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CompletedNCT03763656HUPKUpdated Oct 28, 2024Results posted

Pharmacokinetics of Oral Hydroxyurea Solution

A Phase 1/2 interventional study of Oral Hydroxyurea (100 mg/mL) Solution in Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia and Sickle Cell-beta-thalassemia, sponsored by Nova Laboratories Limited. Completed at 6 sites in 2 countries. Open to participants aged 6 Months to 17 Years. Per ClinicalTrials.gov, last updated 2024-10-28.

Sponsored by Nova Laboratories Limited · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
33
Allocation
Not applicable
Ages
6 Months to 17 Years
Sex
All
01

Study summary

An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 to 15 month treatment period for each participant. The study treatment duration will be for 6 months at the maximum tolerated dose [MTD], which is usually reached by 6 months after initiation of treatment. For patients in whom time to MTD is longer than 6 months or not achieved at all, the maximum duration of study treatment will be 15 months.

02

Conditions studied

  • Sickle Cell Disease
  • Sickle-Cell; Hemoglobin Disease, Thalassemia
  • Sickle Cell-beta-thalassemia
  • Sickle Cell Hemoglobin C

Keywords

  • Hydroxyurea
  • Hydroxycarbamide
  • Xromi
03

In context

Anemia, Sickle Cell

1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.

This study's enrollment of 33 is below the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.

Browse Anemia, Sickle Cell studies →

Lead sponsor

Nova Laboratories Limited is the lead sponsor of 5 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Months to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Male or female aged from 6 months to 17.99 years of age (i.e. to the day before 18th birthday).
  2. Diagnosis of sickle cell anemia (HbSS and HbSβº).
  3. Parent(s)/legal guardian able and willing to provide written for the child to take part in the study.
  4. Where applicable, the child should assent to undergo blood sampling for pharmacokinetic and biochemistry purposes and to allow physiological measurements to be made.

Exclusion criteria

Exclusion Criteria:

  1. Any medical condition or abnormality, which, in the opinion of the Investigator, might have compromised the safety of the patient or which might have interfered with the study.
  2. Hydroxyurea use within 6 months before enrolment.
  3. Renal insufficiency (known creatinine more than twice the (ULN) for age and >1.0 mg/dL [88.4 μmol/L]).
  4. Clinical evidence of hepatic compromise with (ALT) >3 times the ULN (a temporary swing in ALT did not result in exclusion).
  5. Other significant organ system dysfunction based on the site Investigators discretion.
  6. Severe active infections: fungal, viral or bacterial (as confirmed by culture), examples included tuberculosis, malaria, active hepatitis, osteomyelitis or any other illness that would have precluded the use of HU in normal clinical practice.
  7. Active chronic leg ulcers.
  8. Known allergy to oral HU solution or any of the .
  9. Positive pregnancy test for females of (in post-menarcheal females) before initiation of treatment, unless participant was sexually abstinent. Note: True abstinence was considered as being in line with the preferred and usual lifestyle of the participant. Periodic abstinence (such as calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of .
  10. Inadequate contraception measures in sexually active females (post-menarcheal females) and males of child-bearing age (see Section 9.5.1.10.4).
  11. Breastfeeding at study initiation.
  12. Participation in another clinical trial of an IMP.
  13. Known infection with .
Terms in these criteria, in plain language(8)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
33 participants (actual)

Study arms

  • Experimental
    Open Label

    Novel oral solution formulation of hydroxyurea

    Drug: Oral Hydroxyurea (100 mg/mL) Solution

Interventions

  • DrugOral Hydroxyurea (100 mg/mL) Solution

    Drug page: Hydroxyurea

    Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.

06

What researchers measure

Primary outcomes

  1. Clearance (CL/F)

    Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).

    Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

  2. Volume of Distribution (V/F)

    Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).

    Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

  3. Time to Maximum Concentration (Tmax)

    Mean Tmax (h) pharmacokinetic parameter derived using the final population PK model.

    Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

  4. Maximum Plasma Concentration Cmax (ug/mL)

    Mean Cmax (ug/mL) pharmacokinetic parameter derived using the final population PK model.

    Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

  5. Area Under Plasma Concentration Time Curve (AUC 0-Inf)

    Mean AUC 0-Infinity (hr\*ug/mL) pharmacokinetic parameters derived using the final population PK model.

    Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

  6. Terminal Half-life (Hours)

    Mean Terminal Half-life (hours) pharmacokinetic parameter derived using the final population PK model.

    Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

Secondary outcomes

  1. Adverse Events

    Incidence of Adverse events during the course of the trial from screening to final follow up phone call at Week 64. Relatedness refers to 'at least possibly related to the IMP', with severity/toxicity assessed using the CTCAE Toxicity Grade and seriousness based on the standard definition for SAE in accordance with GCP. With the exception of SCA related events, requiring \>7day hospitalisation, or extension of hospitalisation before being reported as an SAE due to their frequency within the disease population. All other non-SCA related SAEs were reportable.

