Clinical trials: Newborn; Anemia
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Hemochromatosis
Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients
Comparison of efficacy and toxicity of the combination treatment of deferiprone and desferrioxamine with the single agent treatment of either drug
- Trial name
- NCT00350662
- Testing
- Deferiprone (L1), Desferrioxamine
- Where
- 2 sites in Egypt, Turkey (Türkiye)
- Volunteers
- 95 people
- Sponsor
- Lipomed
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in Sickle Cell Disease (SCD) patients diagnosed with Pulmonary Hypertension.
- Trial name
- ASSET-2 (NCT00313196)
- Testing
- bosentan
- Volunteers
- 12 people
- Sponsor
- Actelion
Fanconi Anemia Complementation Group A
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
- Trial name
- NCT04069533
- Testing
- RP-L102
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Leukemia, Myelodysplastic Syndromes
3-AP and Cytarabine in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy such as cytarabine use different ways to stop cancer cells from dividing so they stop growing or die.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00064090
- Testing
- cytarabine, triapine
- Where
- United States
- Sponsor
- Vion Pharmaceuticals
Sickle Cell Disease
Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.
- Trial name
- NCT05725902
- Testing
- Etavopivat
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287086
- Testing
- BRL-101 autologous hematopoietic stem and progenitor cells injection
- Volunteers
- 1 people
- Sponsor
- Bioray Laboratories
Beta-Thalassemia
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe…
- Trial name
- NCT04938635
- Testing
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID
- Where
- 7 sites in United States, Bulgaria and 1 more
- Sponsor
- Vifor (International) Inc.
Beta-Thalassemia
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT).
- Trial name
- NCT05577312
- Testing
- BRL-101
- Where
- 7 sites in China
- Volunteers
- 39 people
- Sponsor
- Bioray Laboratories
Sickle Cell Disease
Voxelotor CYP and Transporter Cocktail Interaction Study
This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are…
- Trial name
- NCT05981365
- Testing
- Voxelotor, Bupropion, Repaglinide
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Pfizer
Thalassemia, Beta
Long-term Follow-up Study of BRL-101 for TDT
Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.
- Trial name
- NCT06298630
- Testing
- Assessments
- Where
- 7 sites in China
- Volunteers
- 45 people
- Sponsor
- Bioray Laboratories
Myelodysplastic Syndromes (MDS), Beta-thalassemia
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: Participants receiving luspatercept on a parent protocol at the time of their transition to the…
- Conditions
- Myelodysplastic Syndromes (MDS), Beta-thalassemia, Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
- Trial name
- NCT04064060
- Testing
- Luspatercept
- Where
- 143 sites in United States, Australia and 20 more
- Volunteers
- 665 people
- Sponsor
- Celgene
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia.
- Trial name
- PEAK Registry (NCT03481738)
- Where
- 52 sites in United States, Canada and 14 more
- Volunteers
- 500 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease, Sickle Cell Anemia
A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy
This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043.
- Trial name
- NCT00294541
- Testing
- ICA-17043
- Where
- 31 sites in United States
- Volunteers
- 51 people
- Sponsor
- Icagen
Sickle Cell Disease
Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell…
- Trial name
- NCT05470270
- Testing
- Hydroxycarbamide
- Where
- 3 sites in France
- Volunteers
- 33 people
- Sponsor
- Theravia
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Sickle Cell Disease
Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab.
- Trial name
- NCT05833022
- Where
- 11 sites in United States
- Volunteers
- 297 people
- Sponsor
- Novartis Pharmaceuticals
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Prostatic Neoplasms
Prevalence of HRR-related Genes Mutations and Prognosis in Metastatic Castration Resistant Prostate Cancer (mCRPC) Patients in Real World Setting
The purpose of this study is to investigate the prevalence of tissue homologous recombination repair (HRR)-related gene mutations (positive/negative/Variant of uncertain significance (VUS)), clinical outcome such as…
- Trial name
- ZENSHIN (NCT04425200)
- Where
- 26 sites in Japan
- Volunteers
- 205 people
- Sponsor
- AstraZeneca