Trial Outcomes & Findings for Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease (NCT NCT02225132)

NCT ID: NCT02225132

Last Updated: 2019-08-06

Results Overview

Mean fetal hemoglobin calculated to indicate effectiveness of hydroxyurea dose

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: Baseline
• Results posted on: 2019-08-06

Participant Flow

Participant milestones

Measure	Hydroxyurea Hydroxyurea is given to the patients as per a dosing algorithm.
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

Baseline characteristics by cohort

Measure	Hydroxyurea n=10 Participants Hydroxyurea is given to the patients as per a dosing algorithm.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	10 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline

Mean fetal hemoglobin calculated to indicate effectiveness of hydroxyurea dose

Outcome measures

Measure	Hydroxyurea n=10 Participants Hydroxyurea is given to the patients as per a dosing algorithm.
Fetal Hemoglobin Level	8.3 Percentage Standard Deviation 5.9

PRIMARY outcome

Timeframe: 12 months

Mean fetal hemoglobin calculated to indicate effectiveness of hydroxyurea dose

Outcome measures

Measure	Hydroxyurea n=10 Participants Hydroxyurea is given to the patients as per a dosing algorithm.
Fetal Hemoglobin Level	25.1 Percentage Standard Deviation 6.2

Adverse Events

Hydroxyurea

Serious events: 3 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	Hydroxyurea n=10 participants at risk Hydroxyurea is given to the patients as per a dosing algorithm.
Blood and lymphatic system disorders Acute chest syndrome	10.0% 1/10 • 12 months
Blood and lymphatic system disorders Sickle cell anaemia with crisis	20.0% 2/10 • 12 months
Infections and infestations Viral infection	10.0% 1/10 • 12 months
Surgical and medical procedures Knee arthroplasty	10.0% 1/10 • 12 months

Other adverse events

Measure	Hydroxyurea n=10 participants at risk Hydroxyurea is given to the patients as per a dosing algorithm.
Blood and lymphatic system disorders Anaemia	20.0% 2/10 • 12 months
Blood and lymphatic system disorders Blood disorder	50.0% 5/10 • 12 months
Blood and lymphatic system disorders Lymph node pain	10.0% 1/10 • 12 months
Cardiac disorders Dizziness	20.0% 2/10 • 12 months
Cardiac disorders Dyspnoea	20.0% 2/10 • 12 months
Cardiac disorders Oedema peripheral	20.0% 2/10 • 12 months
Eye disorders Vision blurred	10.0% 1/10 • 12 months
Eye disorders Vitreous floaters	10.0% 1/10 • 12 months
Gastrointestinal disorders Diarrhoea	10.0% 1/10 • 12 months
Gastrointestinal disorders Nausea	30.0% 3/10 • 12 months
Gastrointestinal disorders Vomiting	20.0% 2/10 • 12 months
General disorders Fatigue	20.0% 2/10 • 12 months
General disorders Influenza like illness	10.0% 1/10 • 12 months
General disorders Pain	80.0% 8/10 • 12 months
General disorders Pyrexia	20.0% 2/10 • 12 months
Infections and infestations Bronchitis	10.0% 1/10 • 12 months
Infections and infestations Upper respiratory tract infection	10.0% 1/10 • 12 months
Investigations Alanine aminotransferase increased	20.0% 2/10 • 12 months
Investigations Neutrophil count decreased	70.0% 7/10 • 12 months
Investigations Platelet count decreased	20.0% 2/10 • 12 months
Musculoskeletal and connective tissue disorders Arthralgia	10.0% 1/10 • 12 months
Nervous system disorders Headache	40.0% 4/10 • 12 months
Nervous system disorders Insomnia	10.0% 1/10 • 12 months
Nervous system disorders Nervous system disorder	10.0% 1/10 • 12 months
Nervous system disorders Somnolence	10.0% 1/10 • 12 months
Renal and urinary disorders Chromaturia	10.0% 1/10 • 12 months
Respiratory, thoracic and mediastinal disorders Cough	10.0% 1/10 • 12 months
Skin and subcutaneous tissue disorders Dermatitis bullous	10.0% 1/10 • 12 months
Skin and subcutaneous tissue disorders Nail discolouration	20.0% 2/10 • 12 months
Skin and subcutaneous tissue disorders Pruritus	10.0% 1/10 • 12 months
Skin and subcutaneous tissue disorders Rash maculo-papular	30.0% 3/10 • 12 months
Skin and subcutaneous tissue disorders Skin disorder	10.0% 1/10 • 12 months
Skin and subcutaneous tissue disorders Skin hyperpigmentation	30.0% 3/10 • 12 months
Vascular disorders Embolism	10.0% 1/10 • 12 months
Vascular disorders Thrombophlebitis superficial	10.0% 1/10 • 12 months

Additional Information

Fitzhugh, Courtney

National Heart Lung and Blood Institute

Phone: +1 301 402 6496

Email: courtney.fitzhugh@nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place