Pivotal Trial

No Placebo-Only Group

The purpose of this Phase III study is to evaluate the efficacy and safety of 3APS compared to placebo (inactive substance pill) in patients with mild to moderate Alzheimer's disease.

Evaluation of 3APS in Patients With Mild to Moderate Alzheimer's Disease

3APS for Alzheimer's Disease

This study is evaluating whether a drug called 3APS is safe and effective for people with Alzheimer's disease.

I have been diagnosed with Alzheimer's disease.

I am over 50, and if female, cannot have children.

I live at home or in assisted living with a caregiver who can help me.

I do not have any uncontrolled or significant medical conditions.

I have been on a stable dose of Alzheimer's medication for at least 4 months.

I can speak and write in English, French, or Spanish.

3APS

This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.

EPI-743 for Mitochondrial Respiratory Chain Diseases

EPI-743 for Mitochondrial Disease

This study is evaluating whether a drug called EPI-743 can improve the health of people with mitochondrial respiratory chain diseases.

I have a history of bleeding or abnormal blood clotting times not due to blood thinners.

I have been diagnosed with mitochondrial disease without genetic proof.

I have severe heart failure causing poor blood flow to organs and high lactic acid.

I have a genetic diagnosis of mitochondrial disease.

My liver tests are more than twice the normal levels.

I have a condition that prevents my body from absorbing drugs properly.

My blood's hematocrit levels are within the normal range for my age.

I do not take any pill-form dietary supplements or non-prescribed medications.

EPI-743

The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years).

LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis

Combination Therapy for Langerhans Cell Histiocytosis

This study is evaluating whether a new treatment for Langerhans Cell Histiocytosis is safe and effective.

I do not have lung failure, liver issues, or lung fibrosis due to LCH. I am not pregnant, breastfeeding, and I can sign consent.

My diagnosis of Langerhans cell histiocytosis is confirmed by tissue examination.

I have Langerhans Cell Histiocytosis with specific brain or bone involvement.

My diagnosis of Langerhans cell histiocytosis is confirmed by tissue analysis.

My non-risk organs are at least intermediate AD or my risk organs are better AD after 12 weeks.

I have newly diagnosed single-system Langerhans Cell Histiocytosis not in my bones, brain, or 'CNS-risk' areas.

My disease worsened after specific treatments and I have severe organ issues.

I have LCH with specific brain MRI findings, regardless of my past treatments.

All patients from a specific program (LCH IV) who have not refused to be followed up for a long time.

My LCH is worsening in critical organs but shows no active signs, and I or my guardian haven't consented in writing.

My condition worsened in certain organs after treatment, and I now have severe organ dysfunction.

My cancer has worsened in certain areas after initial treatment.

I have a specific liver condition without liver disease and poor kidney function.

This is a specific group of people being targeted for the study.

I am pregnant or have long-term effects from LCH without active disease, and have had prior systemic therapy.

My condition has worsened in areas of my body not considered at high risk.

This is a specific group within the study.

2-chlorodeoxyadenosine

Cytosine Arabinoside

hematopoietic stem cell transplantation (RIC-HSCT)

INDOMETHACIN

Intravenous immunoglobulin

Mercaptopurine

Methotrexate

Prednisone

Vinblastine

This study is a single-center, prospective randomized clinical study intended to compare and evaluate the efficacy of subjects who are treated with bone marrow aspirate concentrate (BMAC) and allograft to iliac crest bone grafts (ICBG) during posterior lumbar/lumbosacral spinal fusion.

Prospective Study of Thoracolumbar Spinal Fusion Graft

Spinal Fusion Graft Techniques for Degenerative Disc Disease

This study is evaluating whether bone marrow aspirate concentrate (BMAC) is as effective as iliac crest bone graft (ICBG) during spinal fusion.

I have a bone condition like Paget's disease or osteomalacia.

I am not taking medications like chronic steroids that affect bone healing.

My back pain affects my daily life significantly.

I am scheduled for a specific back surgery involving the middle to lower spine.

My spine condition is severe, with one vertebra significantly slipping over another.

I have tried non-surgical treatments for 6 weeks without improvement.

BMAC & Allograft

Iliac Crest Bone Graft

The purpose of this study is to evaluate the safety and effectiveness of the JOURNEY™ II CR Total Knee System in subjects with degenerative joint disease (DJD) requiring primary total knee replacement.

JOURNEY™ II CR Total Knee System

Total Knee Replacement for Osteoarthritis

This study is evaluating whether a new knee replacement system is safe and effective for people with knee problems.

I am a candidate for knee replacement due to arthritis.

I am between 22 and 75 years old with knee joint disease.

I can walk on my own and follow all after-surgery check-up instructions.

I have issues with my other knee that might affect my study participation.

I have hip issues such as needing or having had hip surgery, arthritis, or a hip replacement that's not fully healed.

My knee condition doesn't need a cap replacement, and I've had knee surgery or lack enough bone for the device.

I do not have an immune disorder, cancer, severe bone loss in family, allergies to the study device, or conditions affecting the study's outcome.

