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Haemophilia and Octostim

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Haemophilia Symptoms and Causes

Hemophilia is a rare disorder in which the blood does not clot normally. It is usually inherited. Hemophilia usually occurs in males.

If you have hemophilia, you have little or no clotting factor. Clotting factor is a protein needed for normal blood clotting. Without it, you may bleed for a long time after an injury or accident. You also may bleed into your knees, ankles, and elbows. Bleeding in the joints causes pain and, if not treated, can lead to arthritis. Bleeding in the brain, a very serious complication of hemophilia, requires emergency treatment.

The main symptoms of hemophilia are excessive bleeding and easy bruising. Blood tests can tell if you have it. The main treatment is injecting the missing clotting factor into the bloodstream. You may need it on a regular basis, or just when bleeding occurs.

NIH: National Heart, Lung, and Blood Institute

Check out the latest treatments for haemophilia

haemophilia treatment research studies

Octostim clinical trials, surveys and public health registries


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Haemophilia Clinical Trials and Studies

Treatments might be new drugs or new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments. The goal of clinical trials is to determine if a new test or treatment works and is safe. Clinical trials can also look at other aspects of care, such as improving the quality of life for people with chronic illnesses. People participate in clinical trials for a variety of reasons. Healthy volunteers say they participate to help others and to contribute to moving science forward. Participants with an illness or disease also participate to help others, but also to possibly receive the newest treatment and to have the additional care and attention from the clinical trial staff.
Rank Status Study
1 Recruiting The Effect of Gender on Antidiuresis - Evaluated by Graded Low Dose Desmopressin Infusion
Conditions: Gender Difference in V2 Receptor Function in Response to dDAVP Infusion;   Nocturia;   Nocturnal Enuresis
Intervention: Drug: dDAVP infusion
Outcome Measures: Urine osmolality;   Urine Volume;   Plasma dDAVP;   Plasma sodium
2 Recruiting Analgesic Efficacy of Intranasal Desmopressin in Acute Renal Colic
Conditions: Renal Colic;   Pain
Interventions: Drug: Desmopressin intranasal spray;   Drug: Placebo intranasal spray;   Drug: Indomethacin suppository
Outcome Measures: Pain intensity;   Side effects
3 Not yet recruiting DDAVP in the Reduction of Post-operative Ecchymosis in Rhinoplasty
Condition: Postoperative Ecchymosis
Intervention: Drug: DDAVP
Outcome Measures: Reduction in Ecchymosis and Swelling;   Patient Behavior Factors
4 Unknown  Clinical Trial of Desmopressin on Nocturia in Obstructive Sleep Apnoea Patients
Conditions: Nocturia;   Obstructive Sleep Apnoea
Interventions: Drug: Desmopressin;   Drug: Placebo
Outcome Measures: Change in no. of nocturia;   Side effects related to desmopressin;   Change in quality of Life - NQOL;   Change of quality of Sleep - PSQI;   Change in lower urinary tract symptoms - OABSS
5 Recruiting Desmopressin Melt Therapy in Patients With Nocturnal Polyuria: a Pharmacokinetic/Dynamic Study
Condition: Nocturnal Polyuria
Intervention: Drug: Desmopressin
Outcome Measures: Pharmacokinetic and dynamic evaluation of desmopressin melt for treatment of nocturnal polyuria in adults;   24h miction-incontinence-residue registration: urine collections every 3 hours;   Measurement of blood pressure during 24h
6 Not yet recruiting Desmopressin Melt Therapy in Nocturnal Polyuria Patients: Pharmacodynamic Study
Condition: Nocturia
Intervention: Drug: Desmopressin
Outcome Measures: Blood samples for plasma concentration of Na+, K+, creatinin, osmolality (safety profile)at day 3.;   Urine sample for urine concentration of Na+, K+, creatinin and osmolality;   Area Under Curve (AUC) frequency/volume chart during the first 14 days;   The decrease of number of nocturnal micturition episodes.;   The decrease of the number of nocturnal micturition episodes.;   Registration of number of side effects at day 3.;   Blood samples for plasma concentration of Na+, K+, creatinin, osmolality (safety profile)at day 7.;   Blood samples for plasma concentration of Na+, K+, creatinin, osmolality (safety profile)at day 30.;   Registration of number of side effects at day 7;   Registration of number of side effects at day 30.
7 Unknown  Desmopressin for Bleeding Related to Low Body Temperature
Condition: Hypothermia Induced Impairment of Primary Haemostasis
Interventions: Drug: desmopressin;   Drug: placebo
Outcome Measure: closure time on PFA-100
8 Recruiting Perioperative Use of Desmopressin (DDAVP) in Breast Cancer
Condition: Breast Cancer
Intervention: Drug: Desmopressin
Outcome Measures: Selection of the higher safe dose level for ensuing clinical trials;   Evidence of V2 Receptor Expression;   Evidence of CTC (CIRCULATING TUMOR CELLS);   Evidence of Von Willebrand factor antigen (VWF:Ag);   Evidence of CTC (circulating tumor cells);   Evidence of VWF activity (ristocetin cofactor, VWF:RCo);   Evidence of FVIII coagulant activity (FVIII:C);   Evidence of euglobulin lysis time
