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Results for “Rare Intellectual Disabilities”

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Showing 11 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00004314
What this trial is testing

Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease

Who this might be right for
Lesch-Nyhan Syndrome
National Center for Research Resources (NCRR) 2
Not applicableLooking for participantsNCT06677866
What this trial is testing

Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder

Who this might be right for
Fragile X Syndrome (FXS)Autism Spectrum Disorder
Bambino Gesù Hospital and Research Institute 20
Not applicableLooking for participantsNCT06796920
What this trial is testing

Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy

Who this might be right for
Adrenomyeloneuropathy Without Cerebral Involvement
Third Military Medical University 10
Not applicableNot Yet RecruitingNCT07400913
What this trial is testing

Implementation of Long-read Sequencing for the Diagnosis of Rare Diseases.

Who this might be right for
Rare DiseasesAlbinismIntellectual Disability
University Hospital, Bordeaux 150
Testing effectiveness (Phase 2)Ended earlyNCT03041324
What this trial is testing

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Who this might be right for
Mucopolysaccharidosis IIMPS II
Sangamo Therapeutics 9
Not applicableUnknownNCT03688594
What this trial is testing

Non Invasive Prenatal Test of Rare Genetic Diseases: Application to Rare Intellectual Disabilities

Who this might be right for
Genetic Disorders in Pregnancy
University Hospital, Strasbourg, France 60
Testing effectiveness (Phase 2)Looking for participantsNCT07654114
What this trial is testing

Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome

Who this might be right for
Fragile X Syndrome (FXS)Fragile X Syndrome
Connecta Therapeutics, S.L. 30
Early research (Phase 1)Study completedNCT06480968
What this trial is testing

CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction

Who this might be right for
Fragile X Syndrome
Connecta Therapeutics, S.L. 76
Not applicableLooking for participantsNCT07075107
What this trial is testing

Transcriptomic Analysis of Fibroblasts and Blood in Patients With Rare Diseases

Who this might be right for
Rare Genetic Disease
Assistance Publique Hopitaux De Marseille 62
Not applicableUnknownNCT04768803
What this trial is testing

Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity

Who this might be right for
Angelman SyndromeSmith-Magenis SyndromeX Fragile Syndrome+2 more
University Hospital, Toulouse 300
Not applicableStudy completedNCT04687007
What this trial is testing

SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-linked Adrenoleukodystrophy
Leipzig University Medical Center 68