Clinical trials: Autosomal Recessive Disorder
- Condition Autosomal Recessive Disorder
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Common Variable Immunodeficiency, X-linked Agammaglobulinemia
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
- Conditions
- Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Autosomal Recessive Agammaglobulinemia
- Trial name
- NCT00542997
- Testing
- Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)
- Where
- 19 sites in France, Germany and 7 more
- Volunteers
- 51 people
- Sponsor
- CSL Behring
Myocardial Infarction
Study to Evaluate the Safety and Activity of BB3 to Treat Heart Attack
The study will evaluate the effect of BB3 to preserve myocardial (heart) tissue and function following myocardial infarction (heart attack).
- Trial name
- NCT01539590
- Testing
- BB3, Normal saline
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Angion Biomedica Corp
Colorectal Neoplasms
Study of Tepotinib Combined With Cetuximab in Participants With Left-Sided RAS/BRAF Wild Type Metastatic Colorectal Cancer (PERSPECTIVE)
The purpose of this study was to assess the preliminary antitumor activity, safety and tolerability of tepotinib in combination with cetuximab in participants with RAS/BRAF wild-type left-sided Metastatic Colorectal Cancer (mCRC)…
- Trial name
- NCT04515394
- Testing
- Tepotinib, Cetuximab
- Where
- 65 sites in United States, Belgium and 6 more
- Volunteers
- 3 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Lamellar Ichthyosis
A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis
This is a phase 2 randomized, multi-center, double-blind, vehicle controlled, 90 day, safety, efficacy, and systemic exposure study followed by a 90 day open-label extension of trifarotene cream in adults and adolescents with…
- Trial name
- NCT03738800
- Testing
- CD5789 Cream 200 µg/g, CD5789 Cream 100 µg/g, CD5789 Cream Vehicle
- Where
- 36 sites in United States, Australia and 7 more
- Volunteers
- 65 people
- Sponsor
- Mayne Pharma International Pty Ltd
Obstructive Sleep Apnea Syndrome
Brain Oxygenation and Hemodynamics During Sleep in Obstructive Sleep Apnea Sufferers
The goal of this trial is to assess the performance of the OxiplexTS-an absolute near-infrared oximeter-as an instrument to measure brain oxygenation and hemodynamics in sleep medicine as well as in the broader field of…
- Trial name
- NIRS-OSAS (NCT00591591)
- Testing
- OxiplexTS
- Where
- 4 sites in United States
- Volunteers
- 309 people, healthy volunteers welcome
- Sponsor
- ISS, Inc.
Stargardt's Macular Dystrophy
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).
- Trial name
- NCT03364153
- Testing
- avacincaptad pegol
- Where
- 41 sites in United States, France and 6 more
- Volunteers
- 121 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Parkinson Disease Dementia, Dementia With Lewy Bodies
ATH-1017 Treatment in Subjects With Parkinson's Disease Dementia or Dementia With Lewy Bodies (SHAPE Trial)
This study is designed to evaluate the safety and treatment effects of fosgonimeton (ATH-1017) in subjects with Parkinson's Disease Dementia or Dementia with Lewy Bodies, with a randomized treatment duration of 26 weeks.
- Trial name
- NCT04831281
- Testing
- ATH-1017
- Where
- 10 sites in United States
- Volunteers
- 28 people
- Sponsor
- LeonaBio