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Results for “Intellectual Disabilities Nutritional”

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Showing 20 of 0 results

Early research (Phase 1)Looking for participantsNCT06340685
What this trial is testing

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Jirair Krikor Bedoyan 6
Not applicableAvailableNCT01881334
What this trial is testing

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Who this might be right for
Hematologic MalignanciesInborn Errors of Metabolism DisordersImmune Deficiencies
Joanne Kurtzberg, MD
Post-approval studies (Phase 4)Study completedNCT03616509
What this trial is testing

GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition

Who this might be right for
Prader-Willi Syndrome
Corporacion Parc Tauli 30
Large-scale testing (Phase 3)Study completedNCT03852498
What this trial is testing

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 35
Not applicableActive Not RecruitingNCT04597385
What this trial is testing

Long-term Follow-Up for RGX-121

Who this might be right for
Mucopolysaccharidosis II
REGENXBIO Inc. 27
Large-scale testing (Phase 3)Ended earlyNCT01751802
What this trial is testing

Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

Who this might be right for
Lesch-Nyhan DiseaseSelf-injurious Behavior
Emalex Biosciences Inc. 9
Not applicableLooking for participantsNCT06214507
What this trial is testing

Danon Disease Natural History Study

Who this might be right for
Danon Disease
Rocket Pharmaceuticals Inc. 60
Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
Not applicableLooking for participantsNCT02829684
What this trial is testing

Register of Patients With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
University Hospital, Toulouse 500
Early research (Phase 1)Study completedNCT04539340
What this trial is testing

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 20
Testing effectiveness (Phase 2)Looking for participantsNCT06239116
What this trial is testing

RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment

Who this might be right for
Hypothalamic ObesityPrader-Willi SyndromePWS
Rhythm Pharmaceuticals, Inc. 150
Large-scale testing (Phase 3)Study completedNCT03231878
What this trial is testing

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

Who this might be right for
Adrenoleukodystrophy
Minoryx Therapeutics, S.L. 105
Not applicableWithdrawnNCT04591834
What this trial is testing

Mucopolysaccharidosis Type II Observational

Who this might be right for
Mucopolysaccharidosis II
REGENXBIO Inc.
Not applicableStudy completedNCT01043198
What this trial is testing

Microarray Analysis in Syndromic Obesity

Who this might be right for
Mental RetardationSyndromic Obesity
University Hospital, Bordeaux 90
Not applicableStudy completedNCT01479322
What this trial is testing

Plasma Adiponectin Level and Vascular Endothelial and Smooth Muscle Cell Function in Children With Prader-Willi Syndrome

Who this might be right for
Prader-Willi SyndromeObesity
Samsung Medical Center 51
Large-scale testing (Phase 3)Enrolling By InvitationNCT06075537
What this trial is testing

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 99
Testing effectiveness (Phase 2)Looking for participantsNCT06092034
What this trial is testing

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Who this might be right for
Danon Disease
Rocket Pharmaceuticals Inc. 14
Early research (Phase 1)Study completedNCT01401244
What this trial is testing

Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers

Who this might be right for
Genetic DisorderPrader-Willi SyndromeGrowth Disorder+2 more
Novo Nordisk A/S 30
Not applicableUnknownNCT03278899
What this trial is testing

Prospectively Assess Disease Progression in Male Children With X-ALD

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc. 100
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