Clinical trial of Methotrexate associated to Hydroxyurea versus placebo and Hydroxyurea in patients with sickle cell disease

Phase II, randomized clinical trial on the anti-inflammatory effect of Methotrexate associated to Hydroxyurea versus placebo and Hydroxyurea in pediatric patients and young adults with sickle cell disease and vaso-occlusive symptoms

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

REBEC

Registry ID

RBR-57qbjv

Enrollment

Unknown

Registered

2018-06-18

Start date

2018-08-01

Completion date

Unknown

Last updated

2025-10-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sickle-cell anaemia with crisis

Interventions

Sickle cell disease patients will be randomized in two arms (172 per arm) and treated as follows: Patients in the experimental arm will receive subcutaneously 10 mg/m2 Methotrexate once a week and 5 m

Drug

E02.319

D03.633.100.733.631.192.500

Eligibility

Age

8 Years to 30 Years

Inclusion criteria

Inclusion criteria: Sickle Cell Disease patients; aged 08-30 years; patients who have vaso-occlusive pain episodes despite the regular use of hydroxyurea in the clinically-adjusted dose for a period equal or superior to 24 weeks

Exclusion criteria

Exclusion criteria: Absence of a signed Subject Information and Consent Form/Parents’ or legal guardian’s Informed Permission; renal or hepatic insufficiency; infection at enrollment time; pregnancy or breastfeeding; regular and/or frequent blood transfusions; refusal of contraceptive measures by females in reproductive age

Design outcomes

Primary

Measure	Time frame
50% or higher reduction of C-Reactive protein serum levels after 24-week treatment with Methotrexate	—

Secondary

Measure	Time frame
50% or higher reduction of C-Reactive protein serum levels after 12-week treatment with Methotrexate ;50% or higher reduction of TNF-alpha, IL-1beta and IL-6 plasma levels after 12 and 24 weeks of treatment;30% or higher reduction of VCAM, ICAM-1, E-Selectin and P-Selectin plasma levels after 12 and 24 weeks of treatment;30% or higher variation of CXCL10 and CXCL12 plasma levels after 12 and 24 weeks of treatment;Rheumatoid factor serum level at enrolment and after 24-week treatment;37% or higher reduction of the number of pain days recorded in a pain diary after the 24-week treatment;Pain intensity measured by the Visual Analogue Scale at enrolment and after 12 and 24 weeks of treatment;Number emergency visits for pain over the 24-week treatment;Maintenance of urea and creatinine serum levels after 12 and 24 weeks of treatment;Variation of health status scores (SF-36 and PedsQL questionnaires) and of depression status (HAD-Hospital Anxiety and Depression Scale) measured at enrolment and after 12 and 24 weeks of treatment	—

Measure

Time frame

50% or higher reduction of C-Reactive protein serum levels after 12-week treatment with Methotrexate ;50% or higher reduction of TNF-alpha, IL-1beta and IL-6 plasma levels after 12 and 24 weeks of treatment;30% or higher reduction of VCAM, ICAM-1, E-Selectin and P-Selectin plasma levels after 12 and 24 weeks of treatment;30% or higher variation of CXCL10 and CXCL12 plasma levels after 12 and 24 weeks of treatment;Rheumatoid factor serum level at enrolment and after 24-week treatment;37% or higher reduction of the number of pain days recorded in a pain diary after the 24-week treatment;Pain intensity measured by the Visual Analogue Scale at enrolment and after 12 and 24 weeks of treatment;Number emergency visits for pain over the 24-week treatment;Maintenance of urea and creatinine serum levels after 12 and 24 weeks of treatment;Variation of health status scores (SF-36 and PedsQL questionnaires) and of depression status (HAD-Hospital Anxiety and Depression Scale) measured at enrolment and after 12 and 24 weeks of treatment

—

Countries

Brazil

Contacts

Public ContactSilvia Brandalise

Centro Infantil Boldrini

silvia@boldrini.org.br+55(019)37875000

Outcome results

None listed

Source: REBEC (via WHO ICTRP)