Clinical trials: Genetic Diseases
Down Syndrome (Trisomy 21), Edwards Syndrome (Trisomy 18)
Non-Invasive Screening for Fetal Aneuploidy: A New Maternal Plasma Marker
Validate that circulating cell free fetal nucleic acid can be used to identify a direct marker for fetal aneuploidy, particularly fetal Down Syndrome (DS), that is better than surrogate markers.
- Conditions
- Down Syndrome (Trisomy 21), Edwards Syndrome (Trisomy 18), Patau Syndrome (Trisomy 13), Turner Syndrome
- Trial name
- NCT00770458
- Where
- 7 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Sequenom, Inc.
Dermatitis Atopic
A Study to Investigate the Efficacy and Safety of Subcutaneous Lunsekimig (SAR443765) Compared With Placebo in Adult Participants With Moderate-to-severe Atopic Dermatitis
This is a parallel, Phase 2b, randomized, double-blind, placebo-controlled, multicenter study to assess the efficacy, safety, and tolerability of lunsekimig monotherapy in adult participants (aged 18 to 80 years, inclusive) with…
- Trial name
- NCT06790121
- Testing
- Lunsekimig
- Where
- 52 sites in United States, Czechia and 2 more
- Volunteers
- 150 people
- Sponsor
- Sanofi
Gout, Arthritis, Gouty
Phase 2 to Assess Efficacy and Safety in AR882 Alone or in Combination With Allopurinol in Patients With Tophaceous Gout
This study will assess the serum urate lowering effect, tophi reduction, and safety of AR882 alone and in combination with allopurinol in patients with tophaceous gout at two doses compared to allopurinol over 24 weeks.
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Trial name
- NCT05253833
- Testing
- AR882 Dose 1, AR882 Dose 2, Allopurinol Tablet
- Where
- 12 sites in United States, New Zealand and 1 more
- Volunteers
- 42 people
- Sponsor
- Arthrosi Therapeutics
Atopic Dermatitis
Evaluation of the Efficacy of a Cosmetic Care in Subjects With Moderate to Severe Atopic Dermatitis, Treated by Systemic Treatments
Atopic dermatitis (AD) is a common chronic inflammatory skin disease, affecting both children (15- 20%) and adults (2-5%). The pathogenesis of AD is complex and multifactorial, including skin barrier dysfunction and immune…
- Trial name
- NCT07577921
- Testing
- Investigational cosmetic product RV4427C
- Where
- France
- Volunteers
- 80 people
- Sponsor
- Pierre Fabre Dermo Cosmetique
To Determine Bioequivalence Under Fed Conditions
Bioavailability Study of Doxycycline Monohydrate Capsules and Monodox Under Fasting and Fed Conditions
To compare the rate and extent of absorption of doxycycline monohydrate capsules equivalent to 100 mg doxycycline (Par) versus Monodox (Oclassen Pharmaceuticals)
- Trial name
- NCT00652704
- Testing
- Doxycycline monohydrate, Monodox
- Where
- Canada
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Genetic Disorder, Turner Syndrome
Use of Somatropin in Turner Syndrome
This trial is conducted in Europe. The aim of this trial is to study the dose-response relationship and effect of somatropin (Norditropin®) on final height in girls with Turner Syndrome.
- Trial name
- NCT01518036
- Testing
- somatropin
- Where
- United Kingdom
- Volunteers
- 57 people
- Sponsor
- Novo Nordisk A/S
Niemann-Pick Disease, Type C1
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion…
- Trial name
- NCT03893071
- Testing
- Hydroxypropyl-β-cyclodextrin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Lennox Gastaut Syndrome, Dravet Syndrome
Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- EpiTALY (NCT05485831)
- Testing
- Epidiolex 100 mg/mL Oral Solution
- Where
- 18 sites in Italy
- Volunteers
- 111 people
- Sponsor
- Jazz Pharmaceuticals
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Cystic Fibrosis, Pseudomonas Aeruginosa
Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas Aeruginosa Lung Infections and Cystic Fibrosis
Phase 1b/2a, double-blind, randomized, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability and phage recovery profile of AP-PA02 multi-bacteriophage therapeutic candidate administered…
- Conditions
- Cystic Fibrosis, Pseudomonas Aeruginosa, Pseudomonas, Lung Infection
- Trial name
- SWARM-Pa (NCT04596319)
- Testing
- AP-PA02
- Where
- 20 sites in United States
- Volunteers
- 29 people
- Sponsor
- Armata Pharmaceuticals, Inc.
Urea Cycle Disorders
Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
This clinical food study aims to explore the effect of KB195, a novel mixture of oligosaccharides, on the metabolism of nitrogen by the microbiome in patients with urea cycle disorders (UCDs).
- Trial name
- NCT03797131
- Testing
- KB195 (a novel mixture of oligosaccharides)
- Where
- Switzerland
- Volunteers
- 4 people
- Sponsor
- Kaleido Biosciences
Burns
Fish Skin Compared to Cadaver Skin as Temporary Cover for Full Thickness Burns
Objectives and rationale: Optimal burn management involves removing all the dead or burned tissue as early as feasible and cover with an autograft called split thickness skin graft (STSG) taken from the patient.
- Trial name
- Kereburn (NCT03984331)
- Testing
- Fish skin, Cadaver skin
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Kerecis Ltd.
Hemophilia A, Hemophilia B
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior…
- Trial name
- NCT05568719
- Testing
- Testing of hepatic AAV Vector integration
- Where
- 39 sites in United States, Australia and 11 more
- Volunteers
- 173 people
- Sponsor
- Pfizer
Hemophilia
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA)…
- Trial name
- ATLAS-PPX (NCT03549871)
- Testing
- Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 80 people
- Sponsor
- Genzyme, a Sanofi Company
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia.
- Trial name
- teACH (NCT06732895)
- Testing
- Navepegritide
- Where
- 5 sites in Canada, Denmark and 3 more
- Volunteers
- 25 people
- Sponsor
- Ascendis Pharma A/S
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How Safe the Study Treatment Elinzanetant is and How it Moves Into, Through and Out of the Body When Given as Single Increasing Doses That Are Higher Than Normally Used Compared to Placebo and Moxifloxacin in Healthy Participants
Researchers are looking for a better way to treat vasomotor symptoms, a condition of having hot flashes. The condition is caused by hormonal changes and occurs primarily in women, but can also affect men.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05481528
- Testing
- Moxifloxacin, Elinzanetant (BAY3427080)
- Where
- Germany
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Bayer
Severe Hemophilia A
rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study
The study is designed as a randomized, two-armed, double-blind, single-dose, crossover, two-sequence, active-controlled, multi-center, bioequivalence clinical trial with a primary endpoint of dose-normalized area under the curve…
- Trial name
- NCT06137092
- Testing
- Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)
- Where
- 5 sites in Iran
- Volunteers
- 50 people
- Sponsor
- AryoGen Pharmed Co.
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia…
- Trial name
- NCT05447494
- Testing
- Low-dose CAN103, High-dose CAN103
- Where
- China
- Volunteers
- 40 people
- Sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.
Primary Ciliary Dyskinesia (PCD)
Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance
This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
- Trial name
- NCT06633757
- Testing
- RCT1100
- Where
- 3 sites in United States, Denmark and 1 more
- Volunteers
- 14 people
- Sponsor
- ReCode Therapeutics
Hemophilia A
A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480)
- Trial name
- NCT03061201
- Testing
- SB-525 (PF-07055480)
- Where
- 22 sites in United States
- Volunteers
- 13 people
- Sponsor
- Pfizer
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