Clinical trials: Lymphatic Malformations
Renal Angiomyolipoma
Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).
- Trial name
- NCT03525834
- Testing
- Everolimus
- Where
- 5 sites in China
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Pregnant Individuals, Aneuploidy
The ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) Study is the Largest U.S.-Based Prospective Study Demonstrating a Circulating Fetal Cell (CFC) Based Approach to Non-invasive Fetal Risk Assessment
The goal of the ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) study is to compare the concordance of results of a novel non-invasive circulating fetal cell (CFC) assay to the results of…
- Conditions
- Pregnant Individuals, Aneuploidy, Down Syndrome (Trisomy 21), 22q11.2 Deletion Syndrome
- Trial name
- ADVANCE (NCT07643896)
- Where
- 9 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- BillionToOne Inc.
Complete DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Safety-Efficacy
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection.
- Conditions
- Complete DiGeorge Anomaly, DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT01220531
- Testing
- Cultured Thymus Tissue
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Lymphoma
Study Evaluating Duvelisib in Japanese Subjects With Relapsed or Refractory Lymphoma
This study seeks to evaluate the safety and pharmacokinetics of duvelisib in Japanese participants with relapsed or refractory lymphoma.
- Trial name
- NCT02598570
- Testing
- duvelisib
- Where
- 3 sites in Japan
- Volunteers
- 7 people
- Sponsor
- AbbVie
Microcystic Lymphatic Malformation
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
- Trial name
- NCT05050149
- Testing
- PTX-022
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Palvella Therapeutics, Inc.
Follicular Lymphoma
Study of Duvelisib in Combination With Rituximab vs Rituximab in Subjects With Previously Treated Follicular Lymphoma
A study to evaluate the safety and efficacy of duvelisib administered in combination with rituximab vs placebo in combination with rituximab in patients with previously treated CD20-positive follicular lymphoma who are not…
- Trial name
- DYNAMO + R (NCT02204982)
- Testing
- Duvelisib, Rituximab
- Where
- 5 sites in Australia, France and 2 more
- Volunteers
- 13 people
- Sponsor
- SecuraBio
Down Syndrome, Edwards Syndrome
Whole Blood Specimen Collection From Pregnant Subjects
To obtain whole blood specimens from pregnant subjects to be used for research and development and clinical validation studies of prenatal assays.
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Klinefelter Syndrome
- Trial name
- NCT02430584
- Testing
- Observational - no intervention
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Progenity, Inc.
Cerebral Cavernous Malformation
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in participants with symptomatic…
- Trial name
- SYCAMORE (NCT05085561)
- Testing
- REC-994
- Where
- 15 sites in United States
- Volunteers
- 62 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Hereditary Hemorrhagic Telangiectasia (HHT)
Phase 1b Study of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT).
- Trial name
- NCT07863609
- Testing
- TX002100
- Volunteers
- 20 people
- Sponsor
- Tectonic Operating Company, Inc.
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Dose in DiGeorge #932
One purpose of this study is to determine whether the amount of cultured thymus tissue implanted into DiGeorge anomaly infants has any effect on the immune outcome.
- Conditions
- DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT00576836
- Testing
- Cultured Thymus Tissue Implantation (CTTI), Cultured Thymus Tissue Implantation with Parathyroid Transplantation
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Hereditary Hemorrhagic Telangiectasia
A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
The primary objective of this trial is to assess the efficacy of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in participants with…
- Trial name
- NCT07743671
- Testing
- Engasertib
- Where
- 4 sites in United States
- Volunteers
- 240 people
- Sponsor
- Vaderis Therapeutics AG
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
This is a 2-part study evaluating ATV-1601 in participants with moderate to severe HHT. Part 1 is a randomized, double-blind, placebo-controlled study evaluating 3 dosing regimens of ATV-1601.
- Trial name
- NCT07601425
- Testing
- ATV-1601
- Where
- 7 sites in United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Atavistik Bio, Inc
Hereditary Hemorrhagic Telangiectasia (HHT)
A Trial of TER-1754 in Patients With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of TER-1754 (a novel AKT1 inhibitor) in patients with HHT.
- Trial name
- NCT07255846
- Testing
- TER-1754
- Where
- United States
- Sponsor
- Terremoto Biosciences Inc.
22q11.2 Deletion Syndrome
Safety and Efficacy Study of NFC-1 in Subjects Aged 12-17 Years With 22q11.2DS & Associated Neuropsychiatric Conditions
This is a 5-week, multi-center, open-label, dose optimization trial in subjects aged 12-17 years with 22q11DS who have a diagnosis of anxiety disorder, and/or ADHD, and/or ASD.
- Trial name
- NCT02895906
- Testing
- NFC-1
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
Hereditary Hemorrhagic Telangiectasia (HHT)
Assess Safety and Efficacy of VAD044 in HHT Patients
Part I: The purpose of this Phase 1b proof of concept study, randomised, placebo controlled, double blind, multicentre study is to asssess safety and efficacy of 2 doses of VAD044 in adult HHT patients.
- Trial name
- NCT05406362
- Testing
- VAD044 Part I, VAD044 Part II
- Where
- 7 sites in United States, Belgium and 4 more
- Volunteers
- 75 people
- Sponsor
- Vaderis Therapeutics AG
Autoimmune Hemolytic Anemia
A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that…
- Trial name
- NCT03538041
- Testing
- Parsaclisib
- Where
- 12 sites in United States, Austria and 2 more
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation
MPN (Myeloproliferative Neoplasms)
A Study of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis
The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of parsaclisib and ruxolitinib in subjects with myelofibrosis.
- Trial name
- NCT02718300
- Testing
- Parsaclisib, Ruxolitinib
- Where
- 39 sites in United States
- Volunteers
- 74 people
- Sponsor
- Incyte Corporation
Skin Barrier to Water Loss, Skin Health
Apple Peptide Complex for Post-Surgical and Aesthetic Skin Recovery
This clinical study evaluates a topical botanical formulation for supporting skin recovery following invasive and non-invasive aesthetic procedures.
- Conditions
- Skin Barrier to Water Loss, Skin Health, Skin Discoloration, Skin Irritation
- Trial name
- APC-SKIN (NCT07807085)
- Testing
- Apple Peptide Complex Post-Aesthetic Care Protocol
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Grow Out Oils Clinical Aromatherapy Company
22Q11.2 Deletion Syndrome
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to <18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
- Trial name
- INSPIRE (NCT05149898)
- Testing
- ZYN002
- Where
- 3 sites in United States, Australia
- Volunteers
- 20 people
- Sponsor
- Harmony Biosciences Management, Inc.