Neurofibromatosis Type 1NCT00624234
Neurobiology and Treatment of Reading Disability in NF-1
A study testing Tutoring Program I and Tutoring Program II in people with Neurofibromatosis Type 1 and Reading Disabilities, run by Vanderbilt University at 1 site in United States.
Trial summary
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with idiopathic reading disabilities do, and to determine which intervention is best for particular… Read more about this study
- Who is it for?
- Ages 8 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Neurofibromatosis Type 1, Reading Disabilities
- What is being tested?
- Tutoring Program I and Tutoring Program II
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 184 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2006to Jan 2017
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- This study will be open to all individuals, ages 8 to 17 years, who meet eligibility criteria regardless of race, gender, or socioeconomic status.
- The Reading Disabilities group (including those with NF-1) is defined by scoring equal to or less than the 25th percentile on measures of basic word reading skills.
- The Control group (including those with NF1) is defined by scoring equal to or above the 40th percentile on the average of the Letter Word Identification and Word Attack subtests from the WJ-III.
Who cannot join
- Any child, regardless of which group he/she is recruited for, will be excluded if he/she meets any of the following criteria (determined during phone screening, medical review, and during testing):
- is in foster care;
- previous diagnosis of mental retardation;
- known uncorrectable visual impairment;
- history of known neurological disorder (e.g., epilepsy, spina bifida, cerebral palsy, traumatic brain injury);
- documented hearing impairment greater than 25 dB loss in either ear;
Show all 10Show fewer
- medical contraindication to MRI procedures, if participating in MRI (including exposure to metal and pregnancy);
- individuals known to have an IQ below 70;
- history or presence of a pervasive developmental disorder;
- during the DICA-IV parents indicate the presence of any severe psychiatric diagnoses or pervasive developmental disorder.
Trial locations and contacts
1 site in 1 country.
United States1 site
Vanderbilt University, Vanderbilt University Institute of Imaging Science
Nashville, Tennessee, 37232-2310
About this study
Official title: Neurobiology and Treatment of Reading Disability in Neurofibromatosis Type 1 (NF-1)
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with idiopathic reading disabilities do, and to determine which intervention is best for particular learner profiles.
Read the detailed description
The most common concern of parents of children with neurofibromatosis type 1 (NF-1) is learning disabilities (LD). Approximately one half of all children with NF-1 have LD-the most debilitating and common of which are reading disabilities.
The purpose of this study is to determine if children with NF-1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs known to improve the decoding deficits in children with idiopathic reading disabilities. The trial will also determine which intervention is best for particular learner profiles. The overall purpose of this research is to gain a deeper understanding of the characteristics and treatment of reading disabilities in NF-1.
In the trial, researchers will compare children with NF-1 who show weaknesses in reading to children with reading disabilities of no known cause (idiopathic) using two different interventions and behavioral and neurobiological measures. Both interventions focus on teaching sound-symbol relationships, but vary in terms of relative emphasis on verbal versus visual methods of teaching.
Scientists hope findings from the trial will advance knowledge about the best therapies for LD in children with NF-1. And, by further refining how children with NF-1 who have reading disabilities are similar (or different) to children with idiopathic reading disabilities, the researchers may be able to learn if reading interventions that address areas other than decoding will also benefit children with NF-1. Also, by understanding the similarities and/or differences in the neuropsychological and neurobiological profiles of children with NF-1 who have reading disabilities, and those without, scientists will be able to refine the cognitive phenotype and neurobiological characteristics of NF-1, which will further understanding of central nervous system abnormalities in NF-1.
- Conditions
- Neurofibromatosis Type 1, Reading Disabilities
- Being tested
- Tutoring Program I, Tutoring Program II
- Keywords
- neurofibromatosis type 1, reading disabilities, NF-1, learning disabilities, tutoring intervention
- Medical subject headings
- Neurofibromatosis 1, Dyslexia, Learning Disabilities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tutoring Program IBehavioural
Tutoring Program I is a structured multi-sensory program that is designed to gradually present the range of sounds and letters with focus on accuracy of phonological concepts and application of those concepts in phrases and sentences. The instruction uses a sequenced defined lesson plan with accuracy and automaticity criteria for skill progression. A range of manipulative and kinesthetic activities is outlined to maintain learner engagement in the intensive intervention design.
