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Search Results Within Category "Blood Disorders "

Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.

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8 Study Matches

MT2019-06: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

The purpose of this study is to evaluate the safety and ability of a transplant with your own gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. The goal is to determine if a sufficient amount of hemoglobin that prevents red blood sickling can be produced after the gene modified stem cells are returned to your body. This study may provide information on the potential usefulness of bb1111 for treatment of sickle cell disease

Ashish Gupta
Not specified
This study is NOT accepting healthy volunteers
STUDY00006923
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Inclusion Criteria:

• must be 2 to 50 years old
• diagnosis of Sickle Cell Disease
• weigh a minimum of 6 kg (13.2 pounds)
• treated and followed for at least the past 24 months
• experienced at least 4 protocol-defined VOEs in the past 24 months
• experienced HU failure at any point in the past or must have intolerance to HU
• female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from starting the study to at least 6 months after drug product infusion.
Exclusion Criteria:

• if allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available
• unable to receive a transfusion
• prior allogeneic transplant or gene therapy
• prior or current malignancy or immunodeficiency disorder, except cured tumors such as squamous cell carcinoma of the skin
• women who are pregnant or breast feeding
• additional exclusion criteria (study staff will review)
Blood Disorders
SCD, Sickle Cell Disease
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MT2022-54 A MULTINATIONAL, MULTICENTER, DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY ACTIVITY OF FP-045 IN PATIENTS WITH FANCONI ANEMIA (FuschiA Study)

The purpose of this research study is to determine the best dose of FP-045 for Fanconi anemia pediatric and adolescent participants. The study will look at whether the participants have any side effects and if there are any possible changes in something called “biomarkers,” which are blood proteins that will be checked to see if they change when taking FP-045 and that may indicate if FP-045 can delay or prevent disease symptoms. Every participant will receive FP-045.

Meera Srikanthan
Not specified
This study is NOT accepting healthy volunteers
SITE00001887
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Inclusion Criteria:

• 3 to 25 years old
• documented Fanconi anemia by chromosome breakage analysis
• women of child-bearing potential and males required to use highly effective birth control
Exclusion Criteria:

• history of any cancer except squamous cell or basal cell carcinoma of the skin or carcinoma in situ of cervix
• myelodysplastic syndrome or acute leukemia
• history of any significant medical conditions
• history of bone marrow or stem cell transplant
• see link to clinicaltrials.gov for complete criteria
Blood Disorders, Rare Diseases
Fanconi Anemia
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MT2022-41 A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises (BEACON Trial) (BEACON)

BEAM-101 is an experimental new therapy being developed for treating people with SCD and vaso-occlusive crises. The goal of this study is to see if BEAM-101 is safe and effective for people in the study. The study sponsor and study doctors would also like to see if individuals who are treated with BEAM-101 require fewer blood transfusions and experience fewer vasoocclusive crises requiring hospitalization, compared to before they received BEAM-101. This study will also measure the levels of fetal hemoglobin along with measures that assess quality of life and ability to function following treatment with BEAM-101.

Ashish Gupta
18 years and over
This study is NOT accepting healthy volunteers
STUDY00017341
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Inclusion Criteria:

• 18 to 35 years old
• documented diagnosis of sickle cell disease with specific genotypes (study staff will review)
• disease is severe
Exclusion Criteria:

• HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
• previous transplant
• history of an overt stroke
Rare Diseases, Blood Disorders
Clinics and Surgery Center (CSC), SCD, Sickle Cell Disease
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MT2017-17:T Cell receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in patients with Inherited Bone Marrow Failure (BMF) Disorders

The purpose of this study is to learn if removing the donor T cells from the donor product using this new method will be a better way to reduce the risk of GVHD. The benefit of removing these cells with this new method is that they will prevent GVHD without requiring drugs to suppress the immune system. Potentially, the immune system will recover from the transplant faster, which in turn will also lessen the risk of severe infections. As well, the patient will not have the other common undesired side effects of these immunosuppressive drugs.

Margaret MacMillan, MD
Not specified
This study is NOT accepting healthy volunteers
STUDY00003182
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Inclusion Criteria:

• up to 65 years of age
• have a diagnosis of Fanconi anemia
• have a suitable donor for peripheral blood cells
• women of childbearing potential and men with partners of child-bearing potential must agree to use of contraception for the duration of treatment and 4 months after the transplant
• see link to clinicaltrials.gov for additional criteria
Exclusion Criteria:

• women who are pregnant or breastfeeding
• cancer within previous 2 years
Blood Disorders, Rare Diseases
Clinics and Surgery Center (CSC), Fanconi Anemia, Myelodysplastic Syndromes, Severe Aplastic Anemia
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A phase 3, randomized, double-blind, study to assess efficacy and safety of ianalumab (VAY736) versus placebo in warm autoimmune hemolytic anemia (wAIHA) patients who failed at least one line of treatment (VAYHIA)

The purpose of the study is to see if ianalumab, compared to placebo, is effective and safe for treating wAIHA. A placebo looks like the study drug, ianalumab, but does not contain any active ingredient. Ianalumab belongs to a class of drugs called monoclonal antibodies. Monoclonal antibodies are molecules that can recognize and stick to a specific protein expressed on the cell surface or released free in the body. Participants will receive study drug (ianalumab or placebo) through the vein every 4 weeks (4 doses in total) during the treatment period.

