Clinical trials: Syndrome X
Rett Syndrome
Trial of Dextromethorphan in Rett Syndrome
Increased brain glutamate and its N-methyl-D-aspartate (NMDA) receptors found in the brain of younger Rett syndrome (RTT) patients cause toxic damage to neurons (the brain's nerve cells), and contributing to EEG spikes.
- Trial name
- NCT00593957
- Testing
- Dextromethorphan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Fragile X Syndrome, Genetic Diseases
Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
The purpose of the study is to test the efficacy of a 20 week multi-modal treatment comprised of lovastatin or placebo, and the Parent-implemented Language Intervention (PILI) in children with fragile X syndrome (FXS).
- Trial name
- NCT02642653
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, Davis
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Fragile X Syndrome
AFQ056 for Language Learning in Children With FXS
The purpose of this clinical trial is to investigate the impact of AFQ056 on language learning in 3-6 year old children with Fragile X Syndrome (FXS).
- Trial name
- FX-LEARN (NCT02920892)
- Testing
- AFQ056, Language Intervention
- Where
- 14 sites in United States
- Volunteers
- 110 people
- Sponsor
- Elizabeth Berry-Kravis
Common Variable Immunodeficiency, Agammaglobulinemia
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period.
- Trial name
- NCT00520494
- Testing
- Vivaglobin
- Where
- 6 sites in Canada, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma
T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts
Subjects are being asked to participate in this study because treatment of their disease requires them to receive a stem cell transplant.
- Conditions
- Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma, Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Trial name
- MOHEL (NCT00368355)
- Testing
- Ara-C, Cyclophosphamide, Campath-1H
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Baylor College of Medicine
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Kinky Hair Syndrome
Copper Histidine Therapy for Menkes Diseases
Menkes Disease is a genetic disorder affecting the metabolism of copper. Patient with this disease are both physically and mentally retarded. Menkes disease is usually first detected in the first 2-3 months of life.
- Trial name
- NCT00001262
- Testing
- Copper Histidine
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Rett Syndrome
Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
Dr. Sakkubai Naidu, Principal Investigator, is initiating a double blinded placebo controlled clinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT), at the Pediatric Clinical Research Unit (PCRU) of the Johns…
- Trial name
- PCTDMRTT (NCT01520363)
- Testing
- dextromethorphan
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Fragile X Syndrome
Trial of Sertraline to Treat Children With Fragile X Syndrome
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and…
- Trial name
- NCT01474746
- Testing
- Sertraline
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Randi J. Hagerman, MD
Barth Syndrome
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.
- Trial name
- TAZPOWER (NCT03098797)
- Testing
- Elamipretide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Stealth BioTherapeutics Inc.
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
This study is to discover whether children with severe combined immunodeficiency disease (SCID) or other primary immunodeficiency disorder (PID) for which no satisfactory treatment other than stem cell transplantation (SCT)…
- Conditions
- Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
- Trial name
- MASCI (NCT00579137)
- Testing
- Campath -1H, Fludarabine, Anti-CD45
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Fragile X Syndrome
Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
The study objective is to explore the efficacy, safety and tolerability of STX209 for treatment of irritability in subjects with FSX.
- Trial name
- NCT00788073
- Testing
- STX209
- Where
- 12 sites in United States
- Volunteers
- 63 people
- Sponsor
- Seaside Therapeutics, Inc.
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period.
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome
- Trial name
- NCT01289847
- Testing
- Gammaplex
- Where
- 10 sites in United States, Chile and 1 more
- Volunteers
- 25 people
- Sponsor
- Bio Products Laboratory
Rett Syndrome
Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
The investigators are recruiting children for a research study using a medication known as IGF-1 (mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT).
- Trial name
- NCT01253317
- Testing
- rhIGF-1
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Boston Children's Hospital
Rett Syndrome
Pharmacological Treatment of Rett Syndrome With Statins
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
- Trial name
- Statins (NCT02563860)
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Montefiore Medical Center
Fragile X Syndrome
A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome
The purpose of this study is to determine the effectiveness and tolerability of aripiprazole in the treatment of children and adolescents with Fragile X Syndrome.
- Trial name
- NCT00420459
- Testing
- Aripiprazole
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Indiana University
Rett Syndrome
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome in an initial double blind 24 week period followed by an open label treatment phase of up to 168 weeks…
- Trial name
- STARS (NCT02790034)
- Testing
- Sarizotan low dose, Sarizotan high dose
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 129 people
- Sponsor
- Newron Pharmaceuticals SPA
Fragile x Syndrome
Ganaxolone Treatment in Children With Fragile X Syndrome
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS).
- Trial name
- NCT01725152
- Testing
- Ganaxolone
- Where
- 2 sites in United States, Belgium
- Volunteers
- 59 people
- Sponsor
- Marinus Pharmaceuticals