Tutoring Program IIBehavioural
Tutoring Program II is designed to teach visual and speech elements of reading separately at first, and then bring them together for maximum efficiency. The program uses the idea of teaching concepts about the structure of words. For example, students transfer the rules they have learned about one vowel or structure to another without specific instructions on the new one. Tutoring Program II incorporates pictures and activities to help remember strategies for increasing basic reading skills. Speed drills are also used for development of decoding automaticity.
| Study group | Role | What they receive |
|---|---|---|
| NF-Tutoring Program 1 | Experimental | Tutoring Program I Receives: Behavioral: Tutoring Program I |
| NF-Tutoring Program 2 | Experimental | Tutoring Program II Receives: Behavioral: Tutoring Program II |
| Typically Developing Readers | No intervention | Control group |
| IRD-Tutoring Program 1 | Experimental | Tutoring Program I Receives: Behavioral: Tutoring Program I |
| IRD-Tutoring Program 2 | Experimental | Tutoring Program II Receives: Behavioral: Tutoring Program II |
| Waitlist Control | No intervention | Intervention Control Group (RD) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in WJ-III Basic Reading Normative Update (Woodcock Johnson Psychoeducational Battery - 3rd Edition; WJ-III NU) Standard Score at 15 Hours
This metric measures change in reading abilities, including word recognition and decoding, as assessed by standard educational assessments (Woodcock Johnson Psychoeducational Battery - 3rd Edition Normative Update; WJ-III NU). The scores are reported as change in age-normed standard scores (a change of 15 standard score points would represent a change of 1 standard deviation in the general population).The Basic Reading score is a normed composite of the WJ-III subtests Letter-Word Identification and Word Attack, representing word-level reading skill.
Measured: 0 and 15 hours
Secondary outcomes (1)
Neuroimaging Data
Neuroimaging data consists of functional MRI and structural MRI measures.
Measured: Collected before and after intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2008 (estimated)
- Study startFeb 2006
- Primary completionMar 2013
- Study completionJan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 184 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Laura Cutting, Vanderbilt University
- Investigators
- Laurie E. Cutting, Ph.D., Principal investigator, Vanderbilt University Special Education DepartmentMartha Bridge Denckla, M.D., Principal investigator, Kennedy Krieger Institute, Johns Hopkins University, Johns Hopkins University School of MedicineSheryl L. Rimrodt, M.D., Principal investigator, Vanderbilt University Pediatrics DepartmentJohn Gore, Ph.D., Principal investigator, Vanderbilt University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00624234
- Sponsor's protocol ID
- R01NS049096
- Other IDs
- NF1-R01, R01NS049096
- Status last verified
- Jun 2025
- First submitted
- 25 Feb 2008
- Last updated
- 8 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Yes: De-identified behavioral data (Woodcock Johnson-III NU scores) may be shared with other researchers upon request. De-identified T1 structural neuroimaging scans will be shared with other researchers through a neuroimaging data warehouse or consortium.
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neurobiology and Treatment of Reading Disability in Neurofibromatosis Type 1 (NF-1)
- ClinicalTrials.gov ID
- NCT00624234
- Acronym / sponsor ID
- R01NS049096
- Conditions
- Neurofibromatosis Type 1, Reading Disabilities
- Keywords
- neurofibromatosis type 1, reading disabilities, NF-1, learning disabilities, tutoring intervention
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 184 participants (actual)
- Lead sponsor
- Vanderbilt UniversityUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Feb 2006 (actual)
- Primary completion
- Mar 2013 (actual)
- Study completion
- Jan 2017 (actual)
- First posted
- 27 Feb 2008 (estimated)
- Results
- Posted 10 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with idiopathic reading disabilities do, and to determine which intervention is best for particular learner profiles.
Detailed description
The most common concern of parents of children with neurofibromatosis type 1 (NF-1) is learning disabilities (LD). Approximately one half of all children with NF-1 have LD-the most debilitating and common of which are reading disabilities.
The purpose of this study is to determine if children with NF-1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs known to improve the decoding deficits in children with idiopathic reading disabilities. The trial will also determine which intervention is best for particular learner profiles. The overall purpose of this research is to gain a deeper understanding of the characteristics and treatment of reading disabilities in NF-1.
In the trial, researchers will compare children with NF-1 who show weaknesses in reading to children with reading disabilities of no known cause (idiopathic) using two different interventions and behavioral and neurobiological measures. Both interventions focus on teaching sound-symbol relationships, but vary in terms of relative emphasis on verbal versus visual methods of teaching.
