anakinra for MPS III A

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
The Lundquist Institute at Harbor-UCLA Medical Center, Torrance, CA
MPS III A+1 More
anakinra - Biological
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

This study is evaluating whether anakinra, a drug that decreases inflammation, can improve behavior, sleep, stooling, communication, mood, and gait in people with Sanfilippo syndrome.

See full description

Eligible Conditions

  • MPS III A

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for MPS III A

Study Objectives

This trial is evaluating whether anakinra will improve 1 primary outcome in patients with MPS III A. Measurement will happen over the course of up to 8 weeks.

up to 8 weeks
multi-domain responder index (MDRI) - composite outcome

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for MPS III A

Side Effects for

Actos
right side weakness
14%
access related
14%
arrhythmia
14%
seizure
0%
fluid overload
0%
allergic reaction
0%
injection site reaction
0%
syncope
0%
diarrhea
0%
This histogram enumerates side effects from a completed 2017 Phase 2 trial (NCT02278562) in the Actos ARM group. Side effects include: right side weakness with 14%, access related with 14%, arrhythmia with 14%, seizure with 0%, fluid overload with 0%.

Trial Design

1 Treatment Group

treatment
1 of 1
Experimental Treatment

This trial requires 20 total participants across 1 different treatment group

This trial involves a single treatment. Anakinra is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 & 3 and have had some early promising results.

treatment
Biological
anakinra 100 mg subcutaneous once daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Anakinra
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 8 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 8 weeks for reporting.

Closest Location

The Lundquist Institute at Harbor-UCLA Medical Center - Torrance, CA

Eligibility Criteria

This trial is for patients born any sex of any age. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
If on Genistein, must have been on a stable dose for 6 months prior to enrollment
MPS III
≥ 4 years of age
Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 17 years of age, assent must also be provided when cognitively possible.
If on melatonin or other sleep medications, must have been on stable doses for the past 3 months

Patient Q&A Section

Can mps iii a be cured?

"There is no evidence to suggest that mps iii a can be cured. The available studies suggest that the use of this medicine is harmful. Mps iii a should not be prescribed off-label. There are no trials or meta-analyses that suggest that the use of methylphenidate in children under 12 years old lead to adverse effects and adverse reactions such as suicidal ideation, psychosis, tic disorders, and neurocognitive problems. While the risks and harms of methylphenidate in children are well documented, the evidence does not support the claims of health care providers who administer the drug to children. Physicians can reassure patients and parents that methylphenidate cannot be cured; only the severity of the symptoms will decrease with treatment." - Anonymous Online Contributor

Unverified Answer

What are common treatments for mps iii a?

"Common treatment recommendations based on a review of published literature. One must also be aware of and cautious about the medications used when considering treatment." - Anonymous Online Contributor

Unverified Answer

What is mps iii a?

"It appears that patients with SMA have a slightly higher incidence, and a more rapid progression of chronicity of the disease than does age of onset and number of affected joints." - Anonymous Online Contributor

Unverified Answer

What causes mps iii a?

"(1) There is some evidence that women are affected more often than men; (2) this effect may be more profound among African Americans; (3) there is no relationship between mps iii a and age or smoking; (4) there is little evidence of familial clustering; (5) a linkage exists between mps iii a and SLC6A4 in one family; (6) the genetic locus for mps iii a is on the q arm of the arm of the X chromosome." - Anonymous Online Contributor

Unverified Answer

How many people get mps iii a a year in the United States?

"[The rate of mps iii a a is about 100,000 people per year in the United States, with approximately 5,400 new a cases per year per 100,000, and 2,300 b cases of cancer per year per 100,000 in the United States. a was the third most common cause of cancer death in the United States in 1988, following colon cancer and lung cancer. a is prevalent among all racial and ethnic groups in the United States, and is a major health problem in minorities. The most common symptom seen in a a patient is dizziness or faintness or vertigo; a is associated with vision loss, fatigue, and chest pain in about one in five people at any given time." - Anonymous Online Contributor

Unverified Answer

What are the signs of mps iii a?

"The most common symptoms noticed in our study are: [lower back pain](https://www.withpower.com/clinical-trials/lower-back-pain), loss of interest in sex, unexplained weight loss, and pain in the groin area. Some signs and symptoms were also evident in patients with psoriasis, which may represent a skin-related autoimmune phenomenon known as "acrodermatitis enteropathica diabolica" (AED), the most severe and debilitating form of pemphigus vulgaris. However, patients with psoriasis should not necessarily be excluded a diagnosis of MPA." - Anonymous Online Contributor

Unverified Answer

Does mps iii a run in families?

"Recent findings were obtained by a multidisciplinary collaboration with the expertise of two medical schools and by the collaborative cooperation of several units. Recent findings allows us to estimate that the disease penetrance level is higher in the families in which the subjects have the same MPS IIIa genotype than in the other families. The study also reveals that the homozygous MPS IIIa carriers have significantly impaired hepatic LSM function on magnetic resonance imaging." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of anakinra?

"No major side effects on anakinra or the combination were reported by patients treated with anakinra monotherapy. No patient had to stop taking anakinra. In contrast, with anakinra combination, one patient stopped taking it due to diarrhoea. Anakinra is a drug that can be taken with no drug interactions occurring, and no side effects recorded." - Anonymous Online Contributor

Unverified Answer

What is the latest research for mps iii a?

"Mps iii a is a rare autosomal recessive disease that may benefit from gene therapy. Gene therapy trials are under way in multiple clinical institutes. Data from a recent study of these trials may lead to the development of treatments for MP III a. Please visit this article for more information about gene therapy: http://www.ncbi.nlm.nih.gov/books/NBK1949.shtml." - Anonymous Online Contributor

Unverified Answer

Is anakinra safe for people?

"There were no clinically significant clinical or lab adverse events after administration of anakinra. In the absence of good and well-designed controlled large-scale clinical studies with relevant outcome measures, we feel it is best to manage individual case histories to evaluate for adverse events." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating mps iii a?

"At present, there is no cure for a patient suffering from Mps iii a. However, it is possible to slow down the progression of the disease and the patient will hopefully live a more normal life." - Anonymous Online Contributor

Unverified Answer

Does anakinra improve quality of life for those with mps iii a?

"Although the number of patients was low and the follow-up duration too short to draw definite conclusions, improvements in quality of life were found, and the potential to alter the course of the disease was suggested." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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