    Time frame: Screening Up to Week 64

  2. Absolute Neutrophil Count (ANC)

    Safety review for haematological toxicity (mild myelosuppression target: 1-3x10\^9/L)

    Time frame: Baseline and Week 60 (or final visit); max 15 months on treatment

  3. White Blood Cell Count (Leukocytes)

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Baseline to Week 60 or Final Visit; max 15 months on treatment

  4. Platelets

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Baseline to Week 60 (or Final Visit), max 15 months on treatment

  5. Mean Corpuscular Hemoglobin (MCH)

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Baseline to Week 60 (or Final Visit), max 15 months on treatment

  6. Hematocrit

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Up to Week 60

  7. Bilirubin

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Up to Week 60

  8. Elevation in Liver Function Tests (LFTs)

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry, including Liver Function Tests (LFTs): Alkaline Phosphatase (ALP), Gamma Glutamyl Transferase (GGT), Aspartate Amino Transferase (AST), Alanine Aminotransferase (ALT)

    Time frame: Up to Week 60

  9. Hemoglobin

    Safety and efficacy of hydroxyurea therapy

    Time frame: Baseline to Week 60 (or Final Visit); max 15 months on treatment

  10. Bacterial Infections

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Up to Week 60

  11. Viral Infections

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Up to Week 60

  12. Fungal Infections

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Up to Week 60

  13. Leg Ulcers

    Impact of Hydroxyurea on Safety, Hematology and Biochemistry

    Time frame: Up to Week 60

  14. Fetal Hemoglobin

    Biomarker endpoints of Hydroxyurea efficacy

    Time frame: Baseline to Week 60 (or Final Visit); max 15 months on treatment

  15. Mean Corpuscular Volume (MCV)

    Biomarker endpoints of Hydroxyurea efficacy

    Time frame: Baseline to Week 60 (or Final Visit); max 15 months on treatment

  16. Cystatin C

    Biomarker Endpoints

    Time frame: PK1 (day 1), week 20-32 (6 months) and Week 60 (Final Visit)

  17. Incidence of Acute Vaso-Occlusive Pain Crises (VOC)

    Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for vaso-occlusive crisis (Mean \[SD\])

    Time frame: 12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP

  18. Number and Frequency of Blood Transfusions

    Clinical status endpoints, related to blood transfusions for treatment of SCA, this may be hospitalization, A\&E, or in-clinic treatment from safety population (n=32)

    Time frame: Up to Week 60

  19. Acute Chest Syndrome (ACS)

    Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for Acute Chest Syndrome (Mean \[SD\])

    Time frame: 12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP

  20. Hospitalizations

    Clinical Status endpoints, all hospitalisations (not ER/Accident without hospitalization) (Mean \[SD\])

    Time frame: 12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP

  21. Dose Escalation i.e. Maximum Tolerated Dose (MTD)

    Summary of maximum tolerated dose achieved in mg/kg (Mean \[SD\])

    Time frame: Screening Up to Final Visit (Week 60 or Withdrawal), maximum 15 months on IMP

  22. Other SCA-related Hospitalizations

    Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other Non-SCA related) (mean \[SD\])

    Time frame: 12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP

  23. Parent/Caregiver Palatability and Acceptability Questionnaire

    Clinical status endpoints. Visual Analogue scale used to determine, taste, smell, aftertaste, acceptability and ease of dosing (1-100mm scale) with 1 being worst possible and 100 being best possible. Assessed for \<6 years of age by parents/guardians. Assessed for \>6 years of age, combination of parent/guardian and participant responses.

    Time frame: Taken once at any point after 8 weeks on study medication (or at early Withdrawal)

  24. Vitamin D

    Biochemistry

    Time frame: From Screening Up to Final Visit (Week 60 or WD)

  25. Other Non-SCA-related Hospitalizations

    Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other SCA-related)

    Time frame: 12 months prior to treatment and 12 months post-treatment; maximum 15 months on IMP

Other outcomes

  1. Transcranial Doppler Velocity

    Exploratory Endpoint; clinical output of hydroxyurea treatment

    Time frame: Baseline to Week 40-56 (Follow up scan).

07

Results

Posted Oct 28, 2024

Participant flow

Participants were recruited from one Jamaican sickle cell clinic (n=12) and five UK hospitals (n=20). The first participant was enrolled on 03 Jan 2019 and the last participant was enrolled on 16 Dec 2020.

Participant flow — Overall Study
MilestoneOral Hydroxyurea
Started33
Pre-treatment discontinuation1
Safety population32
Age group (6 months - 1.99 years)6
Discontinuations from age group (6 months - 1.99 years)3
Completed from age group (6 months - 1.99 years)3
Age group (2 - 5.99 years)16
Discontinuations from age group (2 - 5.99 years)2
Completions from age group (2 - 5.99 years)14
Age group (6 - 17.99 years)10
Discontinuations from age group (6 - 17.99 years)3
Completions from age group (6 - 17.99 years)7
Completed24
Not completed9
Withdrew: Physician decision1
Withdrew: Protocol violation1
Withdrew: Withdrawal by subject7

Outcome measures

PrimaryClearance (CL/F)

Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).

Time frame:
0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Reported as:
Mean · L/hr
Clearance (CL/F)
L/hrOral Hydroxyurea
Clearance (CL/F)3.61 (3.29 to 3.92)
PrimaryVolume of Distribution (V/F)

Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).

Time frame:
0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Reported as:
Mean · L
Volume of Distribution (V/F)
LOral Hydroxyurea
Volume of Distribution (V/F)11.4 (9.52 to 13.2)
PrimaryTime to Maximum Concentration (Tmax)

Mean Tmax (h) pharmacokinetic parameter derived using the final population PK model.

Time frame:
0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Reported as:
Mean · hours
Time to Maximum Concentration (Tmax)
hoursOral Hydroxyurea
Tmax (Overall)1.253 ± 0.412
Tmax (Age Group 6 months - 1.99 years)1.265 ± 0.322
Tmax (Age Group 2 - 5.99 years)1.238 ± 0.455
Tmax (Age Group 6 - 17.99 years)1.269 ± 0.423
PrimaryMaximum Plasma Concentration Cmax (ug/mL)

Mean Cmax (ug/mL) pharmacokinetic parameter derived using the final population PK model.

