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Why We Started Power

We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

Bask
Bask GillCEO at Power
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      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      34 Mucopolysaccharidosis Trials Near You

      Power is an online platform that helps thousands of Mucopolysaccharidosis 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 Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      Long-term Follow-up After ABO-102 Treatment for Sanfilippo Syndrome

      Columbus, Ohio
      This trial aims to evaluate the safety and tolerability of ABO-102 in patients with MPS IIIA who have already participated in a previous trial. ABO-102 is a gene therapy that delivers a healthy gene to improve their condition. ABO-102 is a gene therapy that has been developed to treat MPS IIIA by delivering a healthy gene to correct the enzyme deficiency.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Other Trials, Medical Conditions, Others

      41 Participants Needed

      Gene Therapy for Sanfilippo Syndrome

      Columbus, Ohio
      The main objective of this study is to evaluate the efficacy and safety of ABO-102 for the treatment of MPS IIIA.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Age:6+

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      28 Participants Needed

      Tividenofusp Alfa for Hunter Syndrome

      Cincinnati, Ohio
      This trial is testing a new treatment called tividenofusp alfa for patients with a rare genetic disorder called MPS II. The treatment aims to replace missing enzymes to help break down harmful substances in the brain and body.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2, 3
      Age:2 - 25

      Key Eligibility Criteria

      Disqualifiers:Genetic Mutation, Gene Therapy, Others
      Must Be Taking:Enzyme Replacement

      63 Participants Needed

      Infigratinib for Achondroplasia

      Cincinnati, Ohio
      This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:3 - 17

      Key Eligibility Criteria

      Disqualifiers:Hypochondroplasia, Malignancy, Ectopic Calcification, Others
      Must Not Be Taking:CYP3A4 Agents, Antacids

      110 Participants Needed

      Infigratinib for Achondroplasia

      Cincinnati, Ohio
      This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 2
      Age:3 - 18

      Key Eligibility Criteria

      Disqualifiers:Hypochondroplasia, Malignancy, Fracture, Others
      Must Not Be Taking:CYP3A4 Inducers/inhibitors, Growth Hormone

      300 Participants Needed

      Tividenofusp Alfa for Hunter Syndrome

      Cincinnati, Ohio
      This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 2, 3
      Age:< 18

      Key Eligibility Criteria

      Disqualifiers:Unstable Conditions, Significant Comorbidities, Others

      99 Participants Needed

      ELAPRASE + Prophylactic Therapy for Hunter Syndrome

      Twinsburg, Ohio
      The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome. In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR. Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously \[IV\], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle). Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR. Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 4
      Age:< 6
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Investigational Drug, Growth Hormones, Others
      Must Be Taking:Elaprase

      5 Participants Needed

      RGX-121 Gene Therapy for Hunter Syndrome

      Pittsburgh, Pennsylvania
      RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2, 3
      Age:4 - 5
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Neuropsychiatric Condition, Ventricular Shunt, Others
      Must Not Be Taking:Idursulfase, Blood Brain Barrier-crossing ERT

      48 Participants Needed

      AX 250 for Sanfilippo Syndrome

      Pittsburgh, Pennsylvania
      This trial tests the safety and effectiveness of AX 250, a drug delivered directly to the brain through an implanted device, in improving cognitive function in patients with MPS IIIB.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:0 - 18

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      20 Participants Needed

      AX 250 for Sanfilippo Syndrome Type B

      Pittsburgh, Pennsylvania
      This trial tests AX 250, a medicine given directly into the brain, in people with MPS IIIB. The goal is to see if it improves thinking, communication, and quality of life by delivering the medicine straight to where it's needed in the brain.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 4

      Key Eligibility Criteria

      Disqualifiers:Seizure Disorder, Hydrocephalus, Others
      Must Not Be Taking:Stem Cell, Gene Therapy, Others

      15 Participants Needed

      Tividenofusp Alfa for Hunter Syndrome

      Pittsburgh, Pennsylvania
      This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:2 - 18
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Unstable Conditions, Stroke, Others
      Must Be Taking:ERT

      47 Participants Needed

      Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders

      Pittsburgh, Pennsylvania
      The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:2 - 55

      Key Eligibility Criteria

      Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others

      100 Participants Needed

      Infigratinib for Dwarfism

      London, Ontario
      ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib, at one of two doses, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 2, 3
      Age:3 - 18