Scientists hope findings from the trial will advance knowledge about the best therapies for LD in children with NF-1. And, by further refining how children with NF-1 who have reading disabilities are similar (or different) to children with idiopathic reading disabilities, the researchers may be able to learn if reading interventions that address areas other than decoding will also benefit children with NF-1. Also, by understanding the similarities and/or differences in the neuropsychological and neurobiological profiles of children with NF-1 who have reading disabilities, and those without, scientists will be able to refine the cognitive phenotype and neurobiological characteristics of NF-1, which will further understanding of central nervous system abnormalities in NF-1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| NF-Tutoring Program 1 | Experimental | Behavioral: Tutoring Program ITutoring Program I |
| NF-Tutoring Program 2 | Experimental | Behavioral: Tutoring Program IITutoring Program II |
| Typically Developing Readers | No intervention | –Control group |
| IRD-Tutoring Program 1 | Experimental | Behavioral: Tutoring Program ITutoring Program I |
| IRD-Tutoring Program 2 | Experimental | Behavioral: Tutoring Program IITutoring Program II |
| Waitlist Control | No intervention | –Intervention Control Group (RD) |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- This study will be open to all individuals, ages 8 to 17 years, who meet eligibility criteria regardless of race, gender, or socioeconomic status.
- The Reading Disabilities group (including those with NF-1) is defined by scoring equal to or less than the 25th percentile on measures of basic word reading skills.
- The Control group (including those with NF1) is defined by scoring equal to or above the 40th percentile on the average of the Letter Word Identification and Word Attack subtests from the WJ-III.
Key exclusion criteria (10 listed)
- Any child, regardless of which group he/she is recruited for, will be excluded if he/she meets any of the following criteria (determined during phone screening, medical review, and during testing):
- is in foster care;
- previous diagnosis of mental retardation;
- known uncorrectable visual impairment;
- history of known neurological disorder (e.g., epilepsy, spina bifida, cerebral palsy, traumatic brain injury);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tutoring Program ITutoring Program I is a structured multi-sensory program that is designed to gradually present the range of sounds and letters with focus on accuracy of phonological concepts and application of those concepts in phrases and sentences. The instruction uses a sequenced defined lesson plan with accuracy and automaticity criteria for skill progression. A range of manipulative and kinesthetic activities is outlined to maintain learner engagement in the intensive intervention design. | Behavioural | IRD-Tutoring Program 1, NF-Tutoring Program 1 |
| Tutoring Program IITutoring Program II is designed to teach visual and speech elements of reading separately at first, and then bring them together for maximum efficiency. The program uses the idea of teaching concepts about the structure of words. For example, students transfer the rules they have learned about one vowel or structure to another without specific instructions on the new one. Tutoring Program II incorporates pictures and activities to help remember strategies for increasing basic reading skills. Speed drills are also used for development of decoding automaticity. | Behavioural | IRD-Tutoring Program 2, NF-Tutoring Program 2 |
Primary outcome
Change From Baseline in WJ-III Basic Reading Normative Update (Woodcock Johnson Psychoeducational Battery - 3rd Edition; WJ-III NU) Standard Score at 15 Hours
Time frame: 0 and 15 hours
This metric measures change in reading abilities, including word recognition and decoding, as assessed by standard educational assessments (Woodcock Johnson Psychoeducational Battery - 3rd Edition Normative Update; WJ-III NU). The scores are reported as change in age-normed standard scores (a change of 15 standard score points would represent a change of 1 standard deviation in the general population).The Basic Reading score is a normed composite of the WJ-III subtests Letter-Word Identification and Word Attack, representing word-level reading skill.
1 secondary outcome
Neuroimaging Data
Time frame: Collected before and after intervention
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Additional study informationLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified behavioral data (Woodcock Johnson-III NU scores) may be shared with other researchers upon request. De-identified T1 structural neuroimaging scans will be shared with other researchers through a neuroimaging data warehouse or consortium.
Related trials for Neurofibromatosis Type 1
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laurie E. Cutting, Ph.D. principal investigator
Vanderbilt University Special Education Department
Martha Bridge Denckla, M.D. principal investigator
Kennedy Krieger Institute, Johns Hopkins University, Johns Hopkins University School of Medicine
Sheryl L. Rimrodt, M.D. principal investigator
Vanderbilt University Pediatrics Department
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Tutoring Program I and Tutoring Program II in people with Neurofibromatosis Type 1 and Reading Disabilities, run by Vanderbilt University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Apr 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in WJ-III Basic Reading Normative Update (Woodcock Johnson Psychoeducational Battery - 3rd Edition; WJ-III NU) Standard Score at 15 Hours (time frame: 0 and 15 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2025.