Time frame:
0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Reported as:
Mean · ug/mL
Maximum Plasma Concentration Cmax (ug/mL)
ug/mLOral Hydroxyurea
Cmax (Overall)13.124 ± 1.742
Cmax (Age Group 6 months - 1.99 years)12.807 ± 1.399
Cmax (Age Group 2 - 5.99 years)13.068 ± 1.939
Cmax (Age Group 6 - 17.99 years)13.404 ± 1.714
PrimaryArea Under Plasma Concentration Time Curve (AUC 0-Inf)

Mean AUC 0-Infinity (hr\*ug/mL) pharmacokinetic parameters derived using the final population PK model.

Time frame:
0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Reported as:
Mean · hr*ug/mL
Area Under Plasma Concentration Time Curve (AUC 0-Inf)
hr*ug/mLOral Hydroxyurea
AUC Infinity (Overall)64.721 ± 9.339
AUC Infinity (Age Group 6 months - 1.99 years)62.537 ± 9.016
AUC Infinity (Age Group 2 - 5.99 years)62.899 ± 7.373
AUC Infinity (Age Group 6 - 17.99 years)68.948 ± 11.649
PrimaryTerminal Half-life (Hours)

Mean Terminal Half-life (hours) pharmacokinetic parameter derived using the final population PK model.

Time frame:
0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Reported as:
Mean · hours
Terminal Half-life (Hours)
hoursOral Hydroxyurea
Terminal Half-life (Overall)3.880 ± 0.3004
Terminal Half-life (Age Group 6 months - 1.99 years)4.107 ± 0.187
Terminal Half-life (Age Group 2 - 5.99 years)3.949 ± 0.257
Terminal Half-life (Age 6 - 17.99 years)3.634 ± 0.268
SecondaryAdverse Events

Incidence of Adverse events during the course of the trial from screening to final follow up phone call at Week 64. Relatedness refers to 'at least possibly related to the IMP', with severity/toxicity assessed using the CTCAE Toxicity Grade and seriousness based on the standard definition for SAE in accordance with GCP. With the exception of SCA related events, requiring \>7day hospitalisation, or extension of hospitalisation before being reported as an SAE due to their frequency within the disease population. All other non-SCA related SAEs were reportable.

Time frame:
Screening Up to Week 64
Reported as:
Number · events
Adverse Events
eventsOral Hydroxyurea
Any Adverse Events (Overall)339
Treatment Emergent Adverse Events (TEAEs) Overall339
Serious AEs7
Grade >or=3 TEAEs19
Related TEAEs28
Commonly occurring unrelated TEAE: SCA with Crisis53
Commonly occurring unrelated TEAE: Upper Respiratory Tract Infection43
Commonly occurring unrelated TEAE: Pyrexia16
Commonly occurring unrelated TEAE: Cough7
Commonly occurring unrelated TEAE: Vomiting6
Commonly occurring unrelated TEAE: Abdominal Pain7
Commonly occurring possibly related TEAE: Neutropenia/ANC Count Decreased4
Commonly occurring possibly related TEAE: Thrombocytopenia/Platelet Count Decreased6
SecondaryAbsolute Neutrophil Count (ANC)

Safety review for haematological toxicity (mild myelosuppression target: 1-3x10\^9/L)

Time frame:
Baseline and Week 60 (or final visit); max 15 months on treatment
Reported as:
Mean · x10^9 neutrophils/L
Absolute Neutrophil Count (ANC)
x10^9 neutrophils/LOral Hydroxyurea
Screening (Overall)4.90 ± 1.825
Screening (6 months - 1.99 years)4.30 ± 1.608
Screening (2 - 5.99 years)5.38 ± 2.087
Screening (6 - 17.99 years)4.43 ± 1.354
Final Visit (Overall)2.70 ± 1.033
Final Visit (6 months - 1.99 years)2.27 ± 0.671
Final Visit (2 - 5.99 years)2.46 ± 0.837
Final Visit (6 - 17.99 years)3.48 ± 1.255
SecondaryWhite Blood Cell Count (Leukocytes)

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Baseline to Week 60 or Final Visit; max 15 months on treatment
Reported as:
Mean · 10^9 leukocytes/L
White Blood Cell Count (Leukocytes)
10^9 leukocytes/LOral Hydroxyurea
Screening (Overall)13.01 ± 3.514
Screening (6 months - 1.99 years)12.24 ± 2.019
Screening (2 - 5.99 years)14.59 ± 3.856
Screening (6 - 17.99 years)10.88 ± 2.188
Final Visit (Overall)7.48 ± 2.286
Final Visit (6 months - 1.99 years)7.90 ± 2.466
Final Visit (2 - 5.99 years)6.83 ± 2.244
Final Visit (6 - 17.99 years)8.41 ± 2.108
SecondaryPlatelets

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Baseline to Week 60 (or Final Visit), max 15 months on treatment
Reported as:
Mean · 10^9 platelets/L
Platelets
10^9 platelets/LOral Hydroxyurea
Screening (Overall)409.06 ± 131.542
Screening (6 months - 1.99 years)383.80 ± 52.756
Screening (2 - 5.99 years)381.31 ± 104.305
Screening (6 - 17.99 years)466.10 ± 181.577
Final Visit (Overall)315.10 ± 134.057
Final Visit (6 months - 1.99 years)333.67 ± 98.699
Final Visit (2 - 5.99 years)258.00 ± 141.782
Final Visit (6 - 17.99 years)408.25 ± 86.742
SecondaryMean Corpuscular Hemoglobin (MCH)

