Mucopolysaccharidosis

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26 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.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication

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+

28 Participants Needed

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

41 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

99 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 - 26

54 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

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

48 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

47 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

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

15 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

100 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

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:Recruiting
Trial Phase:Phase 3

80 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:Not Yet Recruiting
Trial Phase:Phase 2, 3

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

6 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:Recruiting
Trial Phase:Phase 1, 2
Age:0 - 18

26 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

40 Participants Needed

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

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

80 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

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

2 Participants Needed

Why Other Patients Applied

"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

"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

"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

"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 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

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

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 pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA).
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1
Age:24 - 72

9 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+

14 Participants Needed

Cannabidiol for Sanfilippo Syndrome

Torrance, California
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in treating the neurobehavioral symptoms and functional outcomes of Sanfilippo syndrome. Each participant's caregiver will be asked to complete surveys related to the participant's behavior, mood, sleep, stooling, pain, and caregiver stress intermittently throughout the study. All participants will be enrolled into one of two cohorts based on enrollment order: 1. Sentinel Safety Cohort (first 5 participants) - all participants treated with Epidiolex (cannabidiol) 2. Controlled Cohort (next 30 participants) - participants randomized 1:1 (equal chance) to start treatment with Epidiolex (cannabidiol) or placebo for 16 weeks, followed by an 8-week washout period (no treatment). Participants then switch to the opposite treatment group for 16 weeks followed by all participants treated for 52 weeks with Epidiolex (cannabidiol).
Stay on current meds
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Phase 2, 3
Age:4+

35 Participants Needed

JR-171 for Hurler Syndrome

Oakland, California
This trial continues to test JR-171, an IV medication, in patients with MPS I. The goal is to find the best dose to help manage their symptoms by aiding in the breakdown of certain substances in their bodies.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2

14 Participants Needed

Voxzogo for Mucopolysaccharidosis

Oakland, California
This trial tests vosoritide in children aged 5-10 with severe growth issues from MPS IVA or VI. Vosoritide works by mimicking a natural protein that helps bones grow longer. Vosoritide has been previously tested in children with achondroplasia, showing positive effects on growth.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:5 - 10

6 Participants Needed

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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.

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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?

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 Cannabidiol for Sanfilippo Syndrome, Ambroxol for Sanfilippo Syndrome and GC1130A for Sanfilippo Syndrome to the Power online platform.

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