Marshall Mazepa, MD
18 years and over
This study is NOT accepting healthy volunteers
STUDY00018783
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Inclusion Criteria:

• people with documented primary or secondary wAIHA
• had an insufficient response to or relapsed after one or more treatments
• Hemoglobin concentration at screening between 5 g/dL and 10 g/dL and experiencing symptoms of anemia
• dose of supportive medication must be stable for at least 4 weeks
Exclusion Criteria:

• wAIHA due to disease involving bone marrow
• prior use of B-cell depleting therapy (e.g., rituximab) within 12 weeks prior to starting the study
• active viral, bacterial or other infections that require systemic treatment at time of screening, or a history of recurrent clinically significant infection
• positive for hepatitis C virus, hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb)
• contact study staff for additional criteria
Blood Disorders
hemolytic anemia, wAIHA, warm autoimmune hemolytic anemia
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EFC17574: A Phase 3, single-arm, multicenter, multinational, open label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged >= 12 years with severe hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX (ATLAS-NEO)

A study to test a medicine (fitusiran) injected under the skin for preventing bleeding episodes in male adolescent or adult participants with severe Hemophilia.

Jacob Cogan
Not specified
This study is NOT accepting healthy volunteers
STUDY00017896
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Inclusion Criteria:

• 12 years or older
• diagnosis of severe congenital hemophilia A or B
• participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months
Exclusion Criteria:

• co-existing bleeding disorders other than congenital hemophilia A or B
• current participation in immune tolerance induction therapy (ITI)
• prior treatment with gene therapy
• acute hepatitis, ie, hepatitis A, hepatitis E, acute or chronic hepatitis B infection
• additional exclusion criteria apply (study staff will review)
Blood Disorders
Hemophilia
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Vasomotor symptoms of menopause and cardiovascular disease: What is the link?

Study to examine the physiological responses that occur during a hot flush in postmenopausal women

Manda Keller-Ross
18 years and over
This study is also accepting healthy volunteers
STUDY00013742
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Inclusion Criteria:
Participants must have completed menopause and experience either no menopausal hot flashes or experience three menopausal hot flashes a day.
Exclusion Criteria:

• Reported nicotine/tobacco use within the last six months
• Diabetic or asthmatic
• Diagnosed significant carotid stenosis
• History of significant autonomic dysfunction, heart disease, respiratory disease, or severe neurologic condition such as stroke or traumatic brain injury
• Existing metabolic or endocrine abnormalities
• Use of heart/blood pressure medications that are determined to interfere with study outcomes
• Use of oral contraceptives (or other hormonal contraceptives, including intrauterine devices or contraceptive implants) and/or hormone therapy
• Pregnant or breastfeeding
• Unwilling or unable to refrain from consuming caffeine or alcohol in the 12 hours before visit two and three.
• Unwilling or unable to refrain from vigorous exercise (at least 10 minutes in duration) in the 12 hours before visit two or three
• Unwilling or unable to fast in the eight hours before visit two or three
• Body mass index ? 35 kg/m2
Blood Disorders, Brain & Nervous System, Women's Health
autonomic nervous system, Blood pressure, hot flashes, hypertension, menopause, night sweats
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Pilot of standardized cognitive behavioral therapy for young adults with sickle cell disease

A pilot study looking at an adult-based non-pharmacologic interventional trial in collaboration with psychologist who has expertise in cognitive behavioral therapy (CBT) for adolescents with chronic disease and intellectual disability and developing processes towards independence in non-Sickle Cell Disease settings.

Alexander Boucher
18 years and over
This study is NOT accepting healthy volunteers
STUDY00018692
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Inclusion Criteria:

• 18 to 24 years old
• diagnosis of sickle cell anemia
• transitioning to adult care at UMMC from pediatric care for SCD
Exclusion Criteria:

• unable to speak and write English
• have had more than 2 visits as a new patient in the adult UMMC sickle cell clinic
Blood Disorders, Mental Health & Addiction
SCD, sickle cell, sickle cell anemia, Clinics and Surgery Center (CSC)
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