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Baseline to Week 60 (or Final Visit), max 15 months on treatment
Reported as:
Mean · pg
Mean Corpuscular Hemoglobin (MCH)
pgOral Hydroxyurea
Screening (Overall)27.57 ± 3.741
Screening (6 months - 1.99 years)23.80 ± 3.291
Screening (2 - 5.99 years)28.36 ± 4.665
Screening (6 - 17.99 years)28.53 ± 1.015
Final Visit (Overall)33.36 ± 4.590
Final Visit (6 months - 1.99 years)28.95 ± 8.126
Final Visit (2 - 5.99 years)34.02 ± 3.971
Final Visit (6 - 17.99 years)34.31 ± 2.307
SecondaryHematocrit

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Up to Week 60
Reported as:
Mean · % by volume of red blood cells in blood
Hematocrit
% by volume of red blood cells in bloodOral Hydroxyurea
Screening (Overall)25.02 ± 4.4
Screening (6 months - 1.99 years)30.60 ± 3.161
Screening (2 - 5.99 years)23.24 ± 3.378
Screening (6 - 17.99 years)24.30 ± 4.081
Final Visit (Overall)27.74 ± 3.995
Final Visit (6 months - 1.99 years)30.38 ± 3.035
Final Visit (2 - 5.99 years)28.45 ± 4.001
Final Visit (6 - 17.99 years)25.10 ± 3.208
SecondaryBilirubin

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Up to Week 60
Reported as:
Mean · umol/L
Bilirubin
umol/LOral Hydroxyurea
Screening (Overall)37.43 ± 19.649
Screening (6 months - 1.99 years)14.78 ± 9.763
Screening (2 - 5.99 years)39.60 ± 19.777
Screening (6 - 17.99 years)47.53 ± 12.702
Final Visit (Overall)26.12 ± 16.191
Final Visit (6 months - 1.99 years)7.42 ± 1.941
Final Visit (Screening (2 - 5.99 years)26.25 ± 13.331
Final Visit (Screening (6 - 17.99 years)39.89 ± 13.191
SecondaryElevation in Liver Function Tests (LFTs)

Impact of Hydroxyurea on Safety, Hematology and Biochemistry, including Liver Function Tests (LFTs): Alkaline Phosphatase (ALP), Gamma Glutamyl Transferase (GGT), Aspartate Amino Transferase (AST), Alanine Aminotransferase (ALT)

Time frame:
Up to Week 60
Reported as:
Mean · ukat/L
Elevation in Liver Function Tests (LFTs)
ukat/LOral Hydroxyurea
ALP Screening (Overall)3.55 ± 0.846
ALP Screening (6 months - 1.99 years)4.19 ± 0.983
ALP Screening (2 - 5.99 years)3.60 ± 0.666
ALP Screening (6 - 17.99 years)3.10 ± 0.827
ALP Final Visit (Overall)3.57 ± 0.858
ALP Final Visit (6 months - 1.99 years)4.06 ± 0.820
ALP Final Visit (2 months - 5.99 years)3.41 ± 0.578
ALP Final Visit (6 - 17.99 years)3.52 ± 1.237
GGT Screening (Overall)0.38 ± 0.333
GGT Screening (6 months - 1.99 years)0.16 ± 0.034
GGT Screening (2 - 5.99 years)0.32 ± 0.231
GGT Screening (6 - 17.99 years)0.62 ± 0.437
GGT Final Visit (Overall)0.32 ± 0.233
GGT Final Visit (6 months - 1.99 years)0.16 ± 0.040
GGT Final Visit (2 - 5.99 years)0.29 ± 0.174
GGT Final Visit (6 - 17.99 years)0.49 ± 0.316
AST Screening (Overall)0.85 ± 0.221
AST Screening (6 months - 1.99 years)0.72 ± 0.178
AST Screening (2 - 5.99 years)0.84 ± 0.212
AST Screening (6 - 17.99 years)0.92 ± 0.244
AST Final Visit (Overallk)0.67 ± 0.165
AST Final Visit (6 months - 1.99 years)0.61 ± 0.066
AST Final Visit (2 - 5.99 years)0.68 ± 0.185
AST Final Visit (6 - 17.99 years)0.71 ± 0.181
ALT Screening (Overall)0.33 ± 0.128
ALT Screening (6 months - 1.99 years)0.34 ± 0.053
ALT Screening (2 - 5.99 years)0.30 ± 0.129
ALT Screening (6 - 17.99 years)0.35 ± 0.158
ALT Final Visit (Overall)0.31 ± 0.110
ALT Final Visit (6 months - 1.99 years)0.33 ± 0.067
ALT Final Visit (2 - 5.99 years)0.30 ± 0.109
ALT Final Visit (6 - 17.99 years)0.32 ± 0.142
SecondaryHemoglobin

Safety and efficacy of hydroxyurea therapy

Time frame:
Baseline to Week 60 (or Final Visit); max 15 months on treatment
Reported as:
Mean · g/dL
Hemoglobin
g/dLOral Hydroxyurea
Screening (Overall)8.32 ± 1.114
Screening (6 months - 1.99 years)9.76 ± 0.847
Screening (2 - 5.99 years)8.07 ± 0.980
Screening (6 - 17.99 years)7.99 ± 0.911
Final Visit (Overall)9.47 ± 1.369
Final Visit (6 months - 1.99 years)10.60 ± 1.421
Final Visit (2 - 5.99 years)9.47 ± 1.190
Final Visit (6 - 17.99 years)8.63 ± 1.149
SecondaryBacterial Infections

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Up to Week 60
Reported as:
Number · events
Bacterial Infections
eventsOral Hydroxyurea
Prior to Treatment (Overall)7
Prior to Treatment (6 months - 1.99 years)0
Prior to Treatment (2 - 5.99 years)3
Prior to Treatment (6 - 17.99 years)4
After Treatment (Overall)17
After Treatment (6 months - 1.99 years)1
After Treatment (2 - 5.99 years)8
After Treatment (6 - 17.99 years)8
SecondaryViral Infections

