Clinical trials: Congenital Malformation
Cystic Fibrosis
CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)
This is a Phase 2 study with primary objective of looking whether YPT-01 phage therapy reduces sputum bacterial load in cystic fibrosis subjects with Pseudomonas aeruginosa.
- Trial name
- NCT04684641
- Testing
- Standard Dose YPT-01
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
Atopic Dermatitis
Long-term Safety and Efficacy Study of Lebrikizumab (LY3650150) in Participants With Moderate-to-Severe Atopic Dermatitis (ADjoin)
This is designed to assess the long-term safety and efficacy of lebrikizumab for moderate-to-severe atopic dermatitis. It will last up to 33 months.
- Trial name
- ADjoin (NCT04392154)
- Testing
- Lebrikizumab
- Where
- 321 sites in United States, Australia and 14 more
- Volunteers
- 1,153 people
- Sponsor
- Eli Lilly and Company
Hemophilia B
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05630651
- Testing
- ZS801
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Alpha1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
- Trial name
- NCT04474197
- Testing
- VX-864
- Where
- 30 sites in United States, Canada and 4 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
AV Fistula
Tissue Markers Predictive of Early Stenosis in the Arterovenous Fistula for Hemodialysis
The study involves the recruitment of two populations. POPULATION 1 (surgical sample of excess venous segment): at least 75 consecutive patients, enrolled according to the inclusion criteria and subjected to first AVF surgery as…
- Trial name
- FAV-2019 (NCT06815159)
- Testing
- Identification any tissue markers predictive or favoring early stenosis in AVF for haemodialysis.
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Atopic Dermatitis
A Study to Evaluate the Safety and Efficacy of AK120 in Subjects With Moderate to Severe Atopic Dermatitis
This is a multicenter, randomized, open label phase II clinical study to evaluate the safety and efficacy of AK120 in the treatment of subjects with moderate to severe atopic dermatitis.
- Trial name
- NCT06092762
- Testing
- AK120
- Where
- 26 sites in China
- Volunteers
- 180 people
- Sponsor
- Akeso
Primary Ciliary Dyskinesia, Healthy
Examining Bronchial Hyperresponsiveness in Primary Ciliary Dyskinesia
The purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness".
- Trial name
- BHR (NCT07288827)
- Testing
- Lung Function Testing, Phlebotomy, Allergy skin prick testing
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Indiana University
Colic, Nephropathy
Neonatal Outcome of Children With Antenatal Colic Hyperechogenicity
Fetal colic hyper-echogenicity is a presenting symptoms for cystinuria lysinuria. A few cases of fetuses with dibasic protein intolerance (more complex prognosis) presented with colic hyper-echogenicity antenatal.
- Trial name
- COLONECHOGENE (NCT04983069)
- Where
- France
- Volunteers
- 400 people
- Sponsor
- University Hospital, Montpellier
Retinitis Pigmentosa (RP)
Investigation of Therapeutic Efficacy and Safety of UMSCs for the Management of Retinitis Pigmentosa (RP)
Retinitis pigmentosa (RP) is the most common hereditary retinal disorder (accounts for 20% of children attending blind schools in Pakistan) which causes degeneration of rod and cone photoreceptors.
- Trial name
- NCT04763369
- Testing
- Injection of stem cells in sub-tenon space of eye for the management of retinitis pigmentosa, Injection of stem cells in suprachoroidal space of eye for the management of Retinitis Pigmentosa
- Where
- Pakistan
- Volunteers
- 50 people
- Sponsor
- Jinnah Burn and Reconstructive Surgery Centre, Lahore
Lactose Intolerance
Effects Comparison of A1 and A2 Milk on Gastrointestinal Physiology, Symptoms and Cognitive Behavior
This is a single-site, double-blinded, randomized, controlled, 2X2 cross-over study aiming to compare effects of milk containing only A2 type beta casein versus milk containing both A1 and A2 beta casein proteins on the…
- Trial name
- NCT02406469
- Testing
- Oral consumption milk Tetra Pak
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- a2 Milk Company Ltd.
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
To determine the clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic neurofibromatosis type 1 (NF1) associated or sporadic MPNST.
- Trial name
- NCT03433183
- Testing
- Selumetinib, Sirolimus
- Where
- 5 sites in United States
- Volunteers
- 21 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Retinitis Pigmentosa
Acupuncture for the Treatment of Vision Loss Due to Retinitis Pigmentosa
Retinitis Pigmentosa (RP) patients are interested in trying alternative therapies to attempt to slow, halt or reverse the retinal disease process, and claim success with some approaches such as acupuncture, but this potential…
- Trial name
- NCT01604356
- Testing
- Electro-acupuncture
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Johns Hopkins University
Cystic Fibrosis, Infection, Bacterial
Manuka Honey Sinus Rinse Study
A trial investigating the effect of a sinus rinse in people with CF
- Trial name
- NCT04589897
- Testing
- Manuka honey sinus rinse, Standard sinus rinse
- Where
- United Kingdom
- Volunteers
- 18 people
- Sponsor
- Swansea University
Solid Tumors
Dose Escalation of RMC-4630 Monotherapy in Relapsed/Refractory Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of escalating doses of RMC-4630 monotherapy in adult participants with relapsed/refractory solid tumors…
- Trial name
- NCT03634982
- Testing
- RMC-4630
- Where
- 12 sites in United States
- Volunteers
- 133 people
- Sponsor
- Revolution Medicines, Inc.
Neonatal Thrombocytopenia
Impact of Thrombocytopenia and Platelet Transfusions on Neonatal Bleeding and Inflammation
This is a prospective observational study that was designed with the following two Specific Aims: 1. To determine whether the Immature Platelet Fraction percentage (IPF%) and the Immature Platelet Count (IPC) are better…
- Trial name
- NCT03848923
- Where
- 2 sites in United States
- Volunteers
- 160 people
- Sponsor
- Boston Children's Hospital
Hemophilia B
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per…
- Trial name
- NCT05203679
- Testing
- Single dose intravenous injection of BBM-H901
- Where
- 9 sites in China
- Volunteers
- 32 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Mucopolysaccharidosis IVA, Morquio A Syndrome
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01609062
- Testing
- BMN 110
- Where
- 11 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- BioMarin Pharmaceutical
Retinitis Pigmentosa
Computer-Assisted Rehabilitation Environment Training After Argus Retinal Prosthesis
The goal of the current project is to fill the unmet clinical needs around the objective assessment of visual function and develop outcome-oriented visual rehabilitation approach using the computer assisted rehabilitation…
- Trial name
- NCT03444961
- Testing
- CAREN system training
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- The Cleveland Clinic
Fetal Growth Retardation
Non-invasive Placental Chromosome Exploration of Intrauterine Growth Restriction
The objective of this project is the non-invasive prenatal detection of placenta-limited aneuploidies, in patients whose fetuses have a intrauterine growth restriction below 3rd percentile, in parallel with an amniocentesis.
- Trial name
- DPNI-RCIU (NCT05023161)
- Testing
- Blood samples
- Where
- 3 sites in France
- Volunteers
- 300 people
- Sponsor
- University Hospital, Bordeaux
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