9 Unknown  Desmopressin (DDAVP) in Patients With Colorectal Cancer and Rectal Bleeding
Conditions: Colorectal Cancer;   Rectal Bleeding
Intervention: Drug: Desmopressin
Outcome Measures: Presence or absence of grade 3 or 4 adverse events related to the study drug, in a maximum of 2 out of 6 patients assessed in each dose level.;   Number of patients with grade 3 or 4 local adverse events;   Number of patients with grade 3 or 4 systemic adverse events;   Number of withdrawn from treatment;   Number of patients with partial or complete response in clinical endpoints
10 Recruiting Effects of Desmopressin on Blood Loss and the Quality of the Surgical Field During Endoscopic Sinus Surgery
Condition: Patients Undergoing Endoscopic Sinus Surgery
Intervention: Drug: Desmopressin
Outcome Measures: Blood loss;   Quality of the surgical field using a scoring scale
11 Recruiting Treatment of Supine Hypertension in Autonomic Failure
Condition: Hypertension
Interventions: Drug: Clonidine;   Drug: Nitroglycerin transdermal;   Drug: Dipyridamole/ Aspirin (Aggrenox);   Drug: Desmopressin (DDAVP);   Drug: Sildenafil;   Drug: Nifedipine;   Drug: Hydralazine;   Drug: Hydrochlorothiazide;   Drug: Placebo;   Drug: Bosentan;   Drug: Diltiazem;   Drug: Eplerenone;   Drug: guanfacine;   Dietary Supplement: L-arginine;   Drug: captopril;   Drug: carbidopa;   Drug: losartan;   Drug: metoprolol tartrate;   Drug: nebivolol hydrochloride;   Drug: prazosin hydrochloride;   Drug: tamsulosin hydrochloride;   Other: Head-up tilt.;   Drug: aliskiren;   Other: Local heat stress
Outcome Measures: Decrease in supine systolic blood pressure;   Decrease in pressure natriuresis
12 Recruiting Safety Profiles of Liver Biopsy in Hemodialysis Patients With Chronic Viral Hepatitis Pre-treated With Vasopressin
Conditions: Chronic Hepatitis C;   Chronic Hepatitis B;   Biopsy;   Hemodialysis
Intervention: Procedure: Percutaneous liver biopsy
Outcome Measures: Biopsy-related serious hemorrhage rate by intention-to-treat (ITT) analysis;   Biopsy-related serious hemorrhage rate by per-protocol (PP) analysis
13 Recruiting The Effect of Combined Systematized Behavioural Modification Education Program (SyBeMeP) With DDAVP in Patients With Nocturia
Condition: Nocturia
Intervention: Behavioral: SyBeMeP (Systemized Behavioural Modification education Program)
Outcome Measures: Decrease in urinary frequency (No. of nocturia);   Voiding diary indices;   IPSS;   ICIQ-nocturia;   patient's perception of bladder condition (PPTB);   patient's satisfaction on the education
14 Recruiting Interventional Clinical Trial in Patients in Overactive Bladder With Nocturia in Women
Condition: Overactive Bladder
Interventions: Drug: Tolterodine tartrate extended release capsules + Placebo orally disintegrating tablets;   Drug: Tolterodine tartrate extended release capsules + Desmopressin orally disintegrating tablets
Outcome Measures: Change in mean number of nocturnal voids from baseline;   Change in mean time to first nocturnal void from baseline;   Change in mean nocturnal urine volume from baseline;   Responder status;   Change in mean number of nocturnal voids from baseline for each visit;   Change in the impact on sleep as measured by the sleep rating scales from baseline
15 Recruiting A Study of Minirin Melt in 24 Months Treatment in Patients With Nocturia
Condition: Nocturia
Intervention:
Outcome Measures: Number of patients with adverse events;   Number of changes in dosage regime
16 Recruiting Platelets Antiaggregation Control Enhancement (PACE) Study
Conditions: Bleeding;   Blood Transfusion
Interventions: Device: Aggregometry+Thromboelastography;   Device: Thromboelastography Alone
Outcome Measures: Bleeding Volume;   Use of allogenic blood transfusions.
17 Unknown  Turkish Acquired Haemophilia Registry
Condition: Acquired Haemophilia
Intervention: Drug: rFVIIIa, aPCC, FVIII, DDAVP, Azathioprine, Rituximab, Cyclosporin
Outcome Measure:
18 Unknown  Imidapril and Candesartan on Fibrinolysis and Insulin-Sensitivity in Patients With Mild to Moderate Hypertension
Condition: Essential Hypertension
Interventions: Drug: Imidapril;   Drug: Candesartan
Outcome Measures: PAI-1 level and t-PA activity time course changes;   t-PA activity at the desmopressin test;   Insulin sensitivity state through euglycemic hyperinsulinemic clamp method;   Blood pressure changes
19 Recruiting Thrombocytopathy in Gaucher Disease Patients
Conditions: Gaucher Disease;   Thrombocytopathy
Intervention:
Outcome Measures: Measure thrombocytopathy in a cohort of 70 Gaucher patients using a set of platelet function tests.;   Evaluating the impact of Imiglucerase treatment on platelet function
20 Unknown  RETIC Trial: Reversal of Trauma Induced Coagulopathy Using Coagulation Factor Concentrates or Fresh Frozen Plasma
Condition: Major Trauma
Interventions: Drug: Fibrinogen concentrate, Prothrombin complex concentrate and FXIII concentrate;   Drug: Fresh Frozen Plasma blood type 0, A, B and AB
Outcome Measure: Multiple Organ Failure (MOF)