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Up to Week 60
Reported as:
Number · Events
Viral Infections
EventsOral Hydroxyurea
Prior to Treatment (Overall)8
Prior to Treatment (6 months - 1.99 years)0
Prior to Treatment (2 - 5.99 years)8
Prior to Treatment (6 - 17.99 years)0
After Treatment (Overall)23
After Treatment (6 months - 1.99 years)4
After Treatment (2 - 5.99 years)12
After Treatment (6 - 17.99 years)7
SecondaryFungal Infections

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Up to Week 60
Reported as:
Number · events
Fungal Infections
eventsOral Hydroxyurea
Prior to Treatment (Overall)1
Prior to Treatment (6 months - 1.99 years)0
Prior to Treatment (2 - 5.99 years)0
Prior to Treatment (6 - 17.99 years)1
After Treatment (Overall)4
After Treatment (6 months - 1.99 years)1
After Treatment (2 - 5.99 years)1
After Treatment (6 - 17.99 years)2
SecondaryLeg Ulcers

Impact of Hydroxyurea on Safety, Hematology and Biochemistry

Time frame:
Up to Week 60
Reported as:
Number · events
Leg Ulcers
eventsOral Hydroxyurea
Prior to Treatment (Overall)0
Prior to Treatment (6 months -1.99 years)0
Prior to Treatment (2 - 5.99 years)0
Prior to Treatment (6 -17.99 years)0
After Treatment (Overall)1
After Treatment (6 months -1.99 years)0
After Treatment (2 - 5.99 years)0
After Treatment (6 - 17.99 years)1
SecondaryFetal Hemoglobin

Biomarker endpoints of Hydroxyurea efficacy

Time frame:
Baseline to Week 60 (or Final Visit); max 15 months on treatment
Reported as:
Mean · Hemoglobin F/Total Hemoglobin %
Fetal Hemoglobin
Hemoglobin F/Total Hemoglobin %Oral Hydroxyurea
Screening (Overall)11.90 ± 8.711
Screening (6 months - 1.99 years)21.52 ± 6.889
Screening (2 - 5.99 years)10.94 ± 7.637
Screening (6 - 17.99 years)5.57 ± 4.641
Final Visit (Overall)23.53 ± 11.179
Final Visit (6 months - 1.99 years)26.32 ± 4.893
Final Visit (2 - 5.99 years)27.09 ± 11.888
Final Visit (6 - 17.99 years)12.47 ± 6.467
SecondaryMean Corpuscular Volume (MCV)

Biomarker endpoints of Hydroxyurea efficacy

Time frame:
Baseline to Week 60 (or Final Visit); max 15 months on treatment
Reported as:
Mean · fL
Mean Corpuscular Volume (MCV)
fLOral Hydroxyurea
Screening (Overall)83.13 ± 8.643
Screening (6 months - 1.99 years)76.42 ± 7.682
Screening (2 - 5.99 years)83.25 ± 9.715
Screening (6 - 17.99 years)86.30 ± 5.410
Final Visit (Overall)97.64 ± 13.700
Final Visit (6 months - 1.99 years)81.68 ± 14.506
Final Visit (2 - 5.99 years)102.49 ± 11.736
Final Visit (6 - 17.99 years)100.51 ± 6.859
SecondaryCystatin C

Biomarker Endpoints

Time frame:
PK1 (day 1), week 20-32 (6 months) and Week 60 (Final Visit)
Reported as:
Mean · nmol/L
Cystatin C
nmol/LOral Hydroxyurea
PK1 Day 1 (Overall)7.52 ± 1.288
PK1 Day 1 (6 months - 1.99 years)8.89 ± 1.170
PK1 Day 1 (2 - 5.99 years)6.87 ± 1.001
PK1 Day 1 (6 - 17.99 years)7.18 ± 0.549
Week 24 (Overall)7.50 ± 1.173
Week 24 (6 months - 1.99 years)8.36 ± 0.263
Week 24 (2 - 5.99 years)7.07 ± 1.074
Week 24 (6 - 17.99 years)7.52 ± 1.810
Week 28 (Overall)8.67 ± 0.134
Week 28 (2 - 5.99 years)8.76 ± NA
Week 28 (6 - 17.99 years)8.57 ± NA
Final Visit (Overall)7.44 ± 1.340
Final Visit (6 months - 1.99 years)8.63 ± 0.999
Final Visit (2 - 5.99 years)7.19 ± 1.253
Final Visit (6 - 17.99 years)6.80 ± 1.192
SecondaryIncidence of Acute Vaso-Occlusive Pain Crises (VOC)

Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for vaso-occlusive crisis (Mean \[SD\])

Time frame:
12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP
Reported as:
Mean · hospitalisation events per month
Incidence of Acute Vaso-Occlusive Pain Crises (VOC)
hospitalisation events per monthOral Hydroxyurea
VOC 12months prior (Overall)0.5 ± 0.92
VOC 12months prior (6 months - 1.99 years))0.0 ± 0.0
VOC 12months prior (2 - 5.99 years))0.7 ± 1.08
VOC 12months prior (6 - 17.99 years)0.6 ± 0.84
VOC 12months after treatment (Overall)0.2 ± 0.51
VOC 12months after treatment (6 months - 1.99 years))0.3 ± 0.82
VOC 12months after treatment (2 - 5.99 years)0.0 ± 0.0
VOC 12months after treatment (6 - 17.99 years))0.3 ± 0.67
SecondaryNumber and Frequency of Blood Transfusions

