Clinical Trials and Research Studies

Clinical trials and research studies, such as non-therapeutic studies and market research studies, help identify new ways to prevent, detect or treat disease and understand patient needs. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, non-therapeutic study, or market research study.

Non-therapeutic Study Non-therapeutic Study: The purpose of a non-therapeutic study is to increase the understanding of a disease or condition through observation, data collection, or other methods. A non-therapeutic study does not provide treatment or intervention for participants. This type of research contributes to general knowledge that may benefit others in the future by improving treatment or developing new therapies. Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US

Status

Enrolling

Study Date (Range)

May 31, 2024 - December 31, 2026

Bone Marrow Disease(s)

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Age Group

18 years and older

This study aims to capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. As a participant, you will track what you're noticing about your health in the Folia Health app once per week for 6…

Non-therapeutic Study Non-therapeutic Study: The purpose of a non-therapeutic study is to increase the understanding of a disease or condition through observation, data collection, or other methods. A non-therapeutic study does not provide treatment or intervention for participants. This type of research contributes to general knowledge that may benefit others in the future by improving treatment or developing new therapies. PLEDGE – Patients’ Long-term Experience on VOYDEYA (Danicopan): Gathering Evidence in the Real-World

Status

Recruiting

Study Date (Range)

May 12, 2025 - August 30, 2026

Bone Marrow Disease(s)

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Age Group

18 years and older

The IRB-approved observational Phase IV PLEDGE study is set-up to collect real-world data on adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) with symptomatic extravascular hemolysis who recently started VOYDEYATM (danicopan) as add-on treatment to ULTOMIRIS®…

Market Research Study Market Research Study: Market research study is conducted to understand the patient population by gathering information from patients about their experience with types of treatment, awareness of therapeutic options, resources for making informed decisions, and health care providers and services. This type of research can provide insights that may inform product development, improve patient care, and identify unmet needs of patients. Understanding the Paroxysmal Nocturnal Hemoglobinuria Journey

Status

Recruiting

Study Date (Range)

September 2, 2025 - September 2, 2026

Bone Marrow Disease(s)

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Age Group

21 years and older

The purpose of this study is to better understand the treatment journey and experiences of people living with Paroxysmal Nocturnal Hemoglobinuria (PNH), specifically those currently in a “watch and wait” period after being diagnosed.

Clinical Trial Clinical Trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease. Trials are in four phases. Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment. CureAA: Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

Status

Recruiting

Study Date (Range)

December 1, 2024 - February 1, 2029

Bone Marrow Disease(s)

Aplastic Anemia

Age Group

3 years to 75 years

This is a Phase II study of hematopoietic stem cell transplantation for previously untreated patients with severe aplastic anemia. This study is comprised of two cohorts - patients receiving haploidentical donor transplant and patients receiving unrelated donor transplant. The…

Clinical Trial Clinical Trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease. Trials are in four phases. Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment. Low Dose Danazol for the Treatment of Telomere Related Diseases

Status

Recruiting

Study Date (Range)

March 1, 2018 - April 30, 2026

Bone Marrow Disease(s)

Aplastic Anemia

Age Group

3 years and older

DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers at the…

Clinical Trial Clinical Trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease. Trials are in four phases. Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment. A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, ATG) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients with Refractory or Recurrent Severe Aplastic Anemia

Status

Recruiting

Study Date (Range)

January 4, 2024 - March 26, 2026

Bone Marrow Disease(s)

Aplastic Anemia

Age Group

40 years to 75 years

The current standard treatment for aplastic anemia (AA) is immunosuppressive therapy (IST), with only temporary symptom relief and high rates of paroxysmal nocturnal hemoglobinuria, clonal hematopoiesis, and myelodysplastic syndrome. When a matched donor is available, allogeneic…

Disclaimer

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any clinical trial, non-therapeutic study, or market research study listed on this website. Pharmaceutical company sponsored content is highlighted only to give additional information about the clinical trial or research study. All clinical trials are listed on https://clinicaltrials.gov/. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a clinical trial, non-therapeutic study, or market research study and never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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