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45 Anemia Trials near San Francisco, CA
Power is an online platform that helps thousands of Anemia patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerGene Therapy for Fanconi Anemia
Stanford, California
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A).
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1+
Key Eligibility Criteria
Disqualifiers:HLA-identical Donor, Myelodysplastic Syndrome, Leukemia, Others
Must Not Be Taking:Androgens, Investigational Therapy
5 Participants Needed
FP-045 for Fanconi Anemia
Palo Alto, California
This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. The study will enroll a total of 4 young adult/adolescent patients and a minimum of 8 and up to 12 pediatric patients with mild-moderate bone marrow failure who have not undergone hematopoietic cell transplant. This makes the total patient number between 12-16 total. Dose escalation will occur individually for each patient, within each age group. Each patient will receive each of 3 dose levels of FP 045 (intra-patient dose escalation), beginning with Dose Level 1, followed by Dose Levels 2 and 3. Each dose level will be administered for 28 days prior to escalation to the next higher dose level for that patient.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:3 - 35
Key Eligibility Criteria
Disqualifiers:Not Listed
16 Participants Needed
Stem Cell Transplant + JSP191 for Fanconi Anemia
Stanford, California
The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor after using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method).
Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:2+
Key Eligibility Criteria
Disqualifiers:HIV, Active Infections, Malignancies, Others
Must Not Be Taking:Investigational Agents
12 Participants Needed
Bone Marrow Transplant for Aplastic Anemia
Stanford, California
BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:3 - 75
Key Eligibility Criteria
Disqualifiers:Inherited Bone Marrow Failure, MDS, Others
Must Not Be Taking:Immunosuppressive Therapy
60 Participants Needed
Gene Therapy for Pyruvate Kinase Deficiency
Palo Alto, California
This is an open-label Phase II trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 2
Age:8 - 55
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis, Hypertension, Malignancy, Others
10 Participants Needed
Red Cell Transfusions for Premature Infants
Palo Alto, California
The objective of the TOP trial is to determine whether higher hemoglobin thresholds for transfusing ELBW infants resulting in higher hemoglobin levels lead to improvement in the primary outcome of survival and rates of neurodevelopmental impairment (NDI) at 22-26 months of age, using standardized assessments by Bayley.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:< 48
Key Eligibility Criteria
Disqualifiers:Congenital Heart Disease, Anemia, Others
Must Not Be Taking:Erythropoietin
1824 Participants Needed
Donor Matching Strategies for Blood Cancer Transplants
Stanford, California
The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
1753 Participants Needed
Ruxolitinib for Eosinophilia
Palo Alto, California
This phase II trial studies how well ruxolitinib works in treating patients with hypereosinophilic syndrome or primary eosinophilic disorders.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Life-threatening Complications, Myeloid Neoplasm, Reactive Hypereosinophilia, Invasive Malignancy, Others
Must Not Be Taking:Interferon, Imatinib, Antibodies, Hydroxyurea
10 Participants Needed
Gene Modified T-cells for Blood Disorders
Palo Alto, California
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:1 - 26
Key Eligibility Criteria
Disqualifiers:Severe Cardiovascular, Active Infections, Others
Must Not Be Taking:Immunosuppressants
120 Participants Needed
Gene Therapy for Pyruvate Kinase Deficiency
Stanford, California
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:8 - 50
Key Eligibility Criteria
Disqualifiers:Not Listed
5 Participants Needed
CTX001 for Sickle Cell Disease
Palo Alto, California
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:12 - 35
Key Eligibility Criteria
Disqualifiers:Prior HSCT, Active Infection, Others
63 Participants Needed
Gene Therapy for Sickle Cell Disease
Palo Alto, California
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12 - 40
Key Eligibility Criteria
Disqualifiers:Malignancy, Immunodeficiency, Infection, Pregnancy, Others
15 Participants Needed
Cyclophosphamide-Based Prophylaxis for Leukemia
Palo Alto, California
The purpose of this clinical trial is to compare drug combinations to learn which drugs work best to prevent graft-versus-host-disease (GVHD) in people who have received a stem cell transplant. The source of stem cells is from someone who is not related and has a different blood cell type than the study participant. The researchers will compare the new drug combination to a standard drug combination. They will also learn about the safety of each drug combination.
Participants will:
* Receive the standard or new drug combination after transplant
* Visit the doctor's office for check-ups and tests after transplant that are routine for most transplant patients
* Take surveys about physical and emotional well-being
* Give blood and stool samples.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Prior Transplant, Pregnancy, Infections, Others
358 Participants Needed
BEAM-101 for Sickle Cell Disease
Palo Alto, California
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 35
Key Eligibility Criteria
Disqualifiers:Moyamoya Syndrome, Stroke, Others
Must Be Taking:Hydroxyurea
15 Participants Needed
AG-348 for Pyruvate Kinase Deficiency
Palo Alto, California
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
52 Participants Needed
Why Other Patients Applied
"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."
HZ
Arthritis PatientAge: 78
"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."
WR
Obesity PatientAge: 58
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
AG
Paralysis PatientAge: 50
"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."
ZS
Depression PatientAge: 51
"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."
IZ
Healthy Volunteer PatientAge: 38
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