Clinical status endpoints, related to blood transfusions for treatment of SCA, this may be hospitalization, A\&E, or in-clinic treatment from safety population (n=32)

Time frame:
Up to Week 60
Reported as:
Number · events (number of transfusions)
Number and Frequency of Blood Transfusions
events (number of transfusions)Oral Hydroxyurea
Overall30
2 years - 5.99 years21
6 years - 17.99 years9
SecondaryAcute Chest Syndrome (ACS)

Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for Acute Chest Syndrome (Mean \[SD\])

Time frame:
12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP
Reported as:
Mean · hospitalisation events per month
Acute Chest Syndrome (ACS)
hospitalisation events per monthOral Hydroxyurea
ACS Hospitalisations 12 months prior (Overall)0.5 ± 0.92
ACS Hospitalisations 12 months prior (6 months - 1.99 years)0.00 ± 0.00
ACS Hospitalisations 12 months prior (2 - 5.99 years)0.6 ± 0.89
ACS Hospitalisations 12 months prior (6 - 17.99 years)0.7 ± 1.16
ACS Hospitalisations 12 months after treatment (Overall)0.2 ± 0.37
ACS Hospitalisations 12 months after treatment (6 months - 1.99 years)0.00 ± 0.00
ACS Hospitalisations 12 months after treatment (2 - 5.99 years)0.3 ± 0.45
ACS Hospitalisations 12 months after treatment (6 - 17.99 years)0.1 ± 0.32
SecondaryHospitalizations

Clinical Status endpoints, all hospitalisations (not ER/Accident without hospitalization) (Mean \[SD\])

Time frame:
12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP
Reported as:
Mean · hospitalisation events per month
Hospitalizations
hospitalisation events per monthOral Hydroxyurea
Hospitalisations 12 months prior (Overall)1.7 ± 1.54
Hospitalisations 12 months prior (6 months - 1.99 years)0.3 ± 0.82
Hospitalisations 12 months prior (2 - 5.99 years)2.0 ± 1.59
Hospitalisations 12 months prior (6 - 17.99 years)1.9 ± 1.45
Hospitalisations 12 months after treatment (Overall)0.3 ± 0.65
Hospitalisations 12 months after treatment (6 months - 1.99 years)0.3 ± 0.82
Hospitalisations 12 months after treatment (2 - 5.99 years)0.3 ± 0.45
Hospitalisations 12 months after treatment (6 - 17.99 years)0.5 ± 0.85
SecondaryDose Escalation i.e. Maximum Tolerated Dose (MTD)

Summary of maximum tolerated dose achieved in mg/kg (Mean \[SD\])

Time frame:
Screening Up to Final Visit (Week 60 or Withdrawal), maximum 15 months on IMP
Reported as:
Mean · mg/kg
Dose Escalation i.e. Maximum Tolerated Dose (MTD)
mg/kgOral Hydroxyurea
MTD (Overall)25.63 ± 6.780
MTD (6 months - 1.99 years)23.50 ± 8.944
MTD (2 - 5.99 years)26.11 ± 5.465
MTD (6 - 17.99 years)26.67 ± 7.528
SecondaryOther SCA-related Hospitalizations

Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other Non-SCA related) (mean \[SD\])

Time frame:
12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP
Reported as:
Mean · hospitalisation events per month
Other SCA-related Hospitalizations
hospitalisation events per monthOral Hydroxyurea
Other SCA related Hospitalizations 12 months prior (Overall)0.4 ± 0.67
Other SCA related Hospitalizations 12 months prior (6 months - 1.99 years)0.3 ± 0.82
Other SCA related Hospitalizations 12 months prior (2 - 5.99 years)0.4 ± 0.63
Other SCA related Hospitalizations 12 months prior (6 - 17.99 years)0.4 ± 0.70
Other SCA related Hospitalizations 12 months after treatment (Overall)0.0 ± 0.18
Other SCA related Hospitalizations 12 months after treatment (6 months - 1.99 years)0.00 ± 0.00
Other SCA related Hospitalizations 12 months after treatment (2 - 5.99 years)0.00 ± 0.00
Other SCA related Hospitalizations 12 months after treatment (6 - 17.99 years)0.1 ± 0.32
SecondaryParent/Caregiver Palatability and Acceptability Questionnaire

Clinical status endpoints. Visual Analogue scale used to determine, taste, smell, aftertaste, acceptability and ease of dosing (1-100mm scale) with 1 being worst possible and 100 being best possible. Assessed for \<6 years of age by parents/guardians. Assessed for \>6 years of age, combination of parent/guardian and participant responses.

Time frame:
Taken once at any point after 8 weeks on study medication (or at early Withdrawal)
Reported as:
Mean · mm
Parent/Caregiver Palatability and Acceptability Questionnaire
mmOral Hydroxyurea
Taste (Overall)84.2 ± 26.21
Taste (6 months - 1.99 years)71.3 ± 23.30
Taste (2 - 5.99 years)91.1 ± 23.86
Taste (6 - 17.99 years)80.8 ± 30.14
Smell (Overall)83.8 ± 21.73
Smell (6 months - 1.99 years)74.8 ± 25.51
Smell (2 - 5.99 years)87.5 ± 17.26
Smell (6 - 17.99 years)83.1 ± 26.28
Aftertaste (>6 years)67.6 ± 23.21
SecondaryVitamin D