The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

Casimersen + Golodirsen for Duchenne Muscular Dystrophy

This study is evaluating whether a drug may help treat Duchenne muscular dystrophy.

I have been on a stable dose of oral corticosteroids for at least 24 weeks.

My lung function is at least half of what is expected for someone my age and size.

I have been treated with SMT C1100 recently and PRO045, PRO053, or PRO051 in the last 6 months.

I have not had major surgery in the last 3 months.

I can walk between 300 to 450 meters in 6 minutes.

Both of my biceps or two other upper body muscles are healthy.

My DMD is treatable with exon 45 or 53 skipping.

Placebo

SRP-4045

SRP-4053

The purpose of this study was to assess efficacy, including inhibition of radiographic progression, and safety with upadacitinib versus placebo and versus an active comparator, adalimumab, in adults with with moderately to severely active rheumatoid arthritis (RA) who are on a stable background of methotrexate (MTX and who have an inadequate response to MTX.

A Study Comparing Upadacitinib (ABT-494) to Placebo and to Adalimumab in Adults With Rheumatoid Arthritis Who Are on a Stable Dose of Methotrexate and Who Have an Inadequate Response to Methotrexate

Upadacitinib for Rheumatoid Arthritis

This study is evaluating whether a drug called upadacitinib can help people with RA.

I have 6 or more swollen and tender joints.

I have tried only one bDMARD (not adalimumab) for my condition and either couldn't tolerate it or used it for less than 3 months.

I have stopped taking all rheumatic disease drugs except for methotrexate.

I have previously taken JAK inhibitor medications.

I have tried adalimumab or similar medications without enough improvement.

I have been diagnosed with rheumatoid arthritis for at least 3 months.

I have bone erosions and either a positive rheumatoid factor or anti-CCP antibodies.

I have a history of joint disease, but not RA. Secondary Sjogren's Syndrome is okay.

I have been on methotrexate for over 3 months and take folic or folinic acid.

Adalimumab

Placebo for Adalimumab

Placebo for Upadacitinib

Upadacitinib

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the extension study CLN-PXT3003-03.

Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL).

Period 2: All patients continue on twice dose 1 (2X5mL).

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

PXT3003 for Charcot-Marie-Tooth Disease

This study is evaluating whether a higher dose of PXT3003 might be more effective than the standard dose.

I was in the CLN-PXT3003-02 study, took placebo or dose 1, and completed 15 months including the final visit.

I am not taking any medications that are not allowed in the study.

I have signed, or if under 18, my parents and I have both signed the consent form for this study.

You were previously in a study for the same drug and completed a specific treatment period or had specific procedures done as part of that study.

You were part of a previous study and took a specific dose of the study drug, but the study ended early.

I agree to use birth control during the study.

PXT3003

All Individual Drugs Already Approved

The primary objective of this study is to evaluate the long-term safety and tolerability of filgotinib in participants who have completed one of the parent studies of filgotinib in rheumatoid arthritis (RA).

Long Term Extension Study to Assess the Safety and Efficacy of Filgotinib in Adults With Rheumatoid Arthritis

Long-Term Safety of Filgotinib for Rheumatoid Arthritis

This study is evaluating whether a drug called filgotinib is safe and well tolerated in people with rheumatoid arthritis.

I'm sorry, I cannot provide a summary for this screening criterion as it is incomplete. Please provide more information.

I completed a Gilead filgotinib study for RA and may benefit from continuing it.

There may be additional requirements for participation in the study that are not listed here.

You have a joint disease other than rheumatoid arthritis that could be risky or affect the study results, according to the doctor's judgment.

I am a woman who can have children and I have a negative pregnancy test before starting the study drug.

I agree to use birth control as per the study's requirements.

Filgotinib

Placebo to match filgotinib

This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Risdiplam for Spinal Muscular Atrophy

This study is evaluating whether a drug called risdiplam can be used to treat spinal muscular atrophy.

I have a history of cancer that is not considered cured.

I have previously used chloroquine, hydroxychloroquine, retigabin, vigabatrin, or thioridazine.

I have had an eye disease in the last year.

You are suspected of using drugs regularly.

You have recently tested positive for drugs or alcohol in your urine.

I haven't had a serious illness or fever in the last month.

I started treatment for spinal muscular atrophy with a specific medication less than 6 weeks ago.

You are allergic to risdiplam or any ingredients in it.

I have previously participated in the Moonfish study or have been treated with specific drugs for my condition.

I agree to not have sex or use birth control and not donate sperm.

You cannot meet the requirements of the study.

I have not had gene or cell therapy, except for AVXS-101.

I am willing to consider tube feeding and non-invasive ventilation for my child under 2, as recommended.

I have heart issues shown on an ECG or have cardiovascular disease.

As a caregiver, I am open to considering tube feeding and non-invasive ventilation for my child if recommended.

I have been part of the Moonfish study or treated with Nusinersen, Olesoxime, or AVXS-101.

I agree to either not have sex or use birth control, and not donate sperm.

My child under 2 was hospitalized for a lung problem recently and hasn’t fully recovered.

