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Monoclonal Antibodies
Adalimumab for Mucopolysaccharidosis
Phase 1 & 2
Waitlist Available
Led By Lynda Polgreen, MD
Research Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Weight ≥15 kg
Significant bodily pain reported by the CHQ-PF50 or SF-36 (> 1 SD more severe [below] than the general population mean)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
Study Summary
This trial is studying how well adalimumab works compared to placebo in treating patients with MPS I, II, or VI.
Who is the study for?
This trial is for children and adults over 5 years old with Hurler Syndrome, Hunter Syndrome, or Maroteaux-Lamy Syndrome who have either been treated with ERT for at least a year or not treated at all. Participants must have joint motion limitations in at least 3 joints, weigh more than 15 kg, and experience significant pain.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of Adalimumab injections compared to saline solution (placebo) on joint and skeletal disease in patients with MPS I, II or VI. It's randomized and double-blind: participants don't know if they're getting the real drug or placebo.See study design
What are the potential side effects?
Adalimumab may cause allergic reactions, increased risk of infections including TB, potential heart issues like congestive heart failure, liver problems such as Hepatitis B reactivation, nervous system disorders like multiple sclerosis or Guillain-Barre syndrome.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I weigh at least 15 kilograms.
Select...
I experience more pain than most people do.
Select...
I have been diagnosed with MPS I, II, or VI.
Select...
I have (or have not) been on enzyme replacement therapy for at least a year.
Select...
I have 3 or more joints with limited movement.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Adalimumab trough
Pain - 16 weeks
Secondary outcome measures
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability
Joint range-of-motion - 16 weeks
Joint range-of-motion - 52 weeks
+1 moreTrial Design
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Open-label adalimumabExperimental Treatment1 Intervention
Open-label extension of adalimumab dose
Group II: AdalimumabExperimental Treatment1 Intervention
20 mg subQ every other week (weight 15to <30 kg) 40 mg subQ every other week (weight ≥30 kg). Non-responders will be escalated to weekly dosing.
Group III: PlaceboPlacebo Group1 Intervention
Saline placebo comparator
Find a Location
Who is running the clinical trial?
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterLead Sponsor
101 Previous Clinical Trials
46,287 Total Patients Enrolled
Lynda Polgreen, MDPrincipal InvestigatorThe Lundquist Institute at Harbor-UCLA Medical Center
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I had a stem cell transplant less than 2 years ago.I have an active or chronic Hepatitis B infection.I will not be exposed to live vaccines during the study.I have tested positive for TB or been recently exposed to it.I have a condition like multiple sclerosis or Guillain-Barre syndrome.I have had a serious infection like TB or a fungal infection.I have not been on immune suppression therapy in the last year.I weigh at least 15 kilograms.I have or had lymphoma or another type of cancer.You have problems with your blood, like having low levels of different types of blood cells or a condition called aplastic anemia.I experience more pain than most people do.I have been diagnosed with MPS I, II, or VI.You are allergic to latex.My heart's pumping ability is below normal.I have (or have not) been on enzyme replacement therapy for at least a year.I am 5 years old or older.I have 3 or more joints with limited movement.You have a condition called active graft versus host disease.You have a known or suspected allergy to adalimumab or similar products.I do not have any active infections.
Research Study Groups:
This trial has the following groups:- Group 1: Adalimumab
- Group 2: Open-label adalimumab
- Group 3: Placebo
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Do we have previous data to suggest that Adalimumab Injection [Humira] is effective?
"Currently, there are 51 ongoing clinical trials studying the effects of Adalimumab Injection [Humira]. Of these active studies, 14 are in Phase 3. The majority of research locations for Adalimumab Injection [Humira] are based in London, but there are a total of 2044 medical centres running trials for this medication worldwide."
Answered by AI
How many total subjects are included in the research?
"The listing on clinicaltrials.gov says that the research team is still recruiting patients for this study. The trial was originally posted on 6/5/2017, and the most recent update was 8/9/2022. They are looking to enroll 14 individuals at 2 different sites."
Answered by AI
Are there any available spots for volunteers in this research project?
"This trial, which can be found on clinicaltrials.gov, is still looking for participants. The original posting was on June 5th, 2017 and the most recent update was on August 9th, 2022."
Answered by AI
What conditions does Adalimumab Injection [Humira] help ameliorate?
"Most frequently, Adalimumab Injection [Humira] is given to patients suffering from ankylosing spondylitis. That said, this medication can also be used as treatment for rheumatoid arthritis, psoriatic arthritis, and polyarticular juvenile idiopathic arthritis."
Answered by AI
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