Biochemistry

Time frame:
From Screening Up to Final Visit (Week 60 or WD)
Reported as:
Mean · nmol/L
Vitamin D
nmol/LOral Hydroxyurea
Screening (Overall)59.35 ± 32.613
Screening (6 months - 1.99 years)83.07 ± 32.329
Screening (2 - 5.99 years)65.43 ± 29.076
Screening (6 - 17.99 years)35.39 ± 24.403
Final Visit (Overall)62.26 ± 30.220
Final Visit (6 months - 1.99 years)86.44 ± 25.475
Final Visit (2 - 5.99 years)57.54 ± 34.093
Final Visit (6 - 17.99 years)52.97 ± 14.749
SecondaryOther Non-SCA-related Hospitalizations

Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other SCA-related)

Time frame:
12 months prior to treatment and 12 months post-treatment; maximum 15 months on IMP
Reported as:
Mean · events
Other Non-SCA-related Hospitalizations
eventsOral Hydroxyurea
Other Non-SCA related Hospitalizations 12 months prior (Overall)0.2 ± 0.47
Other Non-SCA related Hospitalizations 12 months prior (6 months - 1.99 years)0.00 ± 0.00
Other Non-SCA related Hospitalizations 12 months prior (2 - 5.99 years)0.3 ± 0.58
Other Non-SCA related Hospitalizations 12 months prior (6 - 17.99 years)0.2 ± 0.42
Other Non-SCA related Hospitalizations 12 months after treatment (Overall)0.00 ± 0.00
Other Non-SCA related Hospitalizations 12 months after treatment (6 months - 1.99 years)0.00 ± 0.00
Other Non-SCA related Hospitalizations 12 months after treatment (2 - 5.99 years)0.00 ± 0.00
Other Non-SCA related Hospitalizations 12 months after treatment (6 - 17.99 years)0.00 ± 0.00
Other pre-specifiedTranscranial Doppler Velocity

Exploratory Endpoint; clinical output of hydroxyurea treatment

Time frame:
Baseline to Week 40-56 (Follow up scan).
Reported as:
Mean · cm/sec
Transcranial Doppler Velocity
cm/secOral Hydroxyurea
Screening (Overall)141.5 ± 34.71
Screening (6 months - 1.99 years)90.5 ± 9.33
Screening (2 - 5.99 years)150.0 ± 34.78
Screening (6 - 17.99 years)148.3 ± 22.42
Week 40-56 (Overall)133.7 ± 26.52
Week 40-56 (6 months - 1.99 years)95.7 ± 15.18
Week 40-56 (2 - 5.99 years)138.6 ± 24.86
Week 40-56 (6 - 17.99 years)140.7 ± 20.79
Baseline Shift (Overall)-11.5 ± 20.74
Baseline Shift (6 months - 1.99 years)-1.0 ± NA
Baseline Shift (2 - 5.99 years)-13.7 ± 22.02
Baseline Shift (6 - 17.99 years)-9.0 ± 20.68

Adverse events

Collected over Screening to safety follow up call at 64 weeks (maximum 15 months on treatment).. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Oral Hydroxyurea0/32 (0%)6/32 (18.8%)31/32 (96.9%)
Most frequent serious events
Most frequent serious events
EventOral Hydroxyurea
Acute Chest Syndrome (ACS) Grade 3Respiratory, thoracic and mediastinal disorders3/32
Vaso-occlusive Crisis (VOC)Blood and lymphatic system disorders2/32
Acute Chest Syndrome (ACS) Grade 2Respiratory, thoracic and mediastinal disorders1/32
Anemia secondary to SCDBlood and lymphatic system disorders1/32
Most frequent other events
Showing 10 of 112
Most frequent other events
EventOral Hydroxyurea
Sickle Cell Anemia with Crisis/Vaso-Occlusive Crisis (VOC)Blood and lymphatic system disorders18/32
Upper Respiratory Tract InfectionInfections and infestations17/32
PyrexiaGeneral disorders9/32
CoughRespiratory, thoracic and mediastinal disorders7/32
Abdominal PainGastrointestinal disorders6/32
VomitingGastrointestinal disorders6/32
Thrombocytopenia/Low Platelet CountBlood and lymphatic system disorders5/32
Pain in extremityMusculoskeletal and connective tissue disorders5/32
Lower Respiratory Tract InfectionInfections and infestations5/32
Viral InfectionInfections and infestations5/32

Baseline characteristics

Participant demographics were summarized for all 33 participants. One participant discontinued prior to receiving a first dose of IMP. The remaining safety population consisted of 32 participants who all received at least one dose of the IMP and one valid primary efficacy PK measurement. This safety population was used for all analyses of baseline characteristics, secondary and exploratory outcomes, as well as safety outcomes.

