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A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders Clinical Trials Information presented on Clinical Trials Search is not intended to be a substitute for qualified health advice, trips or treatment by using a genuine doctor. We aren't doctors. Always consult your mD on A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders conditions. Clinical Trials Search.org is a site committed to listing clinical research studies in human subjects. A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders Clinical research trials and A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders health trials take place in a lot of of cities across the US. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally measure the potency of new drugs. The aim of the studies / projects is to answer specific human medical questions. Clinical trials are a popular manner for physicians, government agencies, and private sector corporations to discover remedies for all kinds of circumstances, like A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders. A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders Clinical Trials and other clinical trials allow for volunteers to have health treatment alternatives before they are available to the general public. Many times the test subjects obtain treatment for without cost, and occasionally they are paid for their time. Sometimes there is a cost for a A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders clinical trial. Subjects oftentimes recieve the most effective healthcare possible for their A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders condition. Hazards are a reality, however, and could include additional or frequent doctor visits, healthcare dangers (perhaps life-threatening), and/or the treatment being ineffective. Trials are federally governed with exacting guidelines to protect clinical trials subjects.
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Home > "A" Clinical Trials Conditions > A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders  A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders
For Condition: hemoglobin SC disease,sickle cell anemia
Status: Completed
Sponsor(s): National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) ,
Synopsis: Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation when deoxygenated. The polymerized hemoglobin leads to impaired deformability and sickling of red blood cells which subsequently lodge in end-arterioles producing the classic and most prominent feature of the disorder, repeated vasoocclusive crises. Despite knowledge of the precise genetic defect for decades, only recently has there been therapeutic impact based upon this knowledge when a clear benefit from treatment with hydroxyurea, a cell cycle-specific agent administered to induce production of fetal hemoglobin (HbF) by stimulating gamma-globin synthesis, was reported in patients with sickle cell disease (SCD). The reduction in the frequency and severity of vasoocclusive crises seen has been attributed to the increase in HbF levels in responsive patients. While the majority of patients demonstrate a rise in HbF, not all such patients benefit from treatment. Given these results, alternative agents that also stimulate the production of HbF warrant investigation in the treatment of SCD. Recombinant-methionyl human stem cell factor (SCF) is a hematopoietic growth factor with activity on immature hematopoietic progenitor cells. SCF stimulates the production of HbF in vitro and in vivo, and this effect is attainable without the myelosuppression associated with hydroxyurea. In this phase I/II trial, we will administer SCF in a dose escalating fashion to patients with sickling disorders. Parameters to be measured are HbF levels, F cell levels, peripheral blood CD34 levels, frequency, duration, and severity of vasoocclusive crises, and toxicity.
Details: Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation when deoxygenated. The polymerized hemoglobin leads to impaired deformability and sickling of red blood cells which subsequently lodge in end-arterioles producing the classic and most prominent feature of the disorder, repeated vasoocclusive crises. Despite knowledge of the precise genetic defect for decades, only recently has there been therapeutic impact based upon this knowledge when a clear benefit from treatment with hydroxyurea, a cell cycle-specific agent administered to induce production of fetal hemoglobin (HbF) by stimulating gamma-globin synthesis, was reported in patients with sickle cell disease (SCD). The reduction in the frequency and severity of vasoocclusive crises seen has been attributed to the increase in HbF levels in responsive patients. While the majority of patients demonstrate a rise in HbF, not all such patients benefit from treatment. Given these results, alternative agents that also stimulate the production of HbF warrant investigation in the treatment of SCD. Recombinant-methionyl human stem cell factor (SCF) is a hematopoietic growth factor with activity on immature hematopoietic progenitor cells. SCF stimulates the production of HbF in vitro and in vivo, and this effect is attainable without the myelosuppression associated with hydroxyurea. In this phase I/II trial, we will administer SCF in a dose escalating fashion to patients with sickling disorders. Parameters to be measured are HbF levels, F cell levels, peripheral blood CD34 levels, frequency, duration, and severity of vasoocclusive crises, and toxicity.
Eligibility:
Study Type: Interventional, Treatment, Safety
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: Patients with Hb SS, Sbeta-thal, SD, or SO-Arab Age greater than or equal to 18 years. Patient must have had a previous neurologic event (either symptomatic or found by imaging alone). More than one painful crises per year for the last 2 years, each requiring hospitalization. A previous acute chest syndrome. Evidence of renal damage but with a creatinine clearance of greater than 50 percent of normal. Red cell alloimmunization. Bilateral retinopathy. Osteonecrosis of multiple bones. Unilateral or bilateral leg ulcers. Patients who have failed a course of hydroxyurea or who have declined to take hydroxyurea. Able to give informed consent. No active sickle cell crises or acute chest syndrome. No active uncontrolled infection. No hydroxyurea, erythropoietin, and/or arginine butyrate therapy in the previous month. No patients receiving hypertransfusion therapy. No current treatment (or within 2 weeks) with hematopoietic growth factors. No allergy to E. coli derived products. No history of seasonal or recurrent asthma within the 5 preceding years. No asthmatic symptoms (e.g. wheezing) related to a current respiratory tract infection. No other significant IgE-mediated hypersensitivities (including but not limited to allergic rhinitis, allergic eczema, anaphylactic reactions, congenital or acquired angioedema, and urticaria,). An isolated episode of urticaria occurring within the 5 years is not a contraindication. Patients with drug allergies manifested solely by rash are not excluded. No concurrent use of beta-adrenergic blocking agents. No concurrent use of monoamine oxidase inhibitors. No significant comorbid conditions including uncontrolled hypertension, congestive heart failure(greater NY class II), poorly controlled diabetes mellitus, and significant coronary artery disease with recent myocardial infarction or angioplasty (within the previous 6 months). No pregnancy, breast feeding, and unwillingness to use contraception. No concurrent use of other investigational products.
Total Enrollment: 50
Location and Contact Information:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Bethesda, Maryland, 20892
United States
Additional Information:
Study ID Numbers: 000087; 00-DK-0087
Study Start Date: March 4, 2000
Record last reviewed: February 9, 2000
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00005783
Other Sickle Cell Anemia Studies:
1. A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders
2. The Effect of Oral Magnesium Pidolate on How Often Painful Crises Happens in Patients with Hemoglobin SC Disease
3. Stem Cell Transplantation after Reduced-Dose Chemotherapy for Patients with Sickle Cell Disease or Thalassemia
Related Studies:
Other sickle cell anemia Clinical Trials
Other Maryland Clinical Trials
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A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients with Sickling Disorders
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