      Key Eligibility Criteria

      Disqualifiers:ACH, Malignancy, Ectopic Calcification, Others
      Must Not Be Taking:Phosphorus, Calcium, Glucocorticoids, Others

      24 Participants Needed

      Elaprase IV Infusion for Hunter Syndrome

      Chicago, Illinois
      This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 4
      Age:< 5
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Blood Transfusions, Non-compliance, Others
      Must Not Be Taking:Investigational Drugs, Idursulfase-IT, Growth Hormones

      21 Participants Needed

      JR-141 for Hunter Syndrome

      Chicago, Illinois
      This trial is testing a new medicine for patients with MPS II to see if it is safe and effective. Previous research in animals helped design this trial to evaluate the new medicine for MPS II patients.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Gene Therapy, HSCT, Brain Surgery, Others
      Must Be Taking:Enzyme Replacement

      86 Participants Needed

      Ambroxol for Sanfilippo Syndrome

      Fairfax, Virginia
      A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2, 3

      Key Eligibility Criteria

      Disqualifiers:Cardiac Disease, Pulmonary Disease, Seizures, Others
      Must Not Be Taking:CYP3A Inducers/inhibitors

      10 Participants Needed

      Idursulfase-IT + Elaprase for Hunter Syndrome

      Toronto, Ontario
      The study is an extension of two previous studies (HGT-HIT-046 \[NCT01506141\] and SHP609-302 \[NCT02412787\]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome and cognitive impairment. Participants will receive the same treatment as in the previous studies.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2, 3
      Age:3 - 19
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Seizure Disorder, Bleeding Disorder, Hypertension, Others
      Must Be Taking:Idursulfase-IT, Elaprase

      6 Participants Needed

      Infigratinib for Achondroplasia

      Baltimore, Maryland
      This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:0 - 32

      Key Eligibility Criteria

      Disqualifiers:Hypochondroplasia, Premature Birth, Others
      Must Be Taking:Vitamin D

      77 Participants Needed

      DNL126 for Sanfilippo Syndrome

      Chapel Hill, North Carolina
      This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks (approximately 6 months) and is followed by a 72-week (approximately 18 month) open-label extension (OLE). Participants with MPS IIIA will be enrolled in two planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:0 - 18

      Key Eligibility Criteria

      Disqualifiers:Unstable Conditions, Non-ambulatory, Others
      Must Not Be Taking:CNS-targeted ERT, Genistein, Anakinra

      20 Participants Needed

      Stem Cell Therapy for Inherited Metabolic Brain Diseases

      Durham, North Carolina
      The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.
      Stay on current meds
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:1 - 22

      Key Eligibility Criteria

      Disqualifiers:Prior Transplant, Seizures, HIV, Others
      Must Not Be Taking:Immunosuppressives, Chemotherapy

      40 Participants Needed

      Why Other Patients Applied

      "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

      "I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

      ID
      Pancreatic Cancer PatientAge: 40

      "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'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

      "I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

      FF
      ADHD PatientAge: 31
      Match to a Mucopolysaccharidosis Trial

      Gene Therapy vs Stem Cell Treatment for Hurler Syndrome

      Philadelphia, Pennsylvania
      This trial compares a new gene therapy called OTL-203 with traditional stem cell transplants in patients with a severe genetic disorder called MPS-IH. The goal is to see if gene therapy can better correct the genetic defect causing the disease compared to replacing damaged cells with healthy ones from a donor.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:28 - 30

      Key Eligibility Criteria

      Disqualifiers:HIV, Hepatitis, Cancer, Seizures, Others
      Must Not Be Taking:Prohibited Medications

      41 Participants Needed

      JR-141 for Hunter Syndrome

      Philadelphia, Pennsylvania
      This trial is testing a medication to see if it is safe and effective for patients with MPS II, a rare genetic disorder. The drug works by replacing or fixing a missing or malfunctioning enzyme to help reduce symptoms. Idursulfase has been used to treat MPS II by replacing the deficient enzyme.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Gene Therapy, Drug Allergy, Others
      Must Not Be Taking:Gene Therapy, NSAIDs

      80 Participants Needed

      Gene Therapy for Hunter Syndrome

      New Brunswick, New Jersey
      RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety, efficacy, and pharmacodynamic dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II (Hunter Syndrome)
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:4 - 5
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Renal Insufficiency, Uncontrolled Hypertension, Cancer, Others
      Must Not Be Taking:Methylprednisolone, Tacrolimus, Sirolimus, Prednisone