Age, Customized
Age, Customized(Participants)Oral Hydroxyurea
Age by Category — Age Group (6 months - 1.99 years)6
Age by Category — Age Group (2 - 5.99 years)16
Age by Category — Age Group (6 - 17.99 years)11
Sex: Female, Male
Sex: Female, Male(Participants)Oral Hydroxyurea
Sex: Female, Male (Overall) — Female17
Sex: Female, Male (Overall) — Male16
Sex: Female, Male (Age group 6 months - 1.99 years) — Female5
Sex: Female, Male (Age group 6 months - 1.99 years) — Male1
Sex: Female, Male (Age group 2 - 5.99 years) — Female5
Sex: Female, Male (Age group 2 - 5.99 years) — Male11
Sex: Female, Male (Age group 6 - 17.99 years) — Female7
Sex: Female, Male (Age group 6 - 17.99 years) — Male4
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Oral Hydroxyurea
Ethnicity Overall — Hispanic or Latino0
Ethnicity Overall — Not Hispanic or Latino33
Ethnicity Overall — Unknown or Not Reported0
Ethnicity (Age Group 6 months - 1.99 years) — Hispanic or Latino0
Ethnicity (Age Group 6 months - 1.99 years) — Not Hispanic or Latino6
Ethnicity (Age Group 6 months - 1.99 years) — Unknown or Not Reported0
Ethnicity (Age Group 2 - 5.99 years) — Hispanic or Latino0
Ethnicity (Age Group 2 - 5.99 years) — Not Hispanic or Latino16
Ethnicity (Age Group 2 - 5.99 years) — Unknown or Not Reported0
Ethnicity (Age Group 6 - 17.99 years) — Hispanic or Latino0
Ethnicity (Age Group 6 - 17.99 years) — Not Hispanic or Latino11
Ethnicity (Age Group 6 - 17.99 years) — Unknown or Not Reported0
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Oral Hydroxyurea
Race (Overall) — American Indian or Alaska Native0
Race (Overall) — Asian0
Race (Overall) — Black or African American31
Race (Overall) — Native Hawaiian or Other Pacific Islander0
Race (Overall) — White0
Race (Overall) — Other (Specify): [sic] Caribbean1
Race (Overall) — Other (Specify): Black British1
Race (Age Group 6 months - 1.99 years) — American Indian or Alaska Native0
Race (Age Group 6 months - 1.99 years) — Asian0
Race (Age Group 6 months - 1.99 years) — Black or African American5
Race (Age Group 6 months - 1.99 years) — Native Hawaiian or Other Pacific Islander0
Race (Age Group 6 months - 1.99 years) — White0
Race (Age Group 6 months - 1.99 years) — Other (Specify): [sic] Caribbean1
Race (Age Group 6 months - 1.99 years) — Other (Specify): Black British0
Race (Age Group 2 - 5.99 years) — American Indian or Alaska Native0
Race (Age Group 2 - 5.99 years) — Asian0
Race (Age Group 2 - 5.99 years) — Black or African American15
Race (Age Group 2 - 5.99 years) — Native Hawaiian or Other Pacific Islander0
Race (Age Group 2 - 5.99 years) — White0
Race (Age Group 2 - 5.99 years) — Other (Specify): [sic] Caribbean0
Race (Age Group 2 - 5.99 years) — Other (Specify): Black British1
Race (Age Group 6 - 17.99 years) — American Indian or Alaska Native0
Race (Age Group 6 - 17.99 years) — Asian0
Race (Age Group 6 - 17.99 years) — Black or African American11
Race (Age Group 6 - 17.99 years) — Native Hawaiian or Other Pacific Islander0
Race (Age Group 6 - 17.99 years) — White0
Race (Age Group 6 - 17.99 years) — Other (Specify): [sic] Caribbean0
Race (Age Group 6 - 17.99 years) — Other (Specify): Black British0
Region of Enrollment
Region of Enrollment(participants)Oral Hydroxyurea
United Kingdom20
Jamaica13
SCA Type
SCA Type(Participants)Oral Hydroxyurea
SCA Type (Overall) — HbSS32
SCA Type (Overall) — HbSbeta01
SCA Type (Age Group 6 months - 1.99 years) — HbSS6
SCA Type (Age Group 6 months - 1.99 years) — HbSbeta00
SCA Type (Age Group 2 - 5.99 years) — HbSS15
SCA Type (Age Group 2 - 5.99 years) — HbSbeta01
SCA Type (Age Group 6 - 17.99 years) — HbSS11
SCA Type (Age Group 6 - 17.99 years) — HbSbeta00
Height
Height(cm)Oral Hydroxyurea
Height (cm) (Overall)109.26 ± 26.038
Height (cm) Age Group (6 months - 1.99 years)79.40 ± 5.237
Height (cm) Age Group (2 - 5.99 years)98.08 ± 6.947
Height (cm) Age Group (6 - 17.99 years)142.09 ± 15.872
Weight
Weight(kg)Oral Hydroxyurea
Weight (kg) Overall19.63 ± 10.955
Weight (kg) Age Group (6 months - 1.99 years)11.18 ± 1.017
Weight (kg) Age Group (2 - 5.99 years)14.74 ± 2.092
Weight (kg) Age Group (6 - 17.99 years)32.52 ± 11.441
08

Study locations

6 sites
  • Dr Angela E Rankine- Mullings
    Kingston, Jamaica
  • Birmingham Women's and Children's NHS Foundation Trust
    Birmingham, United Kingdom
  • Alder Hey Children's NHS Foundation Trust
    Liverpool, United Kingdom
  • Evelina London Children's Hospital
    London, United Kingdom
  • King's College Hospital NHS Foundation Trust
    London, United Kingdom
  • The Royal London Children's Hospital, Barts Health NHS Trust
    London, United Kingdom
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Jan 2019 to primary completion in May 2021, 28 months.

Results were first posted in Oct 2024, 3 years 5 months after primary completion.

Of 37 phase 1/2 interventional studies under Anemia, Sickle Cell, half reached primary completion within 82 months of starting (the median), counting those still running; a quarter within 44 and three quarters within 222. This study's 28 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Dec 3, 2018
First posted
Dec 4, 2018
StartActual
Jan 3, 2019
Primary completionActual
May 19, 2021
CompletionActual
Dec 29, 2021
Results first posted
Oct 28, 2024
Last update
Oct 28, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Oct 28, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03763656
Lead sponsor
Nova Laboratories Limited
Responsible party
Sponsor
First posted
Dec 4, 2018
Start date
Jan 3, 2019
Primary completion
May 19, 2021
Completion
Dec 29, 2021
Results posted
Oct 28, 2024
Last update
Oct 28, 2024

Study contacts

Angela E Rankine- Mullings, MD
principal investigator · University of the West Indies, Mona, Kingston, Jamaica

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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