 3-12. If eligibility criteria are met the subject may continue with 1 weekly vitamin c treatment of 60 grams at week 13-20.

60grams of ascorbate given intravenous infusion in 1000ml sterile water.

Ascorbic Acid (Vitamin C)

Kari J Mikines, MD, DsMC

Can high dose, intravenous Vitamin C prolong life for patients with metastatic prostate cancer?

Prostate cancer is the most common cancer (excluding skin cancer) in men in Denmark and the Unites States. When metastatic disease is present cure is no longer possible. The main treatment at this stage is castration, either surgical or medical, ending the patients testosterone production and causing a temporary regression in disease activity.

Eventually, the cancer will progress, usually within 2 years from the castration, with a more aggressive course and a survival of 2-3 years.

The current treatment option for the patients, who have undergone castration and have disease progression, is chemotherapy with only limited gains in quality of life and survival.

This clinical study is a phase 2 study to evaluate the effects of high dose intravenous vitamin c in subjects with early castration resistant prostate cancer.

Primary endpoint:

* Prostate specific antigen (PSA) changes after 12 to 20 weekly vitamin c infusions

Secondary endpoints:

* Bone metastases changes after 12 to 20 weekly vitamin c infusions
* Changes in bone specific alkaline phosphates, oxidative DNA-damage, PINP, NTX after 12 to 20 weekly vitamin c infusions
* RNA-expression changes in prostatic tumor tissue after 12 to 20 weekly vitamin c infusions
* RNA-expression changes in lymphocytes after 12 to 20 weekly vitamin c infusions

Tertiary endpoints:

* Pharmacokinetics of vitamin c in the elderly cancer patients

Methods and material:

* 80 subjects are included (efficacy evaluation when 20 subjects have been evaluated for extension arm)
* Each subject receives a weekly infusion of 60 grams vitamin c (in the form of ascorbate) for 12 to 20 weeks

Vitamin C for palliative treatment:

Intravenous vitamin C has been used since the 1970's for terminally ill cancer patients claiming big increases in survival time. The efficacy of the drug is questioned and no randomized, controlled trial of Vitamin C's efficacy on cancer patients survival has been made.

Recent results from in vitro and xenograft studies in mice has shown some promise for vitamin c as a cytotoxic agent against cancer cells.

The following parameters are recorded for baseline:

* Biomarkers (PSA, bALP, NTX, PINP)
* Routine blood work (hgb, creatinine, p-vitamin c etc.)
* Radio nucleotide bone scintigraphy
* Prostate biopsies for later microarray (Affymetrix ST1.0)
* Urine samples 8-oxo-guanine(for oxidative DNA-damage measurements)

These parameters are repeated after treatment, usually after 12 to 26 weeks after the first vitamin c infusion.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy.	NCT02663752	Myelodysplastic...	Bone marrow asp... Deferasirox	18 Years -	Novartis
Vitamin C as an Anti-cancer Drug	NCT01080352	Prostatic Neopl...	Ascorbic Acid (...	18 Years -	Copenhagen University Hospital at Herlev

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