Clinical trials: NEWBORN
Lesch-Nyhan Disease, Self-injurious Behavior
Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness…
- Trial name
- NCT01751802
- Testing
- Ecopipam
- Where
- 2 sites in United States, Spain
- Volunteers
- 9 people
- Sponsor
- Emalex Biosciences Inc.
Advanced Retinitis Pigmentosa
AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa
All participants in phase 1 and phase 2a had hand motion visual acuity or worse. If efficacy was demonstrated from phase 1, better vision subjects could be enrolled; however, efficacy was not demonstrated.
- Trial name
- NCT02556736
- Testing
- AGN-151597
- Where
- 4 sites in United States
- Volunteers
- 14 people
- Sponsor
- AbbVie
Neonatal Death, Perinatal Death
Leveraging Interactive Text Messaging to Monitor and Support Maternal Health in Kenya
Mobile health (mHealth) interventions such as interactive short message service (SMS) text messaging with healthcare workers (HCWs) have been proposed as efficient, accessible additions to traditional health care in…
- Conditions
- Neonatal Death, Perinatal Death, Depression
- Trial name
- AI-NEO (NCT05369806)
- Testing
- Interactive two-way SMS dialogue
- Where
- 2 sites in Kenya
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University of Washington
Atopic Dermatitis
Lebrikizumab Pen Ease of Use in Participants With Atopic Dermatitis
The purpose of this study is to assess the ease of use of the lebrikizumab pen. Participants will use a practice pad to simulate administration of a dose.
- Trial name
- NCT06444165
- Testing
- Lebrikizumab Pen
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- Eli Lilly and Company
Colic, Inflammation
Effect of Lactobacillus Rhamnosus GG (LGG) on Infant Colic
This study will compare 2 currently marketed formulas in healthy full term babies: Nutramigen A+ (a hypoallergenic formula) and Nutramigen-Enflora (hypoallergenic formula with Lactobacillus GG (LGG)) during 3 months of formula…
- Trial name
- LGG (NCT01279265)
- Testing
- Nutramigen with Enflora, Nutramigen A+
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Meconium Aspiration
Phase III Randomized Study of Lucinactant in Full Term Newborn Infants With Meconium Aspiration Syndrome
OBJECTIVES: Evaluate the safety and efficacy of lucinactant administered by bronchoalveolar lavage (BAL) in the treatment of meconium aspiration syndrome (MAS) in newborn infants.
- Trial name
- NCT00004500
- Testing
- Lucinactant, Standard Care
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Windtree Therapeutics
Desmoplastic/Nodular Medulloblastoma, Medulloblastoma
Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma
This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma.
- Conditions
- Desmoplastic/Nodular Medulloblastoma, Medulloblastoma, Medulloblastoma With Extensive Nodularity, Nevoid Basal Cell Carcinoma Syndrome
- Trial name
- NCT02017964
- Testing
- Carboplatin, Cognitive Assessment, Cyclophosphamide
- Where
- 173 sites in United States, Australia and 2 more
- Volunteers
- 26 people
- Sponsor
- Children's Oncology Group
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Tolvaptan Extension Study in Participants With ADPKD
To demonstrate whether tolvaptan modifies ADPKD progression as measured by changes from Baseline (from Study 156-04-251) in total kidney volume (TKV) and renal function.
- Trial name
- TEMPO 4/4 (NCT01214421)
- Testing
- Tolvaptan
- Where
- 97 sites in United States, Argentina and 11 more
- Volunteers
- 1,083 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study.
- Trial name
- NCT00630747
- Testing
- Idursulfase
- Where
- 52 sites in United States, Brazil and 8 more
- Volunteers
- 94 people
- Sponsor
- Shire
Polycystic Kidney, Autosomal Dominant
Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease
This study will test to see if metformin is safe and if it is tolerated compared to placebo in adult Autosomal Dominant Polycystic Kidney Disease (ADPKD) patients with beginning stages of chronic kidney disease.
- Trial name
- TAME (NCT02656017)
- Testing
- Metformin
- Where
- 2 sites in United States
- Volunteers
- 97 people
- Sponsor
- Kyongtae Ty Bae, M.D., Ph.D.
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Prader-Willi Syndrome
A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome
The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.
- Trial name
- NCT03458416
- Testing
- Cannabidiol Oral Solution
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Myeloid Leukemia in Remission
Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
This phase II trial studies how well decitabine and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide works in treating patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01707004
- Testing
- decitabine, fludarabine phosphate, busulfan
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Wisconsin, Madison
Huntington Disease
Pilot Study of Minocycline in Huntington's Disease
This study is being conducted to assess the impact of minocycline on the progression of symptoms of HD. The study will also assess whether it is reasonable to continue with further study of minocycline in HD.
- Trial name
- NCT00277355
- Testing
- minocycline
- Where
- 12 sites in United States, Canada
- Volunteers
- 114 people
- Sponsor
- Merit Cudkowicz
Cystic Fibrosis
Aztreonam Lysine for Pseudomonas Infection Eradication Study
This is an open-label, multi-center study in pediatric patients age 3 months to less than 18 years with cystic fibrosis (CF) and newly detected Pseudomonas aeruginosa (PA) pulmonary colonization/infection.
- Trial name
- ALPINE (NCT01375049)
- Testing
- Aztreonam for Inhalation Solution (AZLI)
- Where
- 58 sites in United States, Austria and 7 more
- Volunteers
- 105 people
- Sponsor
- Gilead Sciences
Intracranial Arteriovenous Malformations (AVM)
Protocol for Staged Stereotactic Radiosurgery for Large Arteriovenous Malformations
Stereotactic radiosurgery is a well established treatment option for arteriovenous malformations (AVMs). The potential complications related to radiosurgery are well documented and are predominately related to radiation effects…
- Trial name
- NCT02576535
- Testing
- Fractionated stereotactic radiosurgery, Leskell gamma unit
- Volunteers
- 18 people
- Sponsor
- NYU Langone Health
X-linked Hypophosphatemia
Examining the Effect of Burosumab on Muscle Function
Patients with X-linked hypophosphatemia (XLH) often report symptoms of fatigue and weakness particularly after exertion, in addition to their skeletal complaints.
- Trial name
- NCT04146935
- Testing
- Burosumab Injection [Crysvita]
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Yale University
Cirrhosis, Congenital Heart Disease
Role of Sildenafil for Fontan Associated Liver Disease (SiFALD) Study
The purpose of this study is to determine if the medication, sildenafil (also known as Revatio), can slow or stop the progression of liver disease in patients who previously had a Fontan operation.
- Trial name
- SiFALD (NCT03154476)
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mayo Clinic
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447993
- Testing
- Low Dose NT-501, High Dose NT-501
- Where
- 11 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurotech Pharmaceuticals
Cystic Fibrosis
Supplementation of Oral Reduced Glutathione in Pediatric Cystic Fibrosis Patients
Many individuals with cystic fibrosis experience growth failure. The reasons are not clear, but inflammation of the gut in these patients seems to be one important reason.
- Trial name
- NCT02029521
- Testing
- Oral reduced l-glutathione
- Where
- Italy
- Volunteers
- 47 people
- Sponsor
- Clark Bishop
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