Evaluation of the effectiveness of hormonal treatment in adolescents suffering from gender dysphoria

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Behavior and Behavior Mechanisms [F01]

Trial duration

25 Jun 2024 → ongoing

Decision date (initial)

2024-09-16

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-513250-30-00

EudraCT number

2019-000300-16

ClinicalTrials.gov

NCT06351501

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

To evaluate, in gender dysphoric adolescents, having completed their social transition, having or not undergone prior pubertal suppression, the effectiveness of hormonal treatment with estrogens or testosterone initiated at 13.5 years +/- 6 months of age on the overall functioning of the teenager at 15.5 years +/- 6 months old.

Secondary objectives 3

1. Evaluate, in gender dysphoric adolescents who have completed their social transition, whether or not they have benefited from prior pubertal suppression: - the effectiveness of hormonal treatment started at 13.5 years old +/- 6 months vs. 15.5years old +/- 6 months on the overall functioning of the adolescent at 17.5 years old +/- 6 months,
2. - the safety (side effects) of hormonal treatment started at age 13.5 +/- 6 months,
3. - and the relevance of hormonal treatment started at 13.5 years old +/- 6 months on other parameters assessed at 15.5 +/- 6 months and 17.5 +/- 6 months years old (gender identity, depression, anxiety, emotional, behavioral disorders and other comorbidities, objective and subjective quality of life, body image, height, waist/hip ratio, bone mineral density, BMI).

Conditions and MedDRA coding

gender dysphoria

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. - Adolescents aged 13.5 years old (+/- 6 months)
2. - Having initiated puberty: tanner score ≥2 for FtM; tanner score ≥2 (clinic and/or testosterone >0.3 ng/mL) for MtF
3. - Presenting the criteria for gender dysphoria according to the DSM5 assessed by at least two child psychiatric interviews at least six months apart where the diagnosis of gender dysphoria was clinically established and that of associated autism spectrum disorder refuted and/or associated cognitive impairment, confirmed by specific scales (Gender Identity / Gender Dysphoria Questionnaire for Adults and Adolescents (GIDYQ-AA) and Utrecht Gender Dysphoria Scale (UGDS))
4. - whose indication for hormonal transition was validated in a multidisciplinary consultation meeting after at least one consultation with the pediatric endocrinologist with clinical examination, blood pressure measurement, and information on hormonal treatments in the context of gender dysphoria.

Exclusion criteria 17

1. - Contraindication to experimental hormonal treatment (see paragraph 1.5)
2. - Patients with cancer with a risk of hypercalcemia (and associated hypercalciuria), linked to bone metastases.
3. - Severe cardiac, hepatic or renal failure or ischemic heart disease, due to the risk of severe complications characterized by edema, with or without congestive heart failure.
4. - Uncontrolled arterial hypertension.
5. - Patients suffering from epilepsy and migraine.
6. - Patients with existence or history of thromboembolic events.
7. - Severe untreated chronic depression.
8. - Insufficient knowledge of French
9. - Severe autism Spectrum Disorder (clinical screening, confirmed in cases of doubt by the Social Responsiveness Scale (SRS) Raw-score > 76, carried out as part of usual care in cases of clinical evidence,
10. - Cognitive deficit (clinical screening, confirmed by an QI < 80 on the Weschler scale (WISC V), carried out as part of the usual treatment in the event of clinical evidence.
11. - Refusal to participate in the study by the adolescent or one of the holders of parental authority (both holders and the adolescent must sign written consent after having received appropriate information).
12. - No social security cover
13. - Participation in another intervention research
14. - Ongoing pregnancy
15. - Need for adjustment of hormonal treatment (FtM patient treated with anticoagulants or having thrombophilia).
16. - Patients with risk of worsening of certain diseases under estrogen treatment (MtF patients with uncontrolled diabetes with HBA1C > 8%, patients with cholelithiasis, biliary lithiasis, systemic lupus erythematosus, severe asthma, severe hypertension, severe migraines, otosclerosis, epilepsy not controlled by treatment.
17. - Current anticoagulant treatment

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary outcome measure is the adolescent's global functioning as assessed by the Children's Global Assessment Scale (CGAS) at age 16 years old +/- 6 months.

Secondary endpoints 17

1. - Global Assesment Scale (GAS) at T2.
2. - Weschler scale (WISC V) at T0 et T2,
3. - The Gender Identity/ Gender Dysphoria Questionnaire for Adolescents and Adults scale (GIDYQ-AA) and the Utrecht Gender Dysphoria Scale (UGDS) at selection visit, T1 et T2,
4. - Beck Depression Inventory (BDI) scale at T0, T1 et T2,
5. - Body Image Scale (BIS) scale at T0, T1 et T2,
6. - State-Trait Anxiety Inventory for Children (STAIC) scale at T0, T1 et T2,
7. - Child Behaviour Checklist (CBCL) scale at T0, T1 et T2,
8. - MINI International Neuropsychiatric Interview (MINI) at selection visit, T1 et T2
9. - Youth Self Report (YSR) scale at T0, T1 et T2,
10. - World Health Organization Quality Of Life - BREF (WHOQOL-BREF) scale at T0, T1 et T2,
11. - Satisfaction With Life Scale (SWLS) at T0, T1 et T2.
12. - Height, bone mineral density, BMI, waist to hip ratio at inclusion visit, T1 et T2.
13. - Potential side-effects at T0, T1 et T2.
14. - The emotional and sexual relationship: assessed by specific questions at T0, T1 et T2
15. - Drug use: assessed by specific questions via the DEP-ADO questionnaire (screening grid for problem substance use among adolescents) at T0, T1 and T2
16. - School drop-out / family breakdown: assessed by specific questions at T0, T1 et T2
17. Psycho-affective criteria will be assessed by a psychologist trained in the relevant assessment scales, while somatic criteria will be collected by the pediatric endocrinologist.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr David Cohen

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr David Cohen

Locations

1 EU/EEA country · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications