Clinical trials

-
RAN proteins in Ataxia -
Respiratory training in Friedreich's ataxia - Investigator
- Carmen Leon Astudillo
-
Stimulation-induced ataxia in essential tremor - Investigator
- Adolfo Ramirez-Zamora
-
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as…
- Investigators
- Matthew R Burns, Sub Subramony
- Ages
- 6 Years - N/A
- Sexes
- All
-
JIVE (LARIMAR) To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).
- Investigator
- Sub Subramony
- Ages
- 18 Years - N/A
- Sexes
- All
-
Frataxin The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA). The gene mutation in FRDA leads to low levels of…
- Investigator
- Sub Subramony
- Ages
- 18 Years - 65 Years
- Sexes
- All
-
TRACK-FA This is a natural history study prospectively investigating neuroimaging markers of disease progression in children and adults with Friedreich ataxia (FA). There will be three assessment periods (baseline, 12 and 24 months). The study will include…
- Ages
- 5 Years - N/A
- Sexes
- All
-
ROR-PH-303 (APD811-303) Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.
- Investigator
- Ali Ataya
- Ages
- 18 Years - N/A
- Sexes
- All
-
Sapphire This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.
- Investigator
- Ali Ataya
- Ages
- 18 Years - 99 Years
- Sexes
- All
-
Prosera The primary objective of the study is to determine the effect of seralutinib on improving exercise capacity in subjects with WHO Group 1 PAH who are FC II or III. The secondary objective for this trial is to determine time to clinical worsening.
- Investigator
- Ali Ataya
- Ages
- 18 Years - 75 Years
- Sexes
- All