      2 Participants Needed

      Bisphosphonates for Hearing Loss

      New York, New York
      Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in their 20s. Bisphosphonates have been successfully used to treat otosclerosis, a common cause of hearing loss similar to OI-related hearing loss. As many individuals with OI-related hearing loss also present with otosclerosis and because of their mechanistic similarities, the investigators propose studying the effects of bisphosphonate treatment on individuals diagnosed with both OI type I and hearing loss, thereby determining its effectiveness as a potential treatment for hearing loss. The investigators will enroll 50 individuals diagnosed with type I OI and age 18-100. 25 adults will be enrolled into the treatment arm and receive bisphosphonate treatment (must have at least mild hearing loss), while 25 adults will be enrolled into the control arm. The investigators will enroll 25 children (6-17 years of age) diagnosed with OI who are currently receiving bisphosphonate treatment as part of their care for orthopedic symptoms. The investigators will also observe 25 children (6-17 years of age) diagnosed with OI who are NOT currently receiving bisphosphonate treatment. The study duration is 63 months (approximately 5 years). Enrollment is anticipated to begin in November 2019.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 4
      Age:6 - 100

      Key Eligibility Criteria

      Disqualifiers:Family History Hearing-loss, Pregnancy
      Must Be Taking:Bisphosphonates

      100 Participants Needed

      RGX-121 Gene Therapy for Hunter Syndrome

      Montréal, Quebec
      This trial tests RGX-121, a gene therapy designed to help children with severe MPS II by delivering a healthy gene to their brain cells. The goal is to produce an enzyme that can improve brain function. The study will check if this treatment is safe and effective. Brain-targeted hematopoietic stem cell gene therapy provides a promising therapy for MPS II patients.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:5 - 17
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      6 Participants Needed

      Engineered B Cell Therapy for Mucopolysaccharidosis I

      Minneapolis, Minnesota
      This trial tests ISP-001, which uses modified immune cells to help adults with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie by making a missing enzyme. The goal is to see if this approach is safe and tolerable.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1

      Key Eligibility Criteria

      Disqualifiers:Cancer, Autoimmune Disorders, HSCT, Others
      Must Not Be Taking:Immune Suppressants, Oxygen Therapy

      2 Participants Needed

      Stem Cell Transplantation for Osteopetrosis

      Minneapolis, Minnesota
      This trial tests a treatment using busulfan and fludarabine to prepare patients with certain genetic disorders for a bone marrow transplant. The goal is to safely replace their bone marrow with healthy donor cells by carefully monitoring drug levels. Busulfan is used to clear out bone marrow cells before the transplant, and fludarabine is being tested as a less harmful alternative to another drug.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 55

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Myeloablative Chemotherapy, Infections, Others

      100 Participants Needed

      GC1130A for Sanfilippo Syndrome

      Minneapolis, Minnesota
      The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:12 - 18

      Key Eligibility Criteria

      Disqualifiers:CNS Impairment, MRI Contraindications, Others
      Must Not Be Taking:Investigational Drugs

      9 Participants Needed

      GI Health Product for Diarrhea

      Del Mar, California
      A Randomized, Double-Blind, Placebo-Controlled Direct-to-Consumer Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Unphased
      Age:21+

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Cardiac Dysfunction, Liver Disease, Others
      Must Not Be Taking:Anticoagulants, Antidepressants, Antipsychotics, Others

      550 Participants Needed

      Adalimumab for Mucopolysaccharidosis

      Torrance, California
      Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:5+

      Key Eligibility Criteria

      Disqualifiers:Lymphoma, Active Infection, TB, Others
      Must Not Be Taking:Immune Suppressants

      14 Participants Needed

      12

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      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?
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      Frequently Asked Questions

      How much do Mucopolysaccharidosis clinical trials pay?

      Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

      How do Mucopolysaccharidosis clinical trials work?

      After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Mucopolysaccharidosis trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Mucopolysaccharidosis is 12 months.

      How do I participate in a study as a "healthy volunteer"?

      Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

      What does the "phase" of a clinical trial mean?

      The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

      Do I need to be insured to participate in a Mucopolysaccharidosis medical study?

      Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

      What are the newest Mucopolysaccharidosis clinical trials?

      Most recently, we added GI Health Product for Diarrhea, Gene Therapy for Hunter Syndrome and Infigratinib for Achondroplasia to the Power online platform.