Babble Boot Camp for Galactosemias

Phase-Based Estimates
1
Effectiveness
1
Safety
Arizona State University, Tempe, AZ
Galactosemias+6 More
Babble Boot Camp - Behavioral
Eligibility
< 18
All Sexes
Eligible conditions
Galactosemias

Study Summary

Preventing Speech and Language Disorders in Children With Classic Galactosemia

See full description

Eligible Conditions

  • Galactosemias
  • Dyslalia
  • Language Disorders
  • Disease
  • Language Disorders in Children
  • Classic Galactosemia
  • Speech Disorders
  • Speech Disorders in Children

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether Babble Boot Camp will improve 2 primary outcomes and 3 secondary outcomes in patients with Galactosemias. Measurement will happen over the course of Through study completion, an average of 4 years 2 months.

Year 4
Child health and development using the Ages and Stages Questionnaires 3
Cognitive development
Expressive language skills
Quality of life using the PedsQL questionnaires
Speech sound production accuracy

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

2 Treatment Groups

No Control Group
Treatment cohort with classic galactosemia, delayed start

This trial requires 309 total participants across 2 different treatment groups

This trial involves 2 different treatments. Babble Boot Camp is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

Treatment cohort with classic galactosemia, delayed start
Behavioral
The children in the control cohort enter the study when they are younger than 5 months old and participate in the close monitoring until they are 24 months old. They start getting the same treatment type and intensity as the treatment cohort but at a delayed age, when they turn 15 months.
Treatment cohort with classic galactosemia
Behavioral
These children and their parents receive the Babble Boot Camp intervention and also participate in the close monitoring activities (progress reports that the speech-language pathologist generates during the online meeting with the family; monthly daylong audio recording; questionnaires that are sent out every three to six months; formal speech and language testing at ages 2 1/2, 3 1/2, and 4 1/2 years).

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: through study completion, an average of 4 years 2 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly through study completion, an average of 4 years 2 months for reporting.

Who is running the study

Principal Investigator
B. P.
Prof. Beate Peter, Associate Professor
Arizona State University

Closest Location

Arizona State University - Tempe, AZ

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Newborn diagnosis of classic galactosemia
At least one parent must have at least an 8th grade education to be able to fill out the questionnaires
Any ethnic or racial background
Primary language in the home is English
Any geographic region in the US and other countries because the intervention is done online
Computer and internet access (we can help if a family wants to participate but doesn't have this access)

Patient Q&A Section

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

What is galactosemias?

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Galactosemia is a condition caused by an insufficiency or complete absence of the enzyme galactose-1-phosphate uridylyltransferase. It affects boys first, and tends to affect males in early life. Galactosemia is common in certain ethnic groups.

Unverified Answer

What are the signs of galactosemias?

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Symptoms associated with galactosemias can be observed throughout life, and may manifest as a persistent cough or a sudden worsening of symptoms such as weakness, lethargy or vomiting in infancy. The characteristic signs of galactosemic patients are the enlargement of the liver and spleen and the appearance of jaundice.

Unverified Answer

How many people get galactosemias a year in the United States?

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In children, the estimated prevalence of Gal-FSM/GALT deficiency and non-Gal-FSM/GALT deficiency is 1.08/110,000 and 6.18/103,000, respectively. Children without these rare disorders do not usually get proper care. Among parents of a child diagnosed with Gal-FSM/GALT deficiency, the estimated prevalence of the disorder is 0.02%. Thus, the prevalence of Gal-FSM/GALT deficiency should be considered in infants less than 12 months of age; in particular, in those younger than 5 month's old. However, it is difficult to detect Gal-FSM/GALT deficiency in adults.

Unverified Answer

What are common treatments for galactosemias?

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Galactosemia is often treated with oral foods or fluids containing fructose and/or glucose that will be metabolized into galactitol. Other solutions include insulin or parenteral galactose. Galactosemia is commonly treated with intravenous galactose.\n

Unverified Answer

Can galactosemias be cured?

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It appears unlikely that people suffering from GalQ-deficient galactosemia can be cured from the disorder. However, people affected with a deficiency of GalQ seem to be at risk of acquiring a GalQ null allele from their parents or from de novo errors in the conversion of Galβ-1,4Galβ-1,3Gal into the Galβ1,3Galβ1,4Gal form in their cells. This explains the inheritance of Gal-deficient galactosemia along with GalQ-deficient galactosemia in families with at least two occurrences of GalQ-deficient galactosemia in their pedigree.

Unverified Answer

What causes galactosemias?

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Galactosemia may be a consequence of genetic disorders or due to dietary habits. The high incidence of galactosemia in Asian populations indicates that it is not caused by dietary factors.

Unverified Answer

Have there been other clinical trials involving babble boot camp?

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Results from a recent paper of this study lend some support to the validity of babble boot camp as a clinical intervention for pediatric patients with IGHAD.

Unverified Answer

What are the common side effects of babble boot camp?

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As the disease progresses most patients require supplemental food and medications. Babble boot camp has no specific dietary restrictions. Although the majority of side effects reported occurred at the beginning or after the completion of the program, most were minor and not serious. The most common side effect is headache. Most side effects were self-limited. Patients should contact their healthcare provider if any serious side effects occur.

Unverified Answer

Does galactosemias run in families?

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Galactosemia probably has not emerged because of genetic considerations due to the extremely low frequency and variable expression of mutated GLUT2 alleles. Although this article is based on family studies of only one patient from North America, these data should serve as a foundation to help identify patients throughout the world who are at risk from genetic testing for Galactosemia.

Unverified Answer

What is babble boot camp?

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This is the first article to provide a comprehensive overview of the basic topics taught in babble boot camp. In addition, we also discuss several important aspects of these topics pertinent to current research in the field of genetics, including how specific genes can trigger tumors such as skin cancers and how the environment can modulate the expression of specific genes.

Unverified Answer

Who should consider clinical trials for galactosemias?

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To make the decision from a patient's perspective of whether or not they would be likely to enroll in a clinical trial, a clinician should assess several risk factors from the patient's perspective. If a patient has a positive score (>/= 1) from any parameter, they should be assessed more accurately by a specialist. Otherwise, patients can be reassured that enrollment in a clinical trial is unlikely to yield benefit. A patient's perspective of the risk and potential benefits of a clinical trial should be balanced with the risk of failure to find benefit of clinical trial enrollment.

Unverified Answer

How does babble boot camp work?

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Babble can bring together a diverse group of individuals who may all have different backgrounds and interests, yet they find an environment where they can develop a passion and learn new skills to enhance their careers. One of the best aspects for anyone who wants to learn more about babies and families is the opportunity for a mentor and other people to help them understand better. Babble is a unique camp where you can be part of the learning community. As the first baby boot camp, Babble has helped more families that want to get a better understanding of babies. Babble encourages them to ask more questions about the process of getting diagnosed and treats their question honestly, so that new parents know that Babble can help them with any question about babies.

Unverified Answer
See if you qualify for this trial
Get access to this novel treatment for Galactosemias by sharing your contact details with the study coordinator.