Hereditary disorder of connective tissue

This early study tested a new cell therapy called LUCAR-G79 in just 3 adults with severe autoimmune diseases that did not respond to standard treatments. The therapy uses specially engineered immune cells to target and potentially calm the overactive immune system. The main goals…

This study tests whether an additional dose of a COVID-19 vaccine can improve immune protection in adults and children with autoimmune diseases like rheumatoid arthritis, lupus, multiple sclerosis, and others who are taking immunosuppressive medications. Participants who had a we…

This phase 3 trial tested whether adding asciminib to the standard drug imatinib could help people with chronic myeloid leukemia (CML) achieve a deep remission and safely stop all medication. Only 8 patients were enrolled before the study was terminated early, making it too small…

This study looked at using umbilical cord blood transplants after strong chemotherapy and radiation to treat various blood cancers like leukemia and lymphoma. It involved 200 participants and aimed to see how many were alive one year after transplant. The trial was stopped early,…

This study tested a new drug called Vodobatinib (K0706) in people with chronic myeloid leukemia or a related leukemia who had not responded to at least three other treatments. The trial was split into parts: first in healthy volunteers to check safety, then in patients to find th…

This phase 2 trial tested two different drug combinations to prevent graft-versus-host disease (GVHD) in 150 people with blood cancers receiving a stem cell transplant from an unrelated donor. Both combinations used cyclosporine and sirolimus, plus either mycophenolate mofetil or…

This early-phase trial tested a combination of two drugs—ruxolitinib and CPX-351—in 12 people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The goal was to find the best dose and see if the combo could shrink cancer. However, the study was stop…

This study tested a drug called XmAb23104 in people with advanced sarcoma, a rare cancer. The goal was to see if the drug could shrink tumors and if it was safe. Only 6 people took part before the study was ended early.

This study tested a drug called TPN-101 (censavudine) in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The trial enrolled only 4 participants and aimed to see if the drug could reduce immune system overactivity and check fo…

This study tested a drug called PRV-3279 to see if it could prevent lupus flares in people with active systemic lupus erythematosus (SLE). The trial included 28 adults with moderate to severe lupus who had improved after steroid treatment. The study was terminated early, so resul…

This study tested two different drug combinations given before a stem cell transplant for people with myeloid blood cancers like leukemia. One group got fludarabine and busulfan, the other got those drugs plus low-dose total body irradiation. The goal was to see which regimen led…

This study tested an experimental drug called PF-06823859 for adults with lupus that affects the skin. The goal was to see if the drug could reduce skin inflammation and improve symptoms. The trial was stopped early and only enrolled 8 people instead of the planned 48, so results…

This phase II trial tested adding asciminib (ABL001) to standard tyrosine kinase inhibitor therapy in 7 people with chronic myeloid leukemia who still had traces of cancer. The goal was to see if the combination could eliminate remaining cancer cells. The study was terminated ear…

This early-phase study tested two drugs, AZD1775 and belinostat, together in 20 adults with blood cancers like acute myeloid leukemia that had come back or not responded to treatment. The main goal was to find the safest dose and understand side effects. The study was stopped ear…

This early-phase trial tested a single infusion of IMPT-514, a personalized CAR T cell therapy, in 3 adults with severe lupus, vasculitis, or inflammatory myopathy that did not respond to standard treatments. The goal was to check safety and see if the therapy could reset the imm…

This study tested a weekly injection called telitacicept against a placebo in 91 people with moderate-to-severe lupus. The goal was to see if the drug could reduce disease activity and allow lower steroid doses. However, the trial was terminated early, so we have less information…

This study tested the drug ponatinib in 51 adults with a fast-growing form of chronic myeloid leukemia (CML) who had little or no prior treatment. The goal was to see if ponatinib could control the disease by blocking a protein that drives leukemia growth. However, the drug can c…

This study tested a drug called zetomipzomib for people with active lupus nephritis, a kidney disease caused by lupus. The goal was to see if the drug could improve kidney function better than a placebo over 52 weeks. The trial was terminated early, so results are limited. Partic…

This study tested whether adding rapamycin to high-dose chemotherapy could help people with aggressive blood cancers (like certain leukemias and lymphomas) that had returned or not responded to prior treatment. Only 4 adults aged 18–65 took part before the study was stopped early…

This study tested a drug called ruxolitinib in 73 people with two types of blood cancer—polycythemia vera or essential thrombocythemia—that were not helped by standard treatment. The goal was to see if the drug could safely reduce high blood cell counts and symptoms like an enlar…

This phase 3 trial tested whether adding parsaclisib to the standard drug ruxolitinib could better shrink the spleen and ease symptoms in people with myelofibrosis. The study enrolled 252 participants but was terminated early, so the full results are not available. The goal was t…

This study looked at a new way to prevent severe graft-versus-host disease (GVHD) in children who had a bone marrow transplant for blood cancers. The goal was to see if stopping certain immune-suppressing drugs after day 60 could lower the risk of severe GVHD. Only 3 children too…

This study tested a targeted cancer drug called ponatinib combined with standard chemotherapy in children and young adults up to age 21 with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) that had come back or stopped responding to other treatments. The g…

This phase 2 trial tested the drugs mycophenolate mofetil (CellCept) alone or with voclosporin in 12 people with lupus. The goal was to see if these treatments work better in certain patient groups. However, the study was terminated early, so the results are very limited.

This pilot study tested the blood pressure drug telmisartan (Micardis) to prevent acute graft-versus-host disease (GVHD) in people receiving a stem cell transplant for blood cancers. The trial aimed to enroll 60 participants but was terminated early with 32. Researchers tracked s…

This study looked at whether using a lower fluid pressure (60 mmHg) during hysteroscopy, a procedure to remove fibroids, works as well as the standard pressure (80 mmHg). The goal was to see if it improves surgery time, fluid use, and surgeon's view. The study was stopped early a…

This study looked at whether two simple blood test results—hemoglobin and red blood cell distribution width—could predict if a person with polycythemia vera would stop responding to the drug hydroxyurea. Researchers planned to follow 76 adults starting hydroxyurea for the first t…

This study aimed to help children aged 6-8 from low-income families learn how to avoid foods they are allergic to. The program used fun, hands-on activities to teach safety skills. The study was stopped early, so we don't have clear results on whether it worked.

This study looked at how moderate or severe liver problems change the amount of the cancer drug oral azacitidine in the blood. It was designed for adults with certain blood cancers. The study was stopped early after only 2 people enrolled, so the results are very limited.

This study aimed to understand how the COVID-19 vaccine impacts the immune system in people with lupus, a chronic autoimmune disease. Researchers planned to enroll lupus patients to measure their immune response to the vaccine and see if lupus disease activity or treatments affec…

This study looked at a specific hip implant shell (the Pinnacle Acetabular Shell) used in total hip replacement. Researchers wanted to see how much the implant moved (migrated) over two years using a special X-ray technique called RSA. They also tracked how patients felt and func…

This study tested a new questionnaire called Prolac-10 to see if it can accurately measure quality of life in people with prolactinoma (a type of pituitary tumor) who are starting medical treatment. Only 8 people took part before the study was stopped early. The goal was to check…

This registry aimed to track pregnancy outcomes in women who were exposed to elagolix (brand names Orilissa and Oriahnn) for endometriosis or uterine fibroids. The study planned to enroll 584 pregnant women, half with elagolix exposure and half without, and follow them through pr…

This study looked at how well the COVID-19 vaccine triggers an immune response in people with autoimmune or inflammatory diseases who take immunosuppressants or biologics. Researchers took blood samples before vaccination and at several points after to measure antibodies and T-ce…