Clinical trials: Newborn Diseases
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Atopic Dermatitis
A Study of Two Different Formulations of 611 in Healthy Adult Subjects in China
The main purpose of this study is to compare two different formulations (mixtures) of 611 in healthy participants. This study will compare how much of each formulation gets into the blood stream.
- Trial name
- 611 (NCT07042113)
- Testing
- new formulations of 611, existing formulations of 611
- Where
- 2 sites in China
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd.
Dermatitis, Atopic
A Study of BMS-986166 or Branebrutinib for the Treatment of Participants With Atopic Dermatitis
The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986166 and of branebrutinib, each versus placebo, for the treatment of participants with moderate to severe atopic dermatitis.
- Trial name
- NCT05014438
- Testing
- BMS-986166, Branebrutinib
- Where
- 42 sites in United States, Australia and 5 more
- Volunteers
- 17 people
- Sponsor
- Bristol-Myers Squibb
Alpha 1-Antitrypsin Deficiency
The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency
The objective of this study is to prospectively assess whether there is any interval between first symptom and initial diagnosis that is experienced by patients with newly diagnosed alpha-1 antitrypsin deficiency (AATD) and then…
- Trial name
- NCT03039335
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Biocerna LLC
Polycystic Ovary Syndrome
Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome
The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women.
- Trial name
- PCOS (NCT03152591)
- Testing
- LIK066
- Where
- 5 sites in United States, Germany
- Volunteers
- 29 people
- Sponsor
- Novartis Pharmaceuticals
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Cystic Fibrosis
Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- TIS (NCT01270347)
- Testing
- MP-376 (Levofloxacin Solution for Inhalation), TIS (Tobramycin Inhalation Solution)
- Where
- 130 sites in United States, France and 4 more
- Volunteers
- 282 people
- Sponsor
- Amgen
Down Syndrome, Fetal Aneuploidy
Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy
The specimen collection is designed for the purpose of the development of a noninvasive prenatal test for T21.
- Trial name
- NCT01429389
- Where
- 14 sites in United States, Canada
- Volunteers
- 2,000 people
- Sponsor
- Sequenom, Inc.
Atopic Dermatitis
Tapinarof for the Treatment of Atopic Dermatitis in Children and Adults
This is a double-blind, randomized, vehicle controlled Phase 3 study to evaluate the efficacy and safety of topical tapinarof cream, 1% compared to vehicle control cream in pediatric and adult subjects with atopic dermatitis.
- Trial name
- NCT05014568
- Testing
- tapinarof cream, 1%, Vehicle cream
- Where
- 67 sites in United States, Canada
- Volunteers
- 407 people
- Sponsor
- Organon and Co
Phenylketonuria
Sapropterin Expanded Access Program
The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded…
- Trial name
- NCT00484991
- Testing
- Sapropterin dihydrochloride
- Where
- 36 sites in United States
- Sponsor
- BioMarin Pharmaceutical
Dermatitis, Atopic
Long-Term Safety Follow-Up Study of SCM-AGH in Subjects Who Participated and Administered SCM-AGH in to Severe Atopic Dermatitis Clinical Trials
This is a long-term safety follow-up study of the Phase I/II multicenter study of SCM-AGH in subjects with moderate to severe atopic dermatitis.
- Trial name
- NCT04185584
- Testing
- Not Applicable (Observational Study)
- Where
- South Korea
- Volunteers
- 92 people
- Sponsor
- SCM Lifescience Co., LTD.
Hyperlipoproteinemia Type II, Homozygous Familial Hypercholesterolemia
Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)
This study will evaluate the safety and effect of anacetrapib on low-density lipoprotein-cholesterol (LDL-C) when added to ongoing lipid-lowering therapy.
- Trial name
- NCT01841684
- Testing
- Anacetrapib
- Volunteers
- 2 people
- Sponsor
- Merck Sharp & Dohme LLC
Fetal Growth Retardation
Study Aimed At Improving Height With Genotonorm In Children Born Little And/Or Light With Growth Retardation At The Age
To estimate the percentage of children with serum IGF-1 > 2 standard deviation (compared to a child of the same gender and age and without growth hormone (GH) deficiency) 9 months and 12 months after initiation of GH treatment.
- Trial name
- NCT00174252
- Testing
- Genotonorm (Somatropin)
- Where
- 22 sites in France
- Volunteers
- 57 people
- Sponsor
- Pfizer
Sickle Cell Disease
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD).
- Trial name
- NCT04293185
- Testing
- bb1111
- Where
- 9 sites in United States
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
Atopic Dermatitis (AD), SCORAD Index (15-25)
In-Use Tolerance Study Under Dermatological and Pediatric Controls of Sunscreen in Children With Atopic Dermatitis
Atopic dermatitis is a chronic skin condition requiring sun protection, yet research on sunscreen effectiveness and skin tolerance in Atopic Dermatitis is limited.
- Trial name
- NCT07194408
- Testing
- Sunscreen RV4620A RP2552
- Where
- Mauritius
- Volunteers
- 22 people
- Sponsor
- Pierre Fabre Dermo Cosmetique
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
Retinitis Pigmentosa
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa…
- Trial name
- ABACUS-2 (NCT06628947)
- Testing
- 100 μg KIO-301, 50 μg KIO-301
- Where
- 5 sites in Australia
- Volunteers
- 36 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design.
- Trial name
- TRANSCEND (NCT07257978)
- Testing
- NTI164
- Where
- Australia
- Volunteers
- 40 people
- Sponsor
- Fenix Innovation Group
Heterozygous or Homozygous Familial Hypercholesterolemia
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301)…
- Trial name
- V-PEDS-OLE (NCT05682378)
- Testing
- Inclisiran
- Where
- 52 sites in United States, Argentina and 24 more
- Volunteers
- 195 people
- Sponsor
- Novartis Pharmaceuticals
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