I don't have major issues with my digestive, kidney, liver, hormone, or heart systems.

I am over 6 years old and at high risk for suicidal behavior.

I haven't taken any drugs affecting FMO1 or FMO3 enzymes in the last 2 weeks.

I have been diagnosed with 5q-autosomal recessive SMA.

Risdiplam

Approved for 10 Other Conditions

The study objectives of Period 1 are to compare the efficacy, safety, and tolerability of upadacitinib 15 mg once daily (QD) and 30 mg QD versus placebo for the treatment of signs and symptoms in adults with moderately to severely active psoriatic arthritis (PsA) who have had an inadequate response or intolerance to biologic disease-modifying anti-rheumatic drug (bDMARD).

The objective of Period 2 is to evaluate the long-term safety, tolerability and efficacy of upadacitinib 15 mg QD and 30 mg QD in participants who have completed Period 1.

A Study Comparing Upadacitinib (ABT-494) to Placebo in Participants With Active Psoriatic Arthritis Who Have a History of Inadequate Response to at Least One Biologic Disease Modifying Anti-Rheumatic Drug (bDMARD)

Upadacitinib for Psoriatic Arthritis

This study is evaluating whether a drug called ABT-494 can help people with psoriasis.

I am currently using more than two non-biologic DMARDs or using specific DMARDs not allowed in the trial.

I have at least 3 tender and 3 swollen joints.

I have PsA but no other inflammatory joint diseases diagnosed before I was 17.

I have never taken JAK inhibitor medications.

I have been diagnosed with Psoriatic Arthritis for at least 6 months.

I have been diagnosed with plaque psoriasis.

I tried a biologic drug for over 12 weeks without improvement or couldn't tolerate it.

This study includes two periods. The main objective of Period 1 is to compare the efficacy of upadacitinib 15 mg once daily (QD) and 30 mg QD versus placebo and versus adalimumab (Humira®) in participants with moderately to severely active psoriatic arthritis (PsA) who have had an inadequate response to non-biologic DMARDs (DMARD-IR). Period 1 is also designed to compare the efficacy of upadacitinib 15 mg and 30 mg QD versus placebo for the prevention of structural progression.

The objective of Period 2 is to evaluate the long-term safety, tolerability and efficacy of upadacitinib 15 mg and 30 mg QD in participants who have completed Period 1.

A Study Comparing Upadacitinib (ABT-494) to Placebo and to Adalimumab in Participants With Psoriatic Arthritis Who Have an Inadequate Response to at Least One Non-Biologic Disease Modifying Anti-Rheumatic Drug (DMARD)

Upadacitinib vs. Adalimumab for Psoriatic Arthritis

This study is evaluating whether a drug called ABT-494 can help treat psoriatic arthritis.

I am on 2 or fewer non-biologic DMARDs for at least 12 weeks and at a stable dose for 4 weeks.

I have at least 3 tender and 3 swollen joints currently.

You have at least one erosion seen on an x-ray as confirmed by a central imaging review.

You can still participate if your previous diagnosis of reactive arthritis or certain types of spondyloarthritis changes to psoriatic arthritis (PsA).

You have either erosion on an x-ray, as seen by a central imaging review, or high levels of C-reactive protein in your blood.

My previous treatments for my condition with standard medications were not effective or I couldn't tolerate them.

I am currently taking more than two non-biologic DMARDs or using specific DMARDs not listed.

I was diagnosed with fibromyalgia, but now have a confirmed diagnosis of PsA.

I have not responded well to standard arthritis medication or cannot take them due to side effects.

I have had fibromyalgia, arthritis before 17, or have inflammatory joint disease.

Your high-sensitivity C-reactive protein (hs-CRP) levels are higher than what is considered normal at the lab.

I have previously taken medication that targets Janus Kinase.

Upadacitinib (ABT-494)

This randomized phase III trial studies digital tomosynthesis mammography and digital mammography in screening patients for breast cancer. Screening for breast cancer with tomosynthesis mammography may be superior to digital mammography for breast cancer screening and may help reduce the need for additional imaging or treatment.

Digital Tomosynthesis Mammography and Digital Mammography in Screening Patients for Breast Cancer

Digital Tomosynthesis vs. Standard Mammography for Breast Cancer Screening

This study is evaluating whether a type of mammogram may help detect breast cancer earlier.

I don't need a diagnostic mammogram for any breast disease symptoms.

I plan to, or have scheduled, a mammogram.

I have never had breast cancer or ductal carcinoma in situ before.

Digital Mammography

Digital Tomosynthesis Mammography

The primary objective of this multicenter randomized controlled study is to compare the safety and efficacy of minimally invasive hematoma evacuation with the Artemis Neuro Evacuation Device to best medical management for the treatment of intracerebral hemorrhage (ICH).

MIND: Artemis in the Removal of Intracerebral Hemorrhage

Minimally Invasive Surgery for Stroke

This study is evaluating whether a device which removes blood clots from the brain is safe and effective.

MUSC Clinical Trials 2022

Trials at this site

EPI-743 for Mitochondrial